Active — Not RecruitingNot Applicable

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

United States, Spain, United Kingdom9 participantsStarted 2022-03-09

Plain-language 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

Who can participate

Age range3 Months

SexALL

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Inclusion criteria

✓. Enrolled in the Phase I/II Study RP-L201-0318.
✓. 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.
✓. Able to adhere to the study visit schedule and other protocol requirements.
✓. Provided written informed consent and, as applicable, assent to participate in the current study.

What they're measuring

Hematopoietic stem cell transplant (HSCT) free survival

Timeframe: 15 years

Trial details

NCT IDNCT06282432

SponsorRocket Pharmaceuticals Inc.

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2036-10-04

View on ClinicalTrials.gov More Leukocyte Adhesion Deficiency trials