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NCT04774536 · ClinicalTrials.gov registry record · Phase 1

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

A Phase 1 study of Sickle Cell Disease, sponsored by Mark Walters, MD.

Compiled by PlainTrial · August 8, 2026

Recruiting
Registry status: Phase 1
Development phase: 9
Enrollment target: 2
Study locations

NCT04774536: Recruiting Phase 1 study of Sickle Cell Disease, sponsored by Mark Walters, MD.

NCT04774536 is a Phase 1 study of Sickle Cell Disease that is actively recruiting participants, run by Mark Walters, MD. The registered enrollment target is 9 participants, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower). The trial reports 2 study locations across 1 state. According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT04774536, a Phase 1 study of Sickle Cell Disease, is actively recruiting participants, sponsored by Mark Walters, MD.

RECRUITING
Registry status: Phase 1
Development phase: 9 participants
Enrollment target: 2
Study locations

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).

Primary Outcome

The adverse event rate will be summarized using descriptive statistics, together with 95% confidence intervals where appropriate. No formal statistical hypothesis testing will be performed. Adverse events defined: failure of engraftment, malignant clonal expansion related to genomic editing or death.

Conditions Studied

Sickle Cell Disease

Interventions

DRUG CRISPR_SCD001

Study Locations (2)

California

University of California, Los Angeles - Los Angeles
UCSF Benioff Children's Hospital - Oakland

Trial Details

Field	Value
Enrollment Target	9 participants
Start Date	2024-09-18
Est. Completion	2029-03-01
Phase	Phase 1

Mark Walters, MD

1 total trials

What NCT04774536 shows while recruiting

NCT04774536 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 9 participants, a relatively small participant target, below the 293-participant average among 213 other Sickle Cell Disease trials with a reported enrollment target (97% lower).

The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention - of which CRISPR_SCD001 is the first listed.

NCT04774536 reports a single indexed study location in California.

Frequently Asked Questions

What is clinical trial NCT04774536 about?

NCT04774536 is a clinical study titled "Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients with Severe Sickle Cell Disease". 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 e...

What is the current status of trial NCT04774536?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 9 participants. The study started on 2024-09-18. Estimated completion is 2029-03-01.

What conditions does trial NCT04774536 study?

This clinical trial studies the following conditions: Sickle Cell Disease.

What interventions are being tested in trial NCT04774536?

The interventions under investigation include: CRISPR_SCD001 (DRUG).

Who is sponsoring clinical trial NCT04774536?

This trial is sponsored by Mark Walters, MD, which has 1 total clinical trials registered on ClinicalTrials.gov.

Where is trial NCT04774536 being conducted?

This trial has 2 study locations across California. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Learn More About Clinical Trials

Similar trials for Sickle Cell Disease

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT04774536's enrollment target sits among peer trials

9 199th of 213 higher than 14 of 213 other Sickle Cell Disease trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Sickle Cell Disease trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT04774536, the US trial registry maintained by the National Library of Medicine. NCT04774536 (small enrollment · single site footprint · recruiting) retrieved and formatted by PlainTrial, see methodology.