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


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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).

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

Stanford Investigator(s)


Inclusion Criteria:

   - 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:

   - 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).


biological: RP-L102


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Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Elisabeth Merkel
(650) 497-6746