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Gene Therapy

CRISPR Therapy (CTX001) for Thalassemia

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
History of at least 100 mL/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy <24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up within 42 days after ctx001 infusion
Awards & highlights

Study Summary

This trial will test a new way to treat a rare disease called transfusion-dependent thalassemia (TDT). The treatment involves using a person's own stem cells that have been modified using CRISPR-Cas9. The goal is to see if this is a safe and effective treatment for TDT.

Who is the study for?
This trial is for children with Transfusion-Dependent β-Thalassemia (TDT) who need regular blood transfusions and are suitable for a stem cell transplant. They must have specific genetic forms of TDT confirmed by the study's lab. Those with a perfect match donor, previous transplants, certain sickle cell disease variants, or active infections can't participate.Check my eligibility
What is being tested?
The trial tests CTX001, which involves editing patients' own stem cells using CRISPR-Cas9 technology to potentially treat TDT. It's an open-label study where all participants receive one dose of the modified cells after a conditioning regimen.See study design
What are the potential side effects?
Potential side effects may include reactions related to the stem cell transplant process, immune responses against edited cells, and complications from the gene-editing procedure itself. The exact side effects will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have needed regular blood transfusions for at least 6 months.

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 Achieve Transfusion Independence for at Least 12 Consecutive Months (TI12)
Secondary outcome measures
Change in Fetal Hemoglobin Concentration Over Time
Change in Total Hemoglobin Concentration Over Time
Incidence of All-cause Mortality
+11 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
243 Previous Clinical Trials
32,372 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: NCT05356195 — Phase 3
Blood Diseases Research Study Groups: CTX001
Blood Diseases Clinical Trial 2023: CTX001 Highlights & Side Effects. Trial Name: NCT05356195 — Phase 3
CTX001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05356195 — Phase 3

Frequently Asked Questions

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

Has CTX001 received regulatory approval by the FDA?

"There is some data indicating that CTX001 is effective, and multiple rounds of data supporting its safety, so our team at Power rates it a 3 on a scale of 1 to 3."

Answered by AI

Has recruitment for this experiment begun?

"The clinical trial, which was initially posted on May 3rd, 2022, is still recruiting patients as of August 30th, 2022 according to information found on clinicaltrials.gov."

Answered by AI

How many test subjects are receiving care in this experiment?

"Yes, the trial is recruiting patients right now according to the latest information on clinicaltrials.gov. This study, which was posted on May 3rd, 2022, is looking for 12 patients from 1 location."

Answered by AI

Who meets the requirements to participate in this experiment?

"The eligibility requirements for this clinical trial include being 2-11 years old and having a hematologic disease. So far, 12 patients have been recruited."

Answered by AI

Does this clinical test involve individuals who are over 20 years old?

"To be eligible for this particular study, patients must be aged 2-11. Out of the 201 total trials, 102 are for people under 18 and 99 are for people over 65."

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?
SCRI at the Children's Hospital at TriStar Centennial
What portion of applicants met pre-screening criteria?
Met criteria
Recent research and studies
clinicaltrials.govStudy
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)
2022. "Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05356195.
All > Thalassemia
~8 spots leftby May 2026
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