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Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia (SOAR)

Primary Purpose

Severe Aplastic Anemia

Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
eltrombopag
Cyclosporine
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Severe Aplastic Anemia focused on measuring severe acquired aplastic anemia, SAA, first line, eltrombopag, cyclosporine, h-ATG, severe aplastic anemia, aplastic anemia

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Patient has signed the Informed Consent (ICF) prior to any screening procedures being performed.
  2. Patient is male/female ≥18 years old at the time of informed consent and able to swallow a tablet.
  3. Patient has SAA characterized by:

    1. Bone marrow cellularity <30% (excluding lymphocytes) and
    2. At least two of the following (peripheral blood):

      • Absolute neutrophil count <500/µL
      • Platelet count <20,000/µL
      • Absolute reticulocyte count <60,000/µL
  4. Normal ECG defined as the following as determined via the mean of a triplicate ECG

    • Resting heart rate: 50-90 bpm
    • QTcF at screening <450 msec (for male patients), ≤460 msec (for female patients)

Exclusion Criteria:

  1. Diagnosis of Fanconi anemia.
  2. Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review
  3. Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists.
  4. a. Hypersensitivity to eltrombopag or cyclosporine or their components. b. Contraindications to cyclospsorine.
  5. AST or ALT >3 x ULN.
  6. Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN.
  7. Patient with liver cirrhosis.
  8. a. Infection not adequately controlled with appropriate therapy. b. Patients who are human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients are allowed to be enrolled.
  9. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely.
  10. Patients with cancer who are not considered cure, are on active chemotherapeutic treatment or who take drugs with hematological effects.
  11. Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
  12. Pregnancy statements and contraception requirements:

    Pregnancy or nursing (lactating) women Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they are using highly effective methods of contraception during dosing and for 3 months after stopping medication.

  13. Not able to understand the investigation nature of the study or to give informed consent.
  14. Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG.
  15. Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome.
  16. Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that cannot be discontinued or replaced by safe alternative medication per www.qtdrugs.org.
  17. ECOG performance status of ≥2.
  18. Patients under the age of 40 must be referred for consideration of allogeneic bone marrow transplantation (HSCT) if (human leukocyte antigen) HLA matching has been done and a suitable matched sibling donor is available and the patient is willing to undergo transplantation (i.e. patients who do not have a HLA match or are not medically fit, not willing or unable to undergo transplantation will be considered for enrollment).

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Eltrombopag + cyclosporine

Arm Description

Planned duration of treatment with eltrombopag & cyclosporine is 6 months (for all patients); the planned duration of treatment with cyclosporine (cyclosporine tapering) is 18 months (for responder patients only).

Outcomes

Primary Outcome Measures

Overall hematologic response (CR + PR) rate - 6 month
Overall hematologic response = patients with complete response (CR) + patients with partial response (PR). Partial response is defined as any two of the following parameters at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >60 000/μL Complete response is defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L

Secondary Outcome Measures

Overall hematologic response (CR + PR) rate - 3, 12 and 24 month
Overall hematologic response = patients with complete response (CR) + patients with partial response (PR). Partial response is defined as any two of the following parameters at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >60 000/μL Complete response is defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L
Duration of hematologic response
Time from the date of the start of first response to the date of first relapse (defined as no longer meeting definition of PR (and not CR)
Proportion of patients who relapse
Percentage of patients who relapse. Relapse is defined as no longer meeting the definition of PR (and not CR).
Percentage of patients with clonal evolution to myelodysplasia, paroxysmal nocturnal hemoglobinuria (PNH), and leukemia
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.
Percentage of patients who are red blood cells (RBC) transfusion independent
Percentage of patients who are RBC transfusion independent at least once by 6 months and by 24 months. Independence defined as no RBC transfusion for at least 56 days.
Percentage of patients who are platelet transfusion independence
Percentage of patients who are platelet transfusion independent at least once by 6 months and by 24 months. Independence defined as no platelet transfusion for at least 28 days.
Longest interval without platelet or RBC transfusion
Duration of longest interval without a platelet or RBC transfusion by 6 months and by 24 months.
Change from baseline in scores of FACIT-Fatigue Patient Reported Outcome
FACIT-Fatigue responses will be generated in accordance with the respective scoring manual. Patients with an evaluable baseline score and at least one evaluable post baseline score during the treatment period will be included in the change from baseline analyses. The FACT- Fatigue is a 13-item fatigue subscale that asks the patient to rate their degree of tiredness, weakness and fatigue. All items of the FACT-Fatigue use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total score ranges from 0 to 52. High scores represent less fatigue.
Change from baseline in scores of FACT-TH18 Patient Reported Outcome
FACT-TH18 responses will be generated in accordance with the respective scoring manual. Patients with an evaluable baseline score and at least one evaluable post baseline score during the treatment period will be included in the change from baseline analyses. FACT-TH18 is comprised of FACT-G and a thrombocytopenia specific questionnaire. FACT-G consists of 27-items divided into 4 QOL domains (physical well-being, social/Family well-being, emotional and functional well-being). FACT-TH18 has 18-items which asks the patient to rate degree of thrombocytopenia. All items of the FACT-TH18 use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much".
Frequency and severity of AEs and serious AEs (SAEs)
Safety will be assessed by frequency and severity of AEs, serious AEs (SAEs) based on the CTCAE version 4. 03, and AEs leading to discontinuation, and evaluating changes in laboratory values within 6 months and within 24 months (responders only)
Pharmacokinetic parameter- Cmax of eltrombopag when combined with cyclosporine
Cmax=Observed maximum plasma concentration following administration (mass/volume). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Pharmacokinetic parameter-AUClast of eltrombopag when combined with cyclosporine
AUClast=Area under the curve calculated to the last quantifiable concentration point (Tlast). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Pharmacokinetic parameter- AUCtau of eltrombopag when combined with cyclosporine
AUCtau=Area under the curve calculated to the end of the dosing interval ( tau). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Pharmacokinetic parameter- Ctrough of eltrombopag when combined with cyclosporine
Ctrough=Pre-dose plasma concentration (mass/volume). Observed maximum plasma concentration following administration (mass/volume). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Pharmacokinetic parameter- Tmax of eltrombopag when combined with cyclosporine
Tmax=The time to reach peak or maximum concentration. The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Pharmacokinetic parameter- CLss/F of eltrombopag when combined with cyclosporine
CLss/F=Apparent systemic (or total body) clearance at steady state from plasma. The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly

Full Information

First Posted
November 4, 2016
Last Updated
June 29, 2022
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02998645
Brief Title
Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia
Acronym
SOAR
Official Title
SOAR, Interventional Phase II Single-arm Study to Assess Efficacy and Safety of Eltrombopag Combined With Cyclosporine as First Line Therapy in Adult Patients With Severe Acquired Aplastic Anemia
Study Type
Interventional

2. Study Status

Record Verification Date
June 2022
Overall Recruitment Status
Completed
Study Start Date
May 11, 2017 (Actual)
Primary Completion Date
November 3, 2020 (Actual)
Study Completion Date
May 2, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

Brief Summary
This interventional Phase II, single-arm, multicenter, open-label study will investigate the efficacy and safety of a combination regimen of 6 months eltrombopag and cyclosporine treatment in adult patients with severe aplastic anemia (SAA) as first line therapy, with an additional 18 months follow-up for cyclosporine tapering and duration of response until relapse or 24 months whichever is earlier (responders only who do not relapse prior to 6 months). The usage of eltrombopag and cyclosporine combines two therapies with different modes of action. Cyclosporine acts as an immunosuppressant and eltrombopag acts as a stimulator of bone marrow progenitor cells. Given that SAA is currently viewed as having an autoimmune pathogenesis resulting in bone marrow progenitor cell destruction, the combination of eltrombopag and cyclosporine is attractive. Preliminary experience with their combined use appears favorable, with no untoward toxicity observed to date.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Severe Aplastic Anemia
Keywords
severe acquired aplastic anemia, SAA, first line, eltrombopag, cyclosporine, h-ATG, severe aplastic anemia, aplastic anemia

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
54 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Eltrombopag + cyclosporine
Arm Type
Experimental
Arm Description
Planned duration of treatment with eltrombopag & cyclosporine is 6 months (for all patients); the planned duration of treatment with cyclosporine (cyclosporine tapering) is 18 months (for responder patients only).
Intervention Type
Drug
Intervention Name(s)
eltrombopag
Intervention Description
Film-coated tablets (12.5 mg, 25 mg, 50 mg and 75 mg) administered orally, once daily. Dosing is done according to age and ethnicity (East Asian)
Intervention Type
Drug
Intervention Name(s)
Cyclosporine
Intervention Description
Supplied in oral soft gel capsules or oral solution and dosage is based on body weight and administered every 12 hours. Dosing is titrated individually according to therapeutic trough level for 6 months. After 6 months (for responders), tapering of cyclosporine must be done as follows: 6-9 months: at the 6 months visit, the dose must be reduced by 25% for 3 months 9-12 months: at the 9 months visit, the dose must be further reduced by 25% for another 3 months 12-24 months: maintain dose
Primary Outcome Measure Information:
Title
Overall hematologic response (CR + PR) rate - 6 month
Description
Overall hematologic response = patients with complete response (CR) + patients with partial response (PR). Partial response is defined as any two of the following parameters at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >60 000/μL Complete response is defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L
Time Frame
by 6 months
Secondary Outcome Measure Information:
Title
Overall hematologic response (CR + PR) rate - 3, 12 and 24 month
Description
Overall hematologic response = patients with complete response (CR) + patients with partial response (PR). Partial response is defined as any two of the following parameters at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) >500/μL Platelet count >20 000/μL Reticulocyte count >60 000/μL Complete response is defined as all three parameters meet the following criteria at two consecutive measurements at least 7 days apart during the study: Absolute neutrophil count (ANC) > 1 000/μL Platelet count >100 000/μL Hemoglobin >10 g/L
Time Frame
by 3 months (all patients) and at 12 and 24 months (responders only)
Title
Duration of hematologic response
Description
Time from the date of the start of first response to the date of first relapse (defined as no longer meeting definition of PR (and not CR)
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Proportion of patients who relapse
Description
Percentage of patients who relapse. Relapse is defined as no longer meeting the definition of PR (and not CR).
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Percentage of patients with clonal evolution to myelodysplasia, paroxysmal nocturnal hemoglobinuria (PNH), and leukemia
Description
Clonal evolution to myelodysplasia is defined as a new marrow cytogenic abnormality with or without characteristic dysplastic marrow findings. Evolution to leukemia is defined as greater than 20% peripheral blood and/or marrow blasts. Evolution to paroxysmal nocturnal hemoglobinuria (PNH) is defined as a clone at baseline < 10% that rose to greater than 50% on study.
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Percentage of patients who are red blood cells (RBC) transfusion independent
Description
Percentage of patients who are RBC transfusion independent at least once by 6 months and by 24 months. Independence defined as no RBC transfusion for at least 56 days.
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Percentage of patients who are platelet transfusion independence
Description
Percentage of patients who are platelet transfusion independent at least once by 6 months and by 24 months. Independence defined as no platelet transfusion for at least 28 days.
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Longest interval without platelet or RBC transfusion
Description
Duration of longest interval without a platelet or RBC transfusion by 6 months and by 24 months.
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Change from baseline in scores of FACIT-Fatigue Patient Reported Outcome
Description
FACIT-Fatigue responses will be generated in accordance with the respective scoring manual. Patients with an evaluable baseline score and at least one evaluable post baseline score during the treatment period will be included in the change from baseline analyses. The FACT- Fatigue is a 13-item fatigue subscale that asks the patient to rate their degree of tiredness, weakness and fatigue. All items of the FACT-Fatigue use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much". Total score ranges from 0 to 52. High scores represent less fatigue.
Time Frame
Baseline and by 6 months (all patients) and 24 months (responders only)
Title
Change from baseline in scores of FACT-TH18 Patient Reported Outcome
Description
FACT-TH18 responses will be generated in accordance with the respective scoring manual. Patients with an evaluable baseline score and at least one evaluable post baseline score during the treatment period will be included in the change from baseline analyses. FACT-TH18 is comprised of FACT-G and a thrombocytopenia specific questionnaire. FACT-G consists of 27-items divided into 4 QOL domains (physical well-being, social/Family well-being, emotional and functional well-being). FACT-TH18 has 18-items which asks the patient to rate degree of thrombocytopenia. All items of the FACT-TH18 use a 5 point scale ranging from 0 to 4 with a 0 rating being "not at all" and a 4 rating being "very much".
Time Frame
Baseline and by 6 months (all patients) and 24 months (responders only)
Title
Frequency and severity of AEs and serious AEs (SAEs)
Description
Safety will be assessed by frequency and severity of AEs, serious AEs (SAEs) based on the CTCAE version 4. 03, and AEs leading to discontinuation, and evaluating changes in laboratory values within 6 months and within 24 months (responders only)
Time Frame
by 6 months (all patients) and 24 months (responders only)
Title
Pharmacokinetic parameter- Cmax of eltrombopag when combined with cyclosporine
Description
Cmax=Observed maximum plasma concentration following administration (mass/volume). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0
Title
Pharmacokinetic parameter-AUClast of eltrombopag when combined with cyclosporine
Description
AUClast=Area under the curve calculated to the last quantifiable concentration point (Tlast). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0
Title
Pharmacokinetic parameter- AUCtau of eltrombopag when combined with cyclosporine
Description
AUCtau=Area under the curve calculated to the end of the dosing interval ( tau). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0
Title
Pharmacokinetic parameter- Ctrough of eltrombopag when combined with cyclosporine
Description
Ctrough=Pre-dose plasma concentration (mass/volume). Observed maximum plasma concentration following administration (mass/volume). The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0
Title
Pharmacokinetic parameter- Tmax of eltrombopag when combined with cyclosporine
Description
Tmax=The time to reach peak or maximum concentration. The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0
Title
Pharmacokinetic parameter- CLss/F of eltrombopag when combined with cyclosporine
Description
CLss/F=Apparent systemic (or total body) clearance at steady state from plasma. The plasma samples from all patients will be assayed for eltrombopag concentrations using a validated liquid chromatography - tandem mass spectrometry assay (LC-MS/MS). The lower limit of quantitation (LLOQ) will be 100 ng/mL. Concentration values below the LLOQ will be reported as zero, and missing samples will be labeled accordingly
Time Frame
Week 2:hour 0,2,4,6,8 Week 4,8,12,26: hour 0

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Patient has signed the Informed Consent (ICF) prior to any screening procedures being performed. Patient is male/female ≥18 years old at the time of informed consent and able to swallow a tablet. Patient has SAA characterized by: Bone marrow cellularity <30% (excluding lymphocytes) and At least two of the following (peripheral blood): Absolute neutrophil count <500/µL Platelet count <20,000/µL Absolute reticulocyte count <60,000/µL Normal ECG defined as the following as determined via the mean of a triplicate ECG Resting heart rate: 50-90 bpm QTcF at screening <450 msec (for male patients), ≤460 msec (for female patients) Exclusion Criteria: Diagnosis of Fanconi anemia. Evidence of a clonal hematologic bone marrow disorder on cytogenetics by central review Prior immunosuppressive therapy with cyclosporine, alemtuzumab, rabbit or horse ATG and thrombopoietin receptor (TPO-R) agonists. a. Hypersensitivity to eltrombopag or cyclosporine or their components. b. Contraindications to cyclospsorine. AST or ALT >3 x ULN. Serum creatinine, total bilirubin, or alkaline phosphatase >1.5 x ULN. Patient with liver cirrhosis. a. Infection not adequately controlled with appropriate therapy. b. Patients who are human immune deficiency virus (HIV), hepatitis C virus or hepatitis B surface antigen (HBsAg) positive. HCV-RNA negative patients are allowed to be enrolled. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious, or metabolic disease of such severity that it would preclude the patient's ability to consent, be compliant with study procedures, tolerate protocol therapy, or that death within 30 days is likely. Patients with cancer who are not considered cure, are on active chemotherapeutic treatment or who take drugs with hematological effects. Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment. Pregnancy statements and contraception requirements: Pregnancy or nursing (lactating) women Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant (or female partners of male patients), unless they are using highly effective methods of contraception during dosing and for 3 months after stopping medication. Not able to understand the investigation nature of the study or to give informed consent. Clinically significant ECG abnormality including cardiac arrhythmias (e. g. ventricular tachycardia) complete left bundle branch block, high grade atrioventricular block, or inability to determine the QTcF interval on the ECG. Presence of cardiac disease, or family history of idiopathic sudden death or congenital long QT syndrome. Risk factors for Torsades de Pointe including uncorrected hypokalemia or hypomagnesemia, or use of concomitant medication(s) with a known risk to prolong the QT interval that cannot be discontinued or replaced by safe alternative medication per www.qtdrugs.org. ECOG performance status of ≥2. Patients under the age of 40 must be referred for consideration of allogeneic bone marrow transplantation (HSCT) if (human leukocyte antigen) HLA matching has been done and a suitable matched sibling donor is available and the patient is willing to undergo transplantation (i.e. patients who do not have a HLA match or are not medically fit, not willing or unable to undergo transplantation will be considered for enrollment).
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Ribeirao Preto
State/Province
SP
ZIP/Postal Code
14048-900
Country
Brazil
Facility Name
Novartis Investigative Site
City
São Paulo
State/Province
SP
ZIP/Postal Code
01323-900
Country
Brazil
Facility Name
Novartis Investigative Site
City
Hong Kong
Country
Hong Kong
Facility Name
Novartis Investigative Site
City
Hyderabad
State/Province
Telangana
ZIP/Postal Code
500082
Country
India
Facility Name
Novartis Investigative Site
City
Vellore
Country
India
Facility Name
Novartis Investigative Site
City
Bologna
State/Province
BO
ZIP/Postal Code
40138
Country
Italy
Facility Name
Novartis Investigative Site
City
Brescia
State/Province
BR
ZIP/Postal Code
25123
Country
Italy
Facility Name
Novartis Investigative Site
City
Milano
State/Province
MI
ZIP/Postal Code
20122
Country
Italy
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Novartis Investigative Site
City
Mexico D F
State/Province
Ciudad De Mexico
ZIP/Postal Code
06726
Country
Mexico
Facility Name
Novartis Investigative Site
City
Monterrey
State/Province
Nuevo Leon
ZIP/Postal Code
64460
Country
Mexico
Facility Name
Novartis Investigative Site
City
Puebla
ZIP/Postal Code
72000
Country
Mexico
Facility Name
Novartis Investigative Site
City
San Sebastian
State/Province
Pais Vasco
ZIP/Postal Code
20080
Country
Spain
Facility Name
Novartis Investigative Site
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10330
Country
Thailand
Facility Name
Novartis Investigative Site
City
Bangkok
ZIP/Postal Code
10700
Country
Thailand
Facility Name
Novartis Investigative Site
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Facility Name
Novartis Investigative Site
City
Istanbul
ZIP/Postal Code
34890
Country
Turkey
Facility Name
Novartis Investigative Site
City
Samsun
ZIP/Postal Code
55139
Country
Turkey

12. IPD Sharing Statement

Plan to Share IPD
No

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Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia

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