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Gene Therapy

CRISPR-Cas9 Modified Stem Cells for Sickle Cell Disease

Phase 3
Recruiting
Research Sponsored by Vertex Pharmaceuticals Incorporated
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Diagnosis of severe SCD as defined by documented SCD genotypes
History of at least two severe VOCs events per year for the previous two years prior to enrollment
Timeline
Screening 3 weeks
Treatment Varies
Follow Up within 42 days after ctx001 infusion
Awards & highlights

Study Summary

This trial is testing a new treatment for sickle cell disease using a patient's own stem cells that have been modified with CRISPR-Cas9.

Who is the study for?
This trial is for children with severe Sickle Cell Disease who have had at least two serious pain episodes a year and haven't responded well to or can't tolerate Hydroxyurea treatment. They should be suitable for their own stem cell transplant, not have had one before, and not currently have any major infections.Check my eligibility
What is being tested?
The study tests CTX001, which involves editing the patient's stem cells using CRISPR-Cas9 technology to treat severe SCD. It's an open-label trial meaning everyone knows they're getting this single-dose experimental therapy.See study design
What are the potential side effects?
Potential side effects are likely related to the CRISPR-Cas9 process and stem cell transplant procedure but aren't specified here. Common risks may include immune reactions, infection risk from the procedure, and possible off-target genetic effects.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have been diagnosed with severe sickle cell disease.
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I've had two or more severe pain crises a year for the last two years.
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I have tried hydroxyurea without success or cannot tolerate it.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~within 42 days after ctx001 infusion
This trial's timeline: 3 weeks for screening, Varies for treatment, and within 42 days after ctx001 infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Proportion of Participants who do not Have any Severe Vaso-occlusive Crises (VOCs) for at Least 12 Consecutive Months (VF12)
Secondary outcome measures
Haptoglobins
Change in Indirect Bilirubin Over Time
Change in Lactate Dehydrogenase (LDH) Over Time
+27 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: CTX001Experimental Treatment1 Intervention
CTX001 (autologous CD34+ hHSPCs modified with CRISPR-Cas9 at the erythroid lineage-specific enhancer of the BCL11A gene). Participants will receive single infusion of CTX001 through central venous catheter.

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor
241 Previous Clinical Trials
32,253 Total Patients Enrolled
CRISPR TherapeuticsIndustry Sponsor
5 Previous Clinical Trials
291 Total Patients Enrolled

Media Library

CTX001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05329649 — Phase 3
Sickle Cell Disease Research Study Groups: CTX001
Sickle Cell Disease Clinical Trial 2023: CTX001 Highlights & Side Effects. Trial Name: NCT05329649 — Phase 3
CTX001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05329649 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are new participants being recruited for this clinical research project?

"The study, which was posted on 5/2/2022 and updated on 10/31/2022, is seeking enrollees."

Answered by AI

Does this research include young people under the age of 25?

"Children aged 2 to 11 years old may be eligible for this trial, as specified by the provided eligibility criteria. Out of the 166 trials conducted for patients under 18, 128 are for those over 65."

Answered by AI

Are there any serious health risks that come with taking CTX001?

"There is available evidence from previous clinical trials that support CTX001's efficacy and safety, giving it a score of 3."

Answered by AI

How can I sign up for this clinical trial?

"Up to 12 kids that suffer from hemoglobinopathies and meet the following conditions can enroll in this study: Key, Documented SCD genotypes, History of at least two severe VOCs events per year for the previous two years prior to enrollment, Hydroxyurea therapy failure or intolerance at any point in the past, Eligible for autologous stem cell transplant as per investigators judgment."

Answered by AI

Who else is applying?

What state do they live in?
Louisiana
How old are they?
18 - 65
What site did they apply to?
The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers
What portion of applicants met pre-screening criteria?
Met criteria
How many prior treatments have patients received?
1

How responsive is this trial?

Typically responds via
Email
Most responsive sites:
  1. The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers: < 48 hours
Average response time
  • < 2 Days
~8 spots leftby May 2026