Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I)

Launched by ROCKET PHARMACEUTICALS INC. · Feb 20, 2024

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ClinConnect Summary

Following the end of participation in Study RP-L201-0318, patients will be offered enrollment into this LTFU protocol. Patients will be followed for up to 15 years following the RP-L201 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, annual visits to the study center are required during initial 3 years post- RP-L201 infusion. Visits where a bone ...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Enrolled in the Phase I/II Study RP-L201-0318.
• • 2. Received an autologous infusion of CD34+ hematopoietic stem cells modified with a lentiviral vector containing the ITGB2 gene, encoding for the human CD18 receptor in the parent Study RP-L201-0318.
• • 3. Able to adhere to the study visit schedule and other protocol requirements.
• • 4. Provided written informed consent and, as applicable, assent to participate in the current study.
• Exclusion Criteria:
• • There are no criteria for exclusion in this study.