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Gene Therapy Trial Report

Summary

Gene Therapy for Fanconi Anemia, Complementation Group A


NCTID NCT04248439 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Fanconi Anemia Complementation Group A
Disease Ontology Term DOID:0111095
Compound Name RP-L102
Compound Alias Mozafancogene autotemcel
Compound Description PGK-FANCA.WPRE
Sponsor Rocket Pharmaceuticals Inc.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 5 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant FANCA
Therapy Type Gene transfer
Therapy Route Ex-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Autologous cells
Target Tissue/Cell CD34+ cells
Delivery System Viral transduction
Vector Type LV
Editor Type none
Dose 1 Dose range: 2.0E5 - 4.1E6 transduced CD34+ cells/kg

Study Record Dates


Current Stage Phase2
Submit Date 2020-01-24
Completion Date 2026-05-05
Last Update 2025-12-22

Participation Criteria


Eligible Age >=1 Year
Standard Ages Child, Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Fanconi anemia as diagnosed by chromosomal fragility assay of cultured lymphocytes in the presence of DEB or a similar DNA-crosslinking agent 2. Subject of the complementation group FA-A 3. Minimum age: 1 year and a minimum weight of 8 kg 4. At least 30 CD34+ cells/μL are determined in one bone marrow (BM) aspiration within 3 months prior to CD34+ cell collection OR 6\. Provide informed consent in accordance with current legislation 7. 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 HLA-identical sibling donor. 2. Evidence of myelodysplastic syndrome or leukemia, or cytogenetic abnormalities other than those reported as variant(s) of normal 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. 3. 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 (≥1 NCI CTCAE grade) in at least one blood lineage over time must be documented to enable eligibility, as should \<5% resistance of bone marrow colony forming cells (CFCs) to 10nM MMC; whenever possible potential mosaicism should also be evaluated by gene sequencing of MMC-resistant CFCs). 4. Lansky performance status ≤60%. 5. Any concomitant disease or condition that, in the opinion of the Principal Investigator, renders the subject unfit to participate in the study. 6. Pre-existing sensory or motor impairment ≥grade 2 according to the criteria of the NCI. 7. Pregnant or breastfeeding women. 8. Hepatic dysfunction as defined by either: * Bilirubin \>3.0 × the upper limit of normal (ULN) or * Alanine aminotransferase (ALT) \> 5.0 × ULN or * Aspartate aminotransferase (AST) \> 5.0 × ULN For subjects with bilirubin, ALT or AST above ULN, a workup to identify the etiology of liver abnormality should be conducted prior to confirmation of eligibility as stipulated in exclusion criterion 5, including evaluation of viral hepatitis, iron overload, drug injury or other causes. 9. Renal dysfunction requiring either hemodialysis or peritoneal dialysis. 10. Pulmonary dysfunction as defined by either: * Need for supplemental oxygen during the prior 2 weeks in absence of acute infection or * Oxygen saturation by pulse oximetry \<90%. 11. Evidence of active metastatic or locoregionally advanced malignancy for which survival is anticipated to be less than 3 years. 12. Subject is receiving androgens (i.e. danazol, oxymetholone). 13. Subject is receiving other investigational therapy for treatment/prevention of FA-associated bone marrow failure.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 2
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates Rocket withdrew BLA in October 2025 due to changes in corporate priorities

Resources/Links