Actively Recruiting

Phase 1
Phase 2
Age: 12Years - 35Years
All Genders
NCT04774536

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

Led by Mark Walters, MD · Updated on 2026-04-16

9

Participants Needed

2

Research Sites

236 weeks

Total Duration

On this page

Sponsors

M

Mark Walters, MD

Lead Sponsor

U

University of California, Los Angeles

Collaborating Sponsor

AI-Summary

What this Trial Is About

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

Official Title

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

Who Can Participate

Age: 12Years - 35Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Male or female aged 12.00 to 34.99 years at time of consent
  • History of two or more episodes of acute chest syndrome in the past 2 years despite supportive care
  • History of at least 4 severe vaso-occlusive pain events in the past 2 years despite supportive care
  • Two or more episodes of splenic sequestration in the past 2 years
  • Recurrent priapism lasting at least 4 hours, occurring at least twice in the last 12 months or three times in 24 months
  • Any episode of hepatic sequestration in the past 2 years
  • Leg ulcer resistant to treatment within 2 years prior to enrollment
  • Karnofsky performance score 60 or higher
  • Left ventricular ejection fraction over 40% or LV shortening fraction over 26%
  • Baseline oxygen saturation of 85% or higher and lung diffusion capacity over 40%
  • Serum creatinine no higher than 1.5 times upper limit of normal and creatinine clearance at least 70 mL/min/1.73 m2
  • Serum conjugated bilirubin less than twice upper limit of normal
  • ALT and AST less than 5 times upper limit of normal
  • Prothrombin time or partial thromboplastin time less than 1.5 times upper limit of normal unless on prophylactic anticoagulant
  • Written informed consent or assent and ability to comply with study requirements
Not Eligible

You will not qualify if you...

  • Uncontrolled bacterial, viral, or fungal infection within 6 weeks before enrollment
  • Evidence of HIV infection or active hepatitis B or C
  • Prior hematopoietic cell transplant or solid organ transplant
  • Participation in another clinical trial with investigational or off-label drug/device within 3 months prior to enrollment
  • Female participants who are pregnant or breastfeeding
  • Females of childbearing potential not agreeing to use two effective contraceptive methods or abstinence through 12 months post-infusion
  • Males not agreeing to use effective barrier contraception or abstinence through 12 months post-infusion
  • History of stroke or receiving red blood cell transfusions to prevent stroke
  • Presence of a suitable HLA-identical sibling donor for bone marrow donation
  • Any condition or abnormality compromising safety or data quality as judged by investigator
  • Non-homozygous sickle hemoglobin genotype
  • Diagnosis of myelodysplastic syndrome or pathogenic clonal gene variants associated with hematologic malignancies
  • Identified pathogenic mutation or variant of unknown significance judged pathogenic for myeloid malignancy

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 2 locations

1

University of California, Los Angeles

Los Angeles, California, United States, 90095

Actively Recruiting

2

UCSF Benioff Children's Hospital

Oakland, California, United States, 94609

Actively Recruiting

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Research Team

M

Mark Walters, MD

CONTACT

C

Christina Chun, MPH

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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