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A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
Trial ID: NCT03814408
The objective of this study is to assess the therapeutic safety and preliminary efficacy of a hematopoietic cell-based gene therapy consisting of autologous CD34+ enriched cells transduced with a lentiviral vector carrying the FANCA gene in subjects with Fanconi anemia subtype A (FA-A).
A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the FANCA Gene in Pediatric Subjects With Fanconi Anemia Subtype A
- Fanconi anemia, as diagnosed by chromosomal fragility assay of cultured T-lymphocytes
in the presence of DEB or a similar DNA-crosslinking agent.
- Subjects of Fanconi Anemia complementation group A.
- Minimum age: 1 year and a minimum of 8 kg.
- Maximum age: 12 years.
- At least one of the following hematologic parameters below lower limits of normal:
- Absolute neutrophils
- At least 30 CD34+ cells/μL are determined in one BM aspiration within 3 months prior
to initiation of CD34+ cell collection.
- 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
- Provide informed consent in accordance with current legislation.
- 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
- Subjects with an available and medically eligible human leukocyte antigen
(HLA)-identical sibling donor.
- 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 subject commences the stem
cell mobilization/collection procedures of the clinical trial.
- Subjects 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).
- Lansky performance status ≤60%.
- Any concomitant disease or condition that, in the opinion of the Principal
Investigator, renders the subject unfit to participate in the study.
- Pre-existing sensory or motor impairment ≥grade 2 according to the NCI CTCAE v5.0
- Pregnant or breastfeeding women.
- Hepatic dysfunction as defined by either:
- Bilirubin >3.0 × upper limit of normal (ULN) or
- Alanine aminotransferase (ALT) > 5.0 × ULN or
- Aspartate aminotransferase (AST) > 5.0 × ULN
- Renal dysfunction requiring either hemodialysis or peritoneal dialysis.
- Pulmonary dysfunction as defined by either:
- Need for supplemental oxygen during the prior 2 weeks in absence of acute
- Oxygen saturation by pulse oximetry <90%.
- Evidence of active metastatic or locoregionally advanced malignancy for which survival
is anticipated to be less than 3 years.
- Subject is receiving androgens (i.e. danazol, oxymethalone).