Fanconi anemia (subtype A) MedDRA version: 20.0 Level: LLT Classification code 10055206 Term: Fanconi's anemia System Organ Class: 100000004850
Conditions
Interventions
Product Name: RP-L102
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: CD34+CELLS
Other descriptive name: CD34+CELLS
Concentration unit: IU/kg international unit(s)/kilogram
Concentr
Sponsors
Rocket Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent 2. Patients of the complementation group FA-A 3. Minimum age: 1 year and a minimum weight of 8 kg. 4. Maximum age: 12 years 5. At least one of the following hematologic parameters below lower limits of normal of • Hemoglobin • Absolute neutrophils • Platelets 6. At least 30 CD34+ cells/µL are determined in one BM aspiration within 3 months prior to CD34+ cell collection 7. If the number of C34+ cells/ µL in BM is in the range of 10-29, PB parameters should meet two of the three following criteria: • Hemoglobin: =11g/dL • Neutrophils: =900 cells/µL • Platelets: =60,000 cells/µL 8. Provide informed consent in accordance with current legislation 9. Women of childbearing age must have a negative urine pregnancy test at the baseline visit, and accept the use of an effective contraception method during participation in the trial Are the trial subjects under 18? yes Number of subjects for this age range: 5 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Patients with an HLA-identical sibling donor 2. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities predictive of these conditions in BM aspirate analysis. This assessment should be made by valid studies conducted within the 3 months before the patient enters the clinical trial 3. Patients with somatic mosaicism associated with stable or improved counts in all PB cell lineages 4. Lansky performance index = 60% 5. Any concomitant disease or condition that, in the opinion of the Principal Investigator, deems the patient unfit to participate in the trial 6. Pre-existing sensory or motor impairment > = grade 2 according to the criteria of the National Cancer Institute (NCI) 7. Pregnant or breastfeeding women 8. Hepatic dysfunction as defined by either: • Bilirubin > 1.5 x the upper limit of normal (ULN) • Alanine aminotransferase (ALT ) > 2.5 x ULN • Aspartate aminotransferase (ALT) > 2.5 x ULN. 9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis 10. Pulmonary dysfunction as defined by either: • Need for supplemental oxygen during the prior 2 weeks (in absence of acute infection) • Oxygen saturation (by pulse oximetry) <90%. 11. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The main objective of this trial is to assess the therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene in patients with Fanconi anemia subtype A (complementation group A, FA-A).;Secondary Objective: Not applicable;Primary end point(s): • Level of phenotypic correction of hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Level of engraftment of gene-corrected hematopoietic cells after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Clinical response (prevention or rescue of bone marrow failure (BMF)) after the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A • Ongoing evaluation of the short- and long-term safety of the infusion of autologous CD34+ cells transduced with the therapeutic lentiviral vector in patients with Fanconi anemia subtype A;Timepoint(s) of evaluation of this end point: Up to 3 years after infusion | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable | — |
Countries
Spain
Contacts
Public ContactTeresa Bricio
Alpha Bioresearch, S.L.
Outcome results
None listed