Rocket Pharmaceuticals Inc.
Long-Term Follow-up: Phase I/II clinical study to evaluate the safety and efficacy of the infusion of RP-L102
Following the end of participation in Study(RP-L102-0418, RP-L102-0319, RP-L102-0118), patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L102 infusion in the parent study, until the patient dies, withdraws consent, or is lost to follow-up (whichever occurs first). For all follow-up visits, remote evaluation facilitated by local health care providers (with blood sample shipment to relevant laboratory facilities) is permitted; however, visits to the study center are required for up to 2 years post- RP-L102 infusion. Study…
Inclusion Criteria: 1. Enrolled in one of the RP-L102 parent studies (RP-L102-0418, RP-L102-0319, RP-L102-0118). 2. 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 studies. 3. Able to adhere to the study visit schedule and other protocol requirements. 4. 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 criteria for exclusion in this study.
CD34+ enriched cells from subjects with Fanconi anemia subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the FANCA gene, PGK-FANCA-WPRE
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