A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A

Trial ID or NCT#



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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).

Official Title

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

Eligibility Criteria

Ages Eligible for Study: 1 Year to 12 Years
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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: - Hemoglobin - Absolute neutrophils - Platelets - 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 the trial.
Exclusion Criteria:
  1. - 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 criteria. - 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 infection. - 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).


Agnieszka Czechowicz, MD, PhD

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Elisabeth Merkel
(650) 497-6746