ong-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
Phase 1
Recruiting
- Conditions
- Fanconi anemia subtype A (FA-A)MedDRA version: 20.0Level: LLTClassification code: 10055206Term: Fanconi's anemia Class: 10010331Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-501082-52-00
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 14
Inclusion Criteria
Subject was enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118)., Subject received an autologous infusion of CD34+ enriched cells transduced ex vivo with LV vector carrying the FANCA gene, PGK-FANCA-WPRE (RP-L102), in the parent study., Subject is willing and able to adhere to the study visit schedule and other protocol requirements., Subject has provided written informed consent and, as applicable, assent to participate in the current study in accordance with current regulatory requirements.
Exclusion Criteria
There are no exclusion criteria in this study.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method