Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

Part of paid clinical trials in Los Angeles, California.

Sponsor
Mark Walters, MD
Study ID
NCT04774536
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
12 Years - 35 Years
Healthy Volunteers
Not accepted

Interventions

  • CRISPR_SCD001 — DRUG
    CRISPR\_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.

Study Details

This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will evaluate the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

Key Dates

Start date
Aug 22, 2025
Status verified
Apr 2026
Primary completion
Mar 1, 2030
Completion
Mar 1, 2030

Study Design

Enrollment
9 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: CRISPR_SCD001 Drug Product
    CRISPR\_SCD001 Drug Product (autologous CD34+ cell-enriched population that contains cells modified by the CRISPR-Cas9 ribonucleoprotein) dose will be ≥3.0×106 CD34+ cells/kg recipient weight for each subject and the upper limit cell dose is 20 ×106 CD34+ cells/kg.

Primary Outcome Measure

Incidence of adverse events and grade 3 or higher serious adverse events, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) [ Time Frame: 24 months post-transplant ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
University of California, Los AngelesLos AngelesCalifornia90095
Augustine Fernandes, PhD
[email protected]
Vladimir Kustanovich, PhD
[email protected]
Gary J Schiller, MD (PRINCIPAL_INVESTIGATOR)
UCSF Benioff Children's HospitalOaklandCalifornia94609
Marci Moriarty, RN BSN
[email protected]
(510) 428-3885
Cyrus Bascon
[email protected]
(510) 428-3885
Mark Walters, MD (PRINCIPAL_INVESTIGATOR)

Find similar trials in Los Angeles, CA

By condition
Sickle cell disease
By research site
University of California, Los Angeles· Los Angeles, CAUCSF Benioff Children's Hospital· Oakland, CA

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