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Gene Therapy

CTX001 for Sickle Cell Disease

Phase 2 & 3

Waitlist Available

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 sickle cell disease as defined by: Documented severe sickle cell disease genotype History of at least two severe vaso-occlusive crisis events per year for the previous two years prior to enrollment Eligible for autologous stem cell transplant as per investigators judgment

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up within 42 days after ctx001 infusion

Awards & highlights

Study Summary

This trial is testing a possible new treatment for sickle cell disease using CRISPR-Cas9 technology to modify patients' own stem cells. The goal is to improve safety and efficacy compared to current treatments.

Who is the study for?

This trial is for people with severe sickle cell disease, who've had at least two major pain crises a year over the last two years. They must be suitable for their own stem cell transplant but can't have an available perfect-match donor or previous transplants. Active serious infections disqualify them.Check my eligibility

What is being tested?

The study tests CTX001, which uses CRISPR-Cas9 to modify patients' own blood-forming cells aiming to treat severe sickle cell disease. It's a single-arm trial where all participants receive one dose of this gene-editing therapy and are monitored for safety and effectiveness.See study design

What are the potential side effects?

Potential side effects may include reactions related to stem cell transplantation, such as immune system complications, infection risks, and possible off-target genetic effects due to the CRISPR-Cas9 editing process.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have severe sickle cell disease with at least two major pain crises per year.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ within 42 days after ctx001 infusion

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

All-cause mortality

Frequency and severity of collected adverse events (AEs)

Incidence of TRM within 1 year after CTX001 infusion

+4 more

Secondary outcome measures

Change from baseline in haptoglobin over time

Change from baseline in indirect bilirubin over time

Change from baseline in lactate dehydrogenase over time

+23 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). Subjects will receive a single infusion of CTX001 through a central venous catheter.

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Vertex Pharmaceuticals IncorporatedLead Sponsor

243 Previous Clinical Trials

32,342 Total Patients Enrolled

CRISPR TherapeuticsIndustry Sponsor

5 Previous Clinical Trials

261 Total Patients Enrolled

Media Library

CTX001 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT03745287 — Phase 2 & 3

Sickle Cell Disease Research Study Groups: CTX001

Sickle Cell Disease Clinical Trial 2023: CTX001 Highlights & Side Effects. Trial Name: NCT03745287 — Phase 2 & 3

CTX001 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03745287 — Phase 2 & 3

Frequently Asked Questions

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

Are middle-aged adults welcome in this clinical trial?

"People aged 12-35 who meet the other requirements can participate in this trial. Out of the total number of clinical trials, this one is geared towards a younger demographic than usual, with 167 trials catering to those under 18 and 129 for seniors."

Answered by AI

Are there other examples of CTX001's success in medical trials?

"There are two ongoing Phase 3 trials for CTX001. Additionally, 29 research sites are running studies for this treatment across the globe, with a few based in Rome, Illinois."

Answered by AI

Is this study only taking place in North America or are there other continents with test sites as well?

"The institutions hosting this study include the University of Illinois at Chicago Hospitals and Health Systems, Ann & Robert Lurie Children's Hospital of Chicago, St. Jude Children's Research Hospital, and 10 other locations."

Answered by AI

Are new participants being welcomed into this experiment?

"This particular trial is not currently recruiting patients. Although, it was first posted on 27th November 2018 and was last updated on 26th May 2022. There are 273 other studies which are actively recruiting patients with hemoglobinopathies and 2 studies for CTX001 which need participants."

Answered by AI

What are some of the other studies that this research is building off of?

"As of right now, there are 2 ongoing clinical trials for CTX001 in 7 countries and 9 cities. The first trial, which was completed in 2018, had 45 participants and went through Phases 2 & 3 of the drug approval process. Sponsored by CRISPR Therapeutics, this study was the first of its kind. In the 2 years since 2018, no other studies have been completed."

Answered by AI

Could I possibly participate in this medical research?

"Up to 45 adolescents and young adults with hemoglobinopathies may qualify for this study. The ideal candidate will have a documented severe sickle cell disease genotype, a history of at least two severe vaso-occlusive crisis events per year for the previous two years, and be eligible for autologous stem cell transplant according to the study's investigators."

Answered by AI