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

First listed
Mar 1, 2021
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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