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Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

Primary Purpose

Fanconi Anemia

Status
Completed
Phase
Phase 2
Locations
Spain
Study Type
Interventional
Intervention
filgrastim
plerixafor
Sponsored by
Hospital Universitari Vall d'Hebron Research Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Fanconi Anemia

Eligibility Criteria

2 Years - 64 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Male or female > 1 year
  • diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C
  • At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3
  • Lansky index> 60%.
  • Left ventricular ejection fraction >50%.
  • To grant informed consent in agreement with current law norms
  • Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last

Exclusion Criteria:

  • Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted
  • Patients with active infection process or any other underlaying severe medical process
  • Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria
  • Haematopoietic transplant
  • Any disease or concomitant process that is not compatible with the study as per investigator opinion
  • Patients not elegible because of an psico-social evaluation
  • Patients that received transfusional support during the last 3 months.
  • Pregnant or breastfeeding women

Sites / Locations

  • Hospital Universitari Vall d'Hebron
  • Hospital Infantil Universitario Niño Jesus

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

plerixafor and filgrastim treatment

Arm Description

to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim

Outcomes

Primary Outcome Measures

Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.

Secondary Outcome Measures

Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor
mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter
Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years
the CD34+ cell collection protocol will be determined by the percentage of patients who reach at least one million CD34 + cells per kilogram of body weight projected to 5 years after the mobilization process
Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%
the of CD34+ cell selection process will be determined by the proportion of immunoselected samples where the recovery of CD34 + cells is at least 50%, and where the final percentage of CD34+ cells is at least 50%
Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years
will be determined by the percentage of patients who reach at least one million CD34 + cells per kilo of weight projected to 5 years after the immunomagnetic selection process of all the collected cells

Full Information

First Posted
September 6, 2016
Last Updated
May 25, 2020
Sponsor
Hospital Universitari Vall d'Hebron Research Institute
Collaborators
CIEMAT, CIBERER
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1. Study Identification

Unique Protocol Identification Number
NCT02931071
Brief Title
Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1
Official Title
Clinical Phase II Trial to Evaluate Efficacy and Safety of CD34+ Cells Mobilization and Collection After Treatment With Plerixafor and Filgrastim in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene and Reinfusion in the Patient
Study Type
Interventional

2. Study Status

Record Verification Date
May 2020
Overall Recruitment Status
Completed
Study Start Date
September 2013 (undefined)
Primary Completion Date
October 2018 (Actual)
Study Completion Date
October 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospital Universitari Vall d'Hebron Research Institute
Collaborators
CIEMAT, CIBERER

4. Oversight

5. Study Description

Brief Summary
Fanconi anemia (FA) is a congenital disease characterized by bone marrow failure and increased incidence of malignant tumors. The Project pursue the optimization of the collection of hematopoietic progenitor cells for later use in another clinical trial entitled "Clinical Trial Phase I/II to evaluate the safety and efficacy of the infusion of autologous CD34+ cells mobilized with mozobil and filgrastim, and transduced with a lentiviral vector carrying the FANCA gene (Orphan Drug) for patients with Fanconi Anemia Subtype A ". The objectives of this study are, therefore, to assess the safety and efficacy of CD34+ cells mobilization with mozobil and filgrastim, which is postulated the most efficient for the collection of CD34+ cells from FA patients.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Fanconi 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
13 (Actual)

8. Arms, Groups, and Interventions

Arm Title
plerixafor and filgrastim treatment
Arm Type
Experimental
Arm Description
to assess the safety and efficacy of CD34+ cells mobilization with plerixafor and filgrastim
Intervention Type
Drug
Intervention Name(s)
filgrastim
Intervention Description
G-CSF (12 μg/Kg/12 h) 8 days.
Intervention Type
Drug
Intervention Name(s)
plerixafor
Intervention Description
Plerixafor 0,24 mg/kg/day after the fourth day of G-CSF, and until 5 cells CD34+/μL, max 4 doses of plerixafor
Primary Outcome Measure Information:
Title
Toxicity of the mobilization procedure according to National Cancer Institute (CTC NCI, versión 3.0)
Description
At a year (± 30 days) after the last apheresis, a complete physical examination, blood cell count, basic biochemistry and bone marrow aspirate will be done to the patient in order to control their general health status.
Time Frame
after 12 months
Secondary Outcome Measure Information:
Title
Percentage of patients that reach >5 CD34+ cells/mcl after treatment with filgrastim and plerixafor
Description
mobilization protocol will be determined by the percentage of patients who achieve peripheral blood counts exceeding 5 CD34+ cells /microliter
Time Frame
after 8 days
Title
Percentage of patients that reach a total CD34+ yield >4x10E6/kg, using the estimated weight of the patient in 5 years
Description
the CD34+ cell collection protocol will be determined by the percentage of patients who reach at least one million CD34 + cells per kilogram of body weight projected to 5 years after the mobilization process
Time Frame
after 8 days
Title
Percentage of samples in which the recovery of CD34+ cells after the immunomagnetic selection procedure is >50%
Description
the of CD34+ cell selection process will be determined by the proportion of immunoselected samples where the recovery of CD34 + cells is at least 50%, and where the final percentage of CD34+ cells is at least 50%
Time Frame
after 8 days
Title
Percentage of patients in which the CD34+ cells after the immunomagnetic selection is ³ 4x10E6/kg, using the projected weight at 5 years
Description
will be determined by the percentage of patients who reach at least one million CD34 + cells per kilo of weight projected to 5 years after the immunomagnetic selection process of all the collected cells
Time Frame
after 8 days

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
64 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Male or female > 1 year diagnosed of Fanconi's anemia confirmed by instability chromosomal test with diepoxy-butane or mitomycin C At least one of the following parameters must be higher than these values: Hemoglobin:8,0 g/dL; neutrophils: 750/mm3; platelets: 30.000/mm3 Lansky index> 60%. Left ventricular ejection fraction >50%. To grant informed consent in agreement with current law norms Women in childbearing age must obtain a negative result in the pregnancy test in serum or urine in the visit of selection and accept the use of suitable contraceptive methods since at least 14 days prior to the first dose of mobilizing treatment until the 14 days following the last Exclusion Criteria: Evidence of myelodysplastic syndromes or leukemia, or cytogenetic abnormalities predicted of these syndromes in bone marrow aspiration. Cytogenetic analyses performed 2 months before starting study are accepted Patients with active infection process or any other underlaying severe medical process Severe Functional alteration of organs (hepatic, renal, respiratory)(?3), according to National Cancer Institute (NCI CTCAE v3) criteria Haematopoietic transplant Any disease or concomitant process that is not compatible with the study as per investigator opinion Patients not elegible because of an psico-social evaluation Patients that received transfusional support during the last 3 months. Pregnant or breastfeeding women
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Cristina Díaz de Heredia, MD, PhD
Organizational Affiliation
Hospital Vall d'Hebron
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Universitari Vall d'Hebron
City
Barcelona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Hospital Infantil Universitario Niño Jesus
City
Madrid
ZIP/Postal Code
28009
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
34485595
Citation
Sevilla J, Navarro S, Rio P, Sanchez-Dominguez R, Zubicaray J, Galvez E, Merino E, Sebastian E, Azqueta C, Casado JA, Segovia JC, Alberquilla O, Bogliolo M, Roman-Rodriguez FJ, Gimenez Y, Larcher L, Salgado R, Pujol RM, Hladun R, Castillo A, Soulier J, Querol S, Fernandez J, Schwartz J, Garcia de Andoin N, Lopez R, Catala A, Surralles J, Diaz-de-Heredia C, Bueren JA. Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes. Mol Ther Methods Clin Dev. 2021 Jun 12;22:66-75. doi: 10.1016/j.omtm.2021.06.001. eCollection 2021 Sep 10.
Results Reference
derived

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Clinical Phase II Trial to Evaluate CD34+ Cells Mobilization and Collection in Patients With Fanconi Anemia for Subsequent Transduction With a Lentiviral Vector Carring FANCA Gene. FANCOSTEM-1

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