Leukocyte Adhesion Deficiency Clinical Trial
Official title:
Long-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
Verified date | February 2024 |
Source | Rocket Pharmaceuticals Inc. |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational |
This Long-Term Follow-Up (LTFU) for Gene Therapy of Leukocyte Adhesion Deficiency-I (LAD-I) is a continuation of a Phase 1/2 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
Status | Enrolling by invitation |
Enrollment | 9 |
Est. completion date | March 2037 |
Est. primary completion date | March 2037 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 3 Months and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
Spain | Hospital Infantil Universitario Niño Jesús | Madrid | |
United Kingdom | University College London Great Ormond Street Institute of Child Health (GOSH) | London | |
United States | University of California, Los Angeles (UCLA) | Los Angeles | California |
Lead Sponsor | Collaborator |
---|---|
Rocket Pharmaceuticals Inc. |
United States, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Hematopoietic stem cell transplant (HSCT) free survival | Survival without allogeneic-HSCT. | 15 years | |
Secondary | Incidence of hospitalizations | Incidence of infection-related hospitalizations. | 15 years | |
Secondary | Incidence of significant infections | Incidence of infections requiring hospitalization or intravenous antimicrobials. | 15 years | |
Secondary | Resolution of LAD-I-related skin rash | Partial or complete resolution of LAD-I skin rash evident by photographical images. | 15 years | |
Secondary | Resolution of LAD-I-related periodontal abnormalities | Partial or complete resolution of LAD-I periodontal abnormalities evident by photographical images. | 15 years | |
Secondary | Event free survival | Survival in the absence of graft failure and graft versus host disease. | 15 years | |
Secondary | Overall Survival | Survival in the absence of death from any cause | 15 years | |
Secondary | Long-term genetic correction in peripheral blood mononuclear cells (PBMCs) | Persistence of transgene in PB cells as demonstrated by vector copy number (VCN) of at least 0.1 in PBMCs. | 15 years | |
Secondary | Long-term genetic correction in PB CD15+ granulocytes | Persistence of transgene in PB cells as demonstrated by VCN of at least 0.1 in PB CD15+ granulocytes. | 15 years | |
Secondary | Long-term CD18 neutrophil expression by flow cytometry | Persistence of CD18 neutrophil expression defined by PB neutrophil CD18 expression to at least 10% of normal. | 15 Years | |
Secondary | Long-term CD11 neutrophil expression by flow cytometry | Persistence of CD11 a/b neutrophil co-expression | 15 Years | |
Secondary | Improvement or resolution of LAD-I related neutrophilia | Improvement of LAD-I related neutrophilia based off neutrophils within age-appropriate normal ranges. | 15 Years | |
Secondary | Improvement or resolution of LAD-I-related leukocytosis. | Improvement of LAD-I related leukocytosis based off leukocytes within age-appropriate normal ranges. | 15 Years | |
Secondary | Incidence of Investigational Product (IP) related serious adverse events (SAEs) | Incidence of SAEs related to the IP measured by CTCAE (Common Terminology Criteria for Adverse Events) for V5.0 grading scale. | 15 Years | |
Secondary | Incidence of hematologic malignancy | Incidence of hematologic malignancy related to prior gene therapy or gene-therapy associated medications. | 15 Years |
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