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Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors

Primary Purpose

Neuroblastoma, Rhabdomyosarcoma, Ewing's Sarcoma

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
nab-paclitaxel
nab-paclitaxel
Sponsored by
Celgene
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Neuroblastoma focused on measuring Neuroblastoma, Rhabdomyosarcoma, Soft Tissue, Pediatric Oncology, Abraxane, albumin-bound paclitaxel, nab-paclitaxel, ABI-007, taxane, solid tumors

Eligibility Criteria

6 Months - 24 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Patients must meet all of the following criteria to be enrolled in the study:

    1. Patient has a confirmed solid tumor diagnosis according to the

      following:

      1. Phase 1: patient has a recurrent or refractory solid tumor that has

        progressed or did not respond to standard therapy, or for which no

        standard anticancer therapy exists

      2. Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123^I-metaiodobenzylguanidine [MIBG]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma.
    2. The patient has a Lansky/Karnofsky performance status score of ≥ 70%.
    3. The patient has adequate serum chemistry levels, evidenced by the

      following laboratory values

      1. aspartate aminotransferase (AST)/serum glutamic-oxaloacetric

        transaminase (SGOT), alanine aminotransferase (ALT)/serum glutamic

        pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN)

      2. Total bilirubin ≤ 1.5 × ULN
      3. Creatinine ≤ 1.5 × ULN
    4. The patient has adequate bone marrow function, evidenced by the

      following:

      1. Absolute neutrophil count ≥ 1.0 × 10^9 cells/L
      2. Platelets ≥ 80 × 10^9 cells/L (transfusion independent, defined as not

        receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10^9 cells/L

      3. Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion).
    5. The patient (when applicable) or patient's parent(s) or legal guardian(s)

      understand(s) and voluntarily signed an informed consent document prior

      to any study-related assessments/procedures being conducted. Where

      locally applicable, the patient also understands and voluntarily provides

      his/her assent prior to any study-related assessments/procedures being

      conducted.

    6. Male patients of childbearing potential must use a condom during

      sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.

    7. Female patients of childbearing potential [defined as all female

      patients ≥ 12 years old or who have reached menarche, whichever occurs

      first] must have both of the following:

      a. Agree to the use of two physician-approved contraceptive methods

      simultaneously or practice complete abstinence while on study medication or for a longer period if required by regulations.

      i. True abstinence: When this is in line with the preferred and usual

      lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation,

      symptothermal, postovulation methods) and withdrawal are not

      acceptable methods of contraception.

      ii. Acceptable contraceptive methods include: oral, injectable, or

      implantable hormonal contraceptive; tubal ligation; intra-uterine device;

      barrier contraceptive with spermicide; or vasectomized partner) including

      at least one barrier method.

      b. Have negative serum pregnancy test result at screening confirmed by

      negative urine pregnancy dipstick within 72 hours prior to first dose of

      investigational product (if serum test occurred > 72 hours from first

      dose); pregnancy test with sensitivity of at least 25 mIU/mL.

      Exclusion Criteria:

      • The presence of any of the following will exclude a patient from enrollment:
    1. The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.
    2. The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product.
    3. The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product.
    4. The patient has received any investigational therapy ≤ 28 days prior to start of investigational product. Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric.
    5. The patient has received any biological therapy ≤ 7 days prior to the start of investigational product, or monoclonal antibody ≤ 3 half-lives or 28 days, whichever is shorter, prior to the first dose of investigational product.
    6. The patient has received any hematopoietic stem cell transplantation (HSCT) ≤ 3 months prior to start of investigational product.
    7. The patient has received allogeneic hematopoietic stem cell transplantation (HSCT) ≤ 3 months or autologous HSCT ≤ 21 days prior to start of investigational product.
    8. The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma.
    9. The patient has had minor surgery ≤ 7 days from the start of study treatment (excluding the placement of central/peripheral lines, skin biopsy).
    10. The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis.
    11. The patient has a known history or current diagnosis of human immunodeficiency virus (HIV) infection, regardless of treatment status.
    12. The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
    13. The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study.
    14. The patient has any condition, including the presence of laboratory abnormalities, that places the patient at unacceptable risk if he/she were to participate in the study.
    15. The patient has any condition that confounds the ability to interpret data from the study.
    16. The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator.
    17. The patient has ≥ Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.

Sites / Locations

  • Phoenix Childrens Hospital
  • Columbia University Medical Center
  • The Hospital for Sick Children
  • Institute for Pediatric Hematology - Oncology, Leon Berard Cancer Center
  • Hopital d'Enfants, CHU Nancy
  • Institut Curie
  • Institut Gustave Roussy
  • Azienda Ospedaliera Universitaria Meyer
  • Children's Hospital Largo
  • Istituto Nazionale Tumori
  • Clinica di Oncoematologia
  • Policlinico Agostino Gemelli
  • l'Azienda Ospedaliera Regina Margherita - Sant Anna
  • Hospital Universitario Vall D Hebron
  • Hospital Sant Joan de Deu
  • Spanish National Cancer Research Centre
  • Hospital Universitario Virgen Del Rocio
  • Unidad de Oncologia Pediatrica, Hospital Universitario la Fe
  • Universitäts-Kinderklinik
  • Royal Marsden Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm 9

Arm 10

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

nab-paclitaxel

Phase 1: Nab-Paclitaxel 120 mg/m^2

Phase 1: Nab-Paclitaxel 150 mg/m^2

Phase 1: Nab-Paclitaxel 180 mg/m^2

Phase 1: Nab-Paclitaxel 210 mg/m^2

Phase 1: Nab-Paclitaxel 240 mg/m^2

Phase 1: Nab-Paclitaxel 270 mg/m^2

Phase 2: Ewing's Sarcoma

Phase 2: Neuroblastoma

Phase 2: Rhabdomyosarcoma

Arm Description

nab-paclitaxel 100-240 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle.

nab-paclitaxel 120 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.

Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.

Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.

Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.

Outcomes

Primary Outcome Measures

Phase 1: Number of Participants With Dose Limiting Toxicities (DLTs)
A DLT was defined as investigational product (IP)-related adverse events occurring during the DLT assessment period that led to treatment discontinuation or met one of the following criteria: - Common Terminology Criteria for Adverse Events (CTCAE) Grade (Gr) 3 or 4 nonhematologic toxicity (excluding transient transaminitis) - CTCAE Gr 3 or 4 nausea or vomiting that persisted > 5 days despite maximal anti-emetic treatment - CTCAE Gr 4 thrombocytopenia or anemia that persisted > 7 days or required transfusion > 7 days - CTCAE Gr 3 thrombocytopenia with bleeding - CTCAE Gr 4 uncomplicated neutropenia lasting > 7 days - Febrile neutropenia with confirmed bacterial infection - CTCAE Gr 3 hematologic toxicity requiring treatment (tx) delay > 21 days. Use of "..." in the table rows signifies the continuation of row title per the above list.
Phase 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An adverse event (AE) was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE (SAE) is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of an AE was graded according to the CTCAE, Version 4.0.
Phase 2: Overall Response Rate (ORR)
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR; at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 guidelines. (For Phase 2 neuroblastoma participants who had both RECIST and Curie Score tumor evaluations, both tumor response results were considered and an overall response was derived.) Confidence interval was obtained using the Clopper-Pearson method.

Secondary Outcome Measures

Phase 1: ORR
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met) using RECIST version 1.1 guidelines over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Phase 1: Maximum Observed Concentration of Paclitaxel in Blood Plasma (Cmax)
Phase 1: Cmax - Dose-Normalized
Phase 1: Area Under the Plasma Concentration-Time Curve (AUC)
Measurements include: AUC from time zero to the last measurable concentration (AUCt), AUC from time zero to 24 hours (AUC24), and AUC from time zero to infinity (AUCinf).
Phase 1: AUC - Dose-Normalized
Measurements include: AUC24 and AUCinf.
Phase 1: Clearance (CL)
Measurement of renal clearance from the body.
Phase 1: CL - Body Surface Area (BSA)-Normalized
Measurement of renal clearance from the body.
Phase 1: Volume of Distribution (Vss)
Phase 1: Vss - BSA-Normalized
Phase 1 and 2 Population PK: Volume of Distribution of the Central Compartment (V1)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V1 was 0.888.
Phase 1 and 2 Population PK: Maximum Elimination Rate From the Central Compartment (VMEL)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for VMEL was 1.12.
Phase 1 and 2 Population PK: Concentration in the Central Compartment at 50% of VMEL (KMEL)
Population PK analysis was performed using nonlinear mixed effect modeling.
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the First Peripheral Compartment (Q2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q2 was 1.12.
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the Second Peripheral Compartment (Q3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q3 was 1.12.
Phase 1 and 2 Population PK: Volume of Distribution of the First Peripheral Compartment (V2)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V2 was 0.888.
Phase 1 and 2 Population PK: Volume of Distribution of the Second Peripheral Compartment (V3)
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V3 was 0.888.
Phase 2: Duration of Response (DOR)
Duration of response was defined as the time from the date of the first response (CR/PR, using RECIST version 1.1 guidelines) to disease progression for participants with a confirmed CR or PR. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at time of start of new anticancer therapy, whichever occurred first. (For Phase 2 neuroblastoma patients who had both RECIST version 1.1 and Curie Score tumor evaluations, both tumor responses results were considered and an overall response was derived.)
Phase 2: Disease Control Rate (DCR)
Disease control rate was defined as the percentage of participants who achieved either a stable disease maintained for ≥ 16 weeks or confirmed CR (confirmed no less than 4 weeks after criteria for response were first met) or confirmed PR (confirmed no less than 4 weeks after criteria for response were first met) over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Phase 2: Progression-Free Survival (PFS)
PFS was defined as the time from the first dose date to the start of disease progression or participant death (any cause), whichever occurred first. Disease progression was classed as either a disease progression observed as a response assessment, or a disease progression or symptomatic deterioration at treatment/study discontinuation. Participants who did not have disease progression or had not died were censored at the last known time that the participant was progression free. Disease progression was considered according to RECIST version 1.1 for Phase 2 Ewing's sarcoma and rhabdomyosarcoma participants. (For Phase 2 neuroblastoma participants who had both RECIST 1.1 and Curie score tumor evaluations, both tumor responses results were considered and an overall response was derived.) Median PFS time was estimated through Kaplan-Meier methods. 95% confidence interval about the median time to PFS event was obtained using Greenwood's method.
Phase 2: Kaplan-Meier Estimate of Overall Survival Rate at 1 Year
Overall survival was defined as the time from the first dose date to date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive.
Phase 2: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
An AE was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A SAE is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of the AEs was graded according to the Common Terminology Criteria for Adverse Events, Version 4.0. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.

Full Information

First Posted
September 27, 2013
Last Updated
December 19, 2019
Sponsor
Celgene
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1. Study Identification

Unique Protocol Identification Number
NCT01962103
Brief Title
Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors
Official Title
A Phase 1/2, Multicenter, Open-label, Dose-finding Study to Assess the Safety, Tolerability, and Preliminary Efficacy of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent or Refractory Solid Tumors.
Study Type
Interventional

2. Study Status

Record Verification Date
December 2019
Overall Recruitment Status
Completed
Study Start Date
December 4, 2013 (Actual)
Primary Completion Date
December 5, 2017 (Actual)
Study Completion Date
November 6, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Celgene

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The purpose of this study is to find the safe dose of nab-paclitaxel in children with solid tumors, and to see if it works to treat these solid tumors in children and young adults (in Phase 1 ≤ 18 years old and in Phase 2 ≤ 24 years old). After the final dose has been chosen, patients will be enrolled according to the specific solid tumor type, (neuroblastoma, rhabdomyosarcoma, or Ewing's sarcoma), to see how nab-paclitaxel works in treating these tumors.
Detailed Description
ABI-007-PST-001 is a Phase 1/2, multicenter, open-label, dose-finding study to assess the safety , tolerability, and preliminary efficacy of weekly nab-paclitaxel in pediatric patients with recurrent or refractory solid tumors (excluding brain tumors). The Phase 1 portion of the study, with a dose escalation design, ended and the recommended Phase 2 dose (RP2D) was determined as 240 mg/m^2 intravenously (IV) in patients weighing > 10 kg and 11.5 mg/kg in patients weighing ≤ 10 kg, on Days 1, 8 and 15 of a 28-day cycle. The Phase 2 portion of the study will enroll additional patients at the RP2D into 1 of 3 solid tumor groups [neuroblastomas, rhabdomyosarcomas, Ewing's sarcomas]. Both phases of the study are open-label and conducted at multiple centers. The Phase 2 is using a Simon 2-stage design to monitor patient enrollment for each group separately. The rhabdomyosarcoma group, neuroblastoma or Ewing's sarcoma groups did not reach the expected number of 2 responders out of 14 efficacy eligible patients. Consequently, the groups were stopped.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuroblastoma, Rhabdomyosarcoma, Ewing's Sarcoma, Ewing's Tumor, Sarcoma, Ewing's, Sarcomas, Epitheliod, Sarcoma, Soft Tissue, Sarcoma, Spindle Cell, Melanoma, Malignant Melanoma, Clinical Oncology, Oncology, Medical, Pediatrics, Osteosarcoma, Osteogenic Sarcoma, Osteosarcoma Tumor, Sarcoma, Osteogenic, Tumors, Cancer, Neoplasia, Neoplasm, Histiocytoma, Fibrosarcoma, Dermatofibrosarcoma
Keywords
Neuroblastoma, Rhabdomyosarcoma, Soft Tissue, Pediatric Oncology, Abraxane, albumin-bound paclitaxel, nab-paclitaxel, ABI-007, taxane, solid tumors

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
107 (Actual)

8. Arms, Groups, and Interventions

Arm Title
nab-paclitaxel
Arm Type
Experimental
Arm Description
nab-paclitaxel 100-240 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle.
Arm Title
Phase 1: Nab-Paclitaxel 120 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 120 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 1: Nab-Paclitaxel 150 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 150 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 1: Nab-Paclitaxel 180 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 180 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 1: Nab-Paclitaxel 210 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 210 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 1: Nab-Paclitaxel 240 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 240 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 1: Nab-Paclitaxel 270 mg/m^2
Arm Type
Experimental
Arm Description
nab-paclitaxel 270 mg/m^2 IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity to establish the RP2D.
Arm Title
Phase 2: Ewing's Sarcoma
Arm Type
Experimental
Arm Description
Participants with Ewing's sarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Arm Title
Phase 2: Neuroblastoma
Arm Type
Experimental
Arm Description
Participants with neuroblastoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Arm Title
Phase 2: Rhabdomyosarcoma
Arm Type
Experimental
Arm Description
Participants with rhabdomyosarcoma: nab-paclitaxel at the RP2D (240 mg/m^2 in participants weighing > 10 kg and 11.5 mg/kg in participants weighing ≤ 10 kg) IV on Days 1, 8 and 15 of a 28-day cycle until disease progression, death, withdrawal of consent, or unacceptable toxicity.
Intervention Type
Drug
Intervention Name(s)
nab-paclitaxel
Other Intervention Name(s)
Abraxane
Intervention Description
nab-paclitaxel 120-270 mg/m2 IV on Days 1, 8 and 15 of a 28-day cycle
Intervention Type
Drug
Intervention Name(s)
nab-paclitaxel
Other Intervention Name(s)
Abraxane
Intervention Description
IV infusion
Primary Outcome Measure Information:
Title
Phase 1: Number of Participants With Dose Limiting Toxicities (DLTs)
Description
A DLT was defined as investigational product (IP)-related adverse events occurring during the DLT assessment period that led to treatment discontinuation or met one of the following criteria: - Common Terminology Criteria for Adverse Events (CTCAE) Grade (Gr) 3 or 4 nonhematologic toxicity (excluding transient transaminitis) - CTCAE Gr 3 or 4 nausea or vomiting that persisted > 5 days despite maximal anti-emetic treatment - CTCAE Gr 4 thrombocytopenia or anemia that persisted > 7 days or required transfusion > 7 days - CTCAE Gr 3 thrombocytopenia with bleeding - CTCAE Gr 4 uncomplicated neutropenia lasting > 7 days - Febrile neutropenia with confirmed bacterial infection - CTCAE Gr 3 hematologic toxicity requiring treatment (tx) delay > 21 days. Use of "..." in the table rows signifies the continuation of row title per the above list.
Time Frame
DLT assessment period: For participants > 10 kg: the first 28-day cycle including Cycle 2 Day 1 predose evaluations; for participants ≤ 10 kg: the first two 28-day cycles including Cycle 3 Day 1 predose evaluations
Title
Phase 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Description
An adverse event (AE) was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A serious AE (SAE) is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of an AE was graded according to the CTCAE, Version 4.0.
Time Frame
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
Title
Phase 2: Overall Response Rate (ORR)
Description
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR; at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met using Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 guidelines. (For Phase 2 neuroblastoma participants who had both RECIST and Curie Score tumor evaluations, both tumor response results were considered and an overall response was derived.) Confidence interval was obtained using the Clopper-Pearson method.
Time Frame
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
Secondary Outcome Measure Information:
Title
Phase 1: ORR
Description
Overall response rate was defined as the percentage of participants who achieved a complete response (CR; disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions) confirmed no less than 4 weeks after the criteria for response were first met) using RECIST version 1.1 guidelines over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Time Frame
Median treatment duration in Phase 1 was 7.0 weeks, with minimum and maximum duration of 1 and 49 weeks, respectively.
Title
Phase 1: Maximum Observed Concentration of Paclitaxel in Blood Plasma (Cmax)
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: Cmax - Dose-Normalized
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: Area Under the Plasma Concentration-Time Curve (AUC)
Description
Measurements include: AUC from time zero to the last measurable concentration (AUCt), AUC from time zero to 24 hours (AUC24), and AUC from time zero to infinity (AUCinf).
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: AUC - Dose-Normalized
Description
Measurements include: AUC24 and AUCinf.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: Clearance (CL)
Description
Measurement of renal clearance from the body.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: CL - Body Surface Area (BSA)-Normalized
Description
Measurement of renal clearance from the body.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: Volume of Distribution (Vss)
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1: Vss - BSA-Normalized
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants <6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Volume of Distribution of the Central Compartment (V1)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V1 was 0.888.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Maximum Elimination Rate From the Central Compartment (VMEL)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for VMEL was 1.12.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Concentration in the Central Compartment at 50% of VMEL (KMEL)
Description
Population PK analysis was performed using nonlinear mixed effect modeling.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the First Peripheral Compartment (Q2)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q2 was 1.12.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Intercompartmental CL Between the Central Compartment and the Second Peripheral Compartment (Q3)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for Q3 was 1.12.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Volume of Distribution of the First Peripheral Compartment (V2)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V2 was 0.888.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 1 and 2 Population PK: Volume of Distribution of the Second Peripheral Compartment (V3)
Description
Population PK analysis was performed using nonlinear mixed effect modeling. The estimated allometric function for V3 was 0.888.
Time Frame
Cycle 1 Day 1 (Participants ≥ 6 years: 1-2 minutes prior to the end of infusion [EOI], and 15 minutes, 1, 3, 5, 8, 24, 48, and 72 hours after the EOI. Participants < 6 years: 1-2 minutes prior to the EOI, and 15 minutes, 3, 5, and 24 hours after the EOI.)
Title
Phase 2: Duration of Response (DOR)
Description
Duration of response was defined as the time from the date of the first response (CR/PR, using RECIST version 1.1 guidelines) to disease progression for participants with a confirmed CR or PR. Participants who did not have disease progression or had not died were censored at the time of their last disease assessment or at time of start of new anticancer therapy, whichever occurred first. (For Phase 2 neuroblastoma patients who had both RECIST version 1.1 and Curie Score tumor evaluations, both tumor responses results were considered and an overall response was derived.)
Time Frame
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
Title
Phase 2: Disease Control Rate (DCR)
Description
Disease control rate was defined as the percentage of participants who achieved either a stable disease maintained for ≥ 16 weeks or confirmed CR (confirmed no less than 4 weeks after criteria for response were first met) or confirmed PR (confirmed no less than 4 weeks after criteria for response were first met) over the total number of participants available for the analysis. Confidence interval was obtained using the Clopper-Pearson method.
Time Frame
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13).
Title
Phase 2: Progression-Free Survival (PFS)
Description
PFS was defined as the time from the first dose date to the start of disease progression or participant death (any cause), whichever occurred first. Disease progression was classed as either a disease progression observed as a response assessment, or a disease progression or symptomatic deterioration at treatment/study discontinuation. Participants who did not have disease progression or had not died were censored at the last known time that the participant was progression free. Disease progression was considered according to RECIST version 1.1 for Phase 2 Ewing's sarcoma and rhabdomyosarcoma participants. (For Phase 2 neuroblastoma participants who had both RECIST 1.1 and Curie score tumor evaluations, both tumor responses results were considered and an overall response was derived.) Median PFS time was estimated through Kaplan-Meier methods. 95% confidence interval about the median time to PFS event was obtained using Greenwood's method.
Time Frame
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed until disease progression (if applicable) up to a maximum of 100.3 weeks.
Title
Phase 2: Kaplan-Meier Estimate of Overall Survival Rate at 1 Year
Description
Overall survival was defined as the time from the first dose date to date of death (any cause). Participants who were alive were censored at the last known time that the participant was alive.
Time Frame
1 year
Title
Phase 2: Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Description
An AE was defined as any noxious, unintended, or untoward medical occurrence that may appear or worsen in a participant during the course of a study. A SAE is any AE occurring at any dose that: results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. TEAEs were defined as AEs that began or worsened in severity on or after the date of the first dose of study drug and within 28 days of the date of the last dose of study drug. The severity of the AEs was graded according to the Common Terminology Criteria for Adverse Events, Version 4.0. Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.
Time Frame
Median treatment duration in Phase 2 per group: Ewings Sarcoma = 14 weeks (3-31), Neuroblastoma = 7 weeks (3-23), Rhabdomyosarcoma = 5 weeks (1-13). Participants were followed for 28 days after discontinuing treatment for safety and monitoring of AEs.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
24 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patients must meet all of the following criteria to be enrolled in the study: Patient has a confirmed solid tumor diagnosis according to the following: Phase 1: patient has a recurrent or refractory solid tumor that has progressed or did not respond to standard therapy, or for which no standard anticancer therapy exists Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123^I-metaiodobenzylguanidine [MIBG]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma. The patient has a Lansky/Karnofsky performance status score of ≥ 70%. The patient has adequate serum chemistry levels, evidenced by the following laboratory values aspartate aminotransferase (AST)/serum glutamic-oxaloacetric transaminase (SGOT), alanine aminotransferase (ALT)/serum glutamic pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN) Total bilirubin ≤ 1.5 × ULN Creatinine ≤ 1.5 × ULN The patient has adequate bone marrow function, evidenced by the following: Absolute neutrophil count ≥ 1.0 × 10^9 cells/L Platelets ≥ 80 × 10^9 cells/L (transfusion independent, defined as not receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10^9 cells/L Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion). The patient (when applicable) or patient's parent(s) or legal guardian(s) understand(s) and voluntarily signed an informed consent document prior to any study-related assessments/procedures being conducted. Where locally applicable, the patient also understands and voluntarily provides his/her assent prior to any study-related assessments/procedures being conducted. Male patients of childbearing potential must use a condom during sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication. Female patients of childbearing potential [defined as all female patients ≥ 12 years old or who have reached menarche, whichever occurs first] must have both of the following: a. Agree to the use of two physician-approved contraceptive methods simultaneously or practice complete abstinence while on study medication or for a longer period if required by regulations. i. True abstinence: When this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception. ii. Acceptable contraceptive methods include: oral, injectable, or implantable hormonal contraceptive; tubal ligation; intra-uterine device; barrier contraceptive with spermicide; or vasectomized partner) including at least one barrier method. b. Have negative serum pregnancy test result at screening confirmed by negative urine pregnancy dipstick within 72 hours prior to first dose of investigational product (if serum test occurred > 72 hours from first dose); pregnancy test with sensitivity of at least 25 mIU/mL. Exclusion Criteria: The presence of any of the following will exclude a patient from enrollment: The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis. The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product. The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product. The patient has received any investigational therapy ≤ 28 days prior to start of investigational product. Investigational therapy is defined as any medicinal product that is not approved in the country of treatment for any indication, adult or pediatric. The patient has received any biological therapy ≤ 7 days prior to the start of investigational product, or monoclonal antibody ≤ 3 half-lives or 28 days, whichever is shorter, prior to the first dose of investigational product. The patient has received any hematopoietic stem cell transplantation (HSCT) ≤ 3 months prior to start of investigational product. The patient has received allogeneic hematopoietic stem cell transplantation (HSCT) ≤ 3 months or autologous HSCT ≤ 21 days prior to start of investigational product. The patient has not recovered from the acute toxic effects of prior chemotherapy, radiation, or major surgery/significant trauma. The patient has had minor surgery ≤ 7 days from the start of study treatment (excluding the placement of central/peripheral lines, skin biopsy). The patient has a known history of stroke, myocardial infarction, peripheral vascular disease, or recent (within 3 months) uncontrolled deep venous thrombosis. The patient has a known history or current diagnosis of human immunodeficiency virus (HIV) infection, regardless of treatment status. The patient has an uncontrolled intercurrent illness including but not limited to ongoing or active infection requiring antibiotic, antifungal, or antiviral therapy, symptomatic heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. The patient has any significant medical condition, laboratory abnormality, or psychiatric illness that would prevent the patient from participating in the study. The patient has any condition, including the presence of laboratory abnormalities, that places the patient at unacceptable risk if he/she were to participate in the study. The patient has any condition that confounds the ability to interpret data from the study. The patient or parent(s)/guardian(s) is/are unable to comply with the study visit schedule and other protocol requirements, in the opinion of the investigator. The patient has ≥ Grade 2 peripheral neuropathy by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) at screening.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Ileana Elias, M.D.
Organizational Affiliation
Celgene Corporation
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Childrens Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
The Hospital for Sick Children
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X8
Country
Canada
Facility Name
Institute for Pediatric Hematology - Oncology, Leon Berard Cancer Center
City
Lyon
ZIP/Postal Code
69008
Country
France
Facility Name
Hopital d'Enfants, CHU Nancy
City
Nancy
ZIP/Postal Code
54000
Country
France
Facility Name
Institut Curie
City
Paris
ZIP/Postal Code
75005
Country
France
Facility Name
Institut Gustave Roussy
City
Villejuif
ZIP/Postal Code
94805
Country
France
Facility Name
Azienda Ospedaliera Universitaria Meyer
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Children's Hospital Largo
City
Genova
ZIP/Postal Code
16147
Country
Italy
Facility Name
Istituto Nazionale Tumori
City
Milan
Country
Italy
Facility Name
Clinica di Oncoematologia
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Policlinico Agostino Gemelli
City
Rome
ZIP/Postal Code
00168
Country
Italy
Facility Name
l'Azienda Ospedaliera Regina Margherita - Sant Anna
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
Hospital Universitario Vall D Hebron
City
Barcelona
ZIP/Postal Code
8035
Country
Spain
Facility Name
Hospital Sant Joan de Deu
City
Barcelona
ZIP/Postal Code
8950
Country
Spain
Facility Name
Spanish National Cancer Research Centre
City
Madrid
ZIP/Postal Code
28029
Country
Spain
Facility Name
Hospital Universitario Virgen Del Rocio
City
Sevilla
ZIP/Postal Code
41013
Country
Spain
Facility Name
Unidad de Oncologia Pediatrica, Hospital Universitario la Fe
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Universitäts-Kinderklinik
City
Zurich
ZIP/Postal Code
8032
Country
Switzerland
Facility Name
Royal Marsden Hospital
City
Sutton
ZIP/Postal Code
SM2 5PT
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
29936064
Citation
Moreno L, Casanova M, Chisholm JC, Berlanga P, Chastagner PB, Baruchel S, Amoroso L, Gallego Melcon S, Gerber NU, Bisogno G, Fagioli F, Geoerger B, Glade Bender JL, Aerts I, Bergeron C, Hingorani P, Elias I, Simcock M, Ferrara S, Le Bruchec Y, Slepetis R, Chen N, Vassal G. Phase I results of a phase I/II study of weekly nab-paclitaxel in paediatric patients with recurrent/refractory solid tumours: A collaboration with innovative therapies for children with cancer. Eur J Cancer. 2018 Sep;100:27-34. doi: 10.1016/j.ejca.2018.05.002. Epub 2018 Jun 21.
Results Reference
background
PubMed Identifier
32554315
Citation
Amoroso L, Castel V, Bisogno G, Casanova M, Marquez-Vega C, Chisholm JC, Doz F, Moreno L, Ruggiero A, Gerber NU, Fagioli F, Hingorani P, Melcon SG, Slepetis R, Chen N, le Bruchec Y, Simcock M, Vassal G. Phase II results from a phase I/II study to assess the safety and efficacy of weekly nab-paclitaxel in paediatric patients with recurrent or refractory solid tumours: A collaboration with the European Innovative Therapies for Children with Cancer Network. Eur J Cancer. 2020 Aug;135:89-97. doi: 10.1016/j.ejca.2020.04.031. Epub 2020 Jun 15.
Results Reference
derived
PubMed Identifier
31401903
Citation
Hattinger CM, Patrizio MP, Magagnoli F, Luppi S, Serra M. An update on emerging drugs in osteosarcoma: towards tailored therapies? Expert Opin Emerg Drugs. 2019 Sep;24(3):153-171. doi: 10.1080/14728214.2019.1654455. Epub 2019 Aug 14.
Results Reference
derived

Learn more about this trial

Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors

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