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

Trial ID or NCT#

NCT03814408

Status

not recruiting iconNOT RECRUITING

Purpose

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:
  2. * 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:
  3. * 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:
  2. * 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:
  3. * 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).
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Investigator(s)

Agnieszka Czechowicz, MD, PhD

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Contact

Elisabeth Merkel
(650) 497-6746

View on ClinicalTrials.gov