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Clinical Trial Details — Status: Enrolling by invitation

Administrative data

NCT number NCT06282432
Other study ID # RP-L201-0121-LTFU
Secondary ID
Status Enrolling by invitation
Phase
First received
Last updated
Start date March 9, 2022
Est. completion date March 2037

Study information

Verified date February 2024
Source Rocket Pharmaceuticals Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

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


Description:

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 marrow sample is being collected are required to be performed at the study center for the duration of the study. Peripheral Blood samples and bone marrow samples will be archived and tested when clinically or scientifically indicated, as in the event of development of a second malignancy.


Recruitment information / eligibility

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.

Study Design


Related Conditions & MeSH terms


Locations

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

Sponsors (1)

Lead Sponsor Collaborator
Rocket Pharmaceuticals Inc.

Countries where clinical trial is conducted

United States,  Spain,  United Kingdom, 

Outcome

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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