ong-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I).Phase I/II clinical study to evaluate the safety and efficacy of the infusion of autologous hematopoietic stem cells transduced with a lentiviral vector encoding the ITGB2 gene
Phase 1
- Conditions
- eukocyte Adhesion Deficiency-I (LAD-I)MedDRA version: 23.0Level: PTClassification code: 10083936Term: Leukocyte adhesion deficiency type I Class: 100000004850Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- CTIS2022-501086-41-00
- Lead Sponsor
- Rocket Pharmaceuticals Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 9
Inclusion Criteria
Patient was enrolled in the Phase I/II Study RP-L201-0318., Patient 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., Patient is willing and able to adhere to the study visit schedule and other protocol requirements., Patient 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.
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