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

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ClinicalTrials.gov Identifier: NCT06282432
Recruitment Status : Enrolling by invitation
First Posted : February 28, 2024
Last Update Posted : February 28, 2024
Sponsor:
Information provided by (Responsible Party):
Rocket Pharmaceuticals Inc.

Brief 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

Condition or disease
Leukocyte Adhesion Deficiency

Detailed 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.

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Study Type : Observational
Estimated Enrollment : 9 participants
Observational Model: Case-Only
Time Perspective: Prospective
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
Actual Study Start Date : March 9, 2022
Estimated Primary Completion Date : March 2037
Estimated Study Completion Date : March 2037


Group/Cohort
Subjects that received RP-L201 on the RP-L201-0318 Parent Study
Subjects that received RP-L201 on the RP-L201-0318 Parent Study and either completed the study or discontinued early.



Primary Outcome Measures :
  1. Hematopoietic stem cell transplant (HSCT) free survival [ Time Frame: 15 years ]
    Survival without allogeneic-HSCT.


Secondary Outcome Measures :
  1. Incidence of hospitalizations [ Time Frame: 15 years ]
    Incidence of infection-related hospitalizations.

  2. Incidence of significant infections [ Time Frame: 15 years ]
    Incidence of infections requiring hospitalization or intravenous antimicrobials.

  3. Resolution of LAD-I-related skin rash [ Time Frame: 15 years ]
    Partial or complete resolution of LAD-I skin rash evident by photographical images.

  4. Resolution of LAD-I-related periodontal abnormalities [ Time Frame: 15 years ]
    Partial or complete resolution of LAD-I periodontal abnormalities evident by photographical images.

  5. Event free survival [ Time Frame: 15 years ]
    Survival in the absence of graft failure and graft versus host disease.

  6. Overall Survival [ Time Frame: 15 years ]
    Survival in the absence of death from any cause

  7. Long-term genetic correction in peripheral blood mononuclear cells (PBMCs) [ Time Frame: 15 years ]
    Persistence of transgene in PB cells as demonstrated by vector copy number (VCN) of at least 0.1 in PBMCs.

  8. Long-term genetic correction in PB CD15+ granulocytes [ Time Frame: 15 years ]
    Persistence of transgene in PB cells as demonstrated by VCN of at least 0.1 in PB CD15+ granulocytes.

  9. Long-term CD18 neutrophil expression by flow cytometry [ Time Frame: 15 Years ]
    Persistence of CD18 neutrophil expression defined by PB neutrophil CD18 expression to at least 10% of normal.

  10. Long-term CD11 neutrophil expression by flow cytometry [ Time Frame: 15 Years ]
    Persistence of CD11 a/b neutrophil co-expression

  11. Improvement or resolution of LAD-I related neutrophilia [ Time Frame: 15 Years ]
    Improvement of LAD-I related neutrophilia based off neutrophils within age-appropriate normal ranges.

  12. Improvement or resolution of LAD-I-related leukocytosis. [ Time Frame: 15 Years ]
    Improvement of LAD-I related leukocytosis based off leukocytes within age-appropriate normal ranges.

  13. Incidence of Investigational Product (IP) related serious adverse events (SAEs) [ Time Frame: 15 Years ]
    Incidence of SAEs related to the IP measured by CTCAE (Common Terminology Criteria for Adverse Events) for V5.0 grading scale.

  14. Incidence of hematologic malignancy [ Time Frame: 15 Years ]
    Incidence of hematologic malignancy related to prior gene therapy or gene-therapy associated medications.


Biospecimen Retention:   Samples With DNA
Human Tissue (including blood and bone marrow)


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   3 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects that have been treated with RP-L201 on the RP-L201-0318 study.
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06282432


Locations
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United States, California
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095-1489
Spain
Hospital Infantil Universitario Niño Jesús
Madrid, Spain, 28009
United Kingdom
University College London Great Ormond Street Institute of Child Health (GOSH)
London, United Kingdom, WC1N 1EH
Sponsors and Collaborators
Rocket Pharmaceuticals Inc.
Investigators
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Principal Investigator: Donald Kohn, MD University of California, Los Angeles
Principal Investigator: Claire Booth, MBBS, PhD, MSc University College London Great Ormond Street Institute of Child Health
Principal Investigator: Julian Sevilla, MD, PhD Hospital Infantil Universitario Niño Jesús
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Responsible Party: Rocket Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT06282432    
Other Study ID Numbers: RP-L201-0121-LTFU
First Posted: February 28, 2024    Key Record Dates
Last Update Posted: February 28, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Rocket Pharmaceuticals Inc.:
LAD-I
LAD
Additional relevant MeSH terms:
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Leukocyte-Adhesion Deficiency Syndrome
Tissue Adhesions
Cicatrix
Fibrosis
Pathologic Processes
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes
Immune System Diseases