A Phase II Clinical Trial to Evaluate the Efficacy of the Infusion of Autologous CD34+ Cells Transduced with a Lentiviral Vector Carrying the FANCA Gene (Orphan Drug) in Patients with Fanconi Anemia Subtype A

Phase 1

Conditions: MedDRA version: 20.0Level: LLTClassification code 10055206Term: Fanconi's anemiaSystem Organ Class: 100000004850
Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Fanconi anemia (subtype A)

Registration Number: EUCTR2018-002502-31-ES

Lead Sponsor: Rocket Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 5

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

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