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

Status unconfirmed · Phase 1

Conditions studied: Fanconi Anemia Complementation Group A

In brief

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).

Key facts

Study ID

NCT03814408

Run by

Rocket Pharmaceuticals Inc.

People needed

2

Starts

2019-01-11

Expected to finish

2022-03-01

Last updated by the study team

2020-11-24

Who can join

Age: 1 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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).

Where it is running

• Stanford University Institute for Stem Cell Biology and Regenerative Medicine Lucille Packard Children's Hospital, Stanford University — Stanford, California, United States

Full record on ClinicalTrials.gov

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