Status:
UNKNOWN
A Clinical Trial to Evaluate the Safety of RP-L102 in Pediatric Subjects With Fanconi Anemia Subtype A
Lead Sponsor:
Rocket Pharmaceuticals Inc.
Conditions:
Fanconi Anemia Complementation Group A
Eligibility:
All Genders
1-12 years
Phase:
PHASE1
Brief Summary
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 lent...
Detailed Description
This is a pediatric open-label Phase 1 clinical trial and will include a safety evaluation and preliminary assessment of the efficacy of hematopoietic gene therapy consisting of autologous CD34+ enric...
Eligibility Criteria
Inclusion
- 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
- 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).
Key Trial Info
Start Date :
January 11 2019
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
March 1 2022
Estimated Enrollment :
2 Patients enrolled
Trial Details
Trial ID
NCT03814408
Start Date
January 11 2019
End Date
March 1 2022
Last Update
November 24 2020
Active Locations (1)
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1
Stanford University Institute for Stem Cell Biology and Regenerative Medicine Lucille Packard Children's Hospital, Stanford University
Stanford, California, United States, 94304