EUCTR2018-002502-31-GB
Active, not recruiting
Phase 1
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 - FANCOLEN-II
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Fanconi anemia (subtype A)
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Enrollment
- 5
- Status
- Active, not recruiting
- Last Updated
- 5 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •1\. FA 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: 17 years
- •5\. At least 30 CD34\+ cells/µL are determined in one BM aspiration within 3 months prior to CD34\+ cell collection OR (see subsequent criterion)
- •6\. If the number of CD34\+ 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
- •7\. Provide informed consent in accordance with current legislation
Exclusion Criteria
- •1\. Patients with an available and medically eligible HLA\-identical sibling donor
- •2\. Evidence of myelodysplastic syndrome or leukaemia, or cytogenetic abnormalities other than those reported as variant(s) of normal 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 (If T\-lymphocyte chromosomal fragility analysis indicates potential mosaicism, a medically significant decrease in at least one blood lineage over time must be documented to enable eligibility)
- •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 \> 3\.0× the upper limit of normal (ULN)
- •Alanine aminotransferase (ALT) \>5\.0× ULN
Outcomes
Primary Outcomes
Not specified
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