NCT04069533Phase 2Unknown

Lentiviral-mediated Gene Therapy for Pediatric Patients With Fanconi Anemia Subtype A

Study sponsor: Rocket Pharmaceuticals Inc. · RCKT

Compare the available registry edits below. Primary completion is the date data collection ends for the main study measures; it does not tell you when results will be published.

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Primary completion2025-02Estimated

Enrollment · participants7Actual

Source updated2024-05-22Last update listed in the registry record

Version history

Changes to this trial

Last 12 months

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No recorded changes are available for this view and time period. Our history may be incomplete; this does not establish that the trial stayed unchanged.

Study details

Conditions studied

Fanconi Anemia Complementation Group A

Treatments or interventions studied

RP-L102Biological

Primary outcomes · main study measures

measure: Phenotypic correction of bone marrow colony forming units after infusion of RP-L102; description: During months 12-36 post-infusion, the survival of bone marrow colony forming units to 10nM mitomycin C (MMC) increases to over or equal to 10% with respect to values determined at baseline (pretreatment evaluation).; timeFrame: 3 years

History coverage

Research sample: selected versions from May 2023 onward plus a preceding baseline where available. Only selected fields were retained; endpoint measures were truncated to 300 characters. This is not complete trial or sponsor history. Daily snapshots begin when collection starts.

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