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

NCT04774536 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 9

Last updated 2026-04-16

No results posted yet for this study

Summary

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).

Conditions

Interventions

DRUG

CRISPR_SCD001

CRISPR\_SCD001 is administered by IV infusion following myeloablative conditioning with busulfan.

Sponsors & Collaborators

Principal Investigators

  • Mark Walters, MD · UCSF Benioff Children's Hospital Oakland

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
35 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-08-22
Primary Completion
2030-03-01
Completion
2030-03-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04774536 on ClinicalTrials.gov