CTX001 for Sickle Cell Disease

Recruiting at 17 trial locations

Overseen ByMedical Information

Age: < 65

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Vertex Pharmaceuticals Incorporated

Disqualifiers: Prior HSCT, Active infection, others

No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Prior Safety DataThis treatment has passed at least one previous human trial

Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What makes the drug CTX001 unique for treating Sickle Cell Disease?

CTX001 is a novel gene therapy that uses CRISPR technology to edit the patient's own stem cells, enabling them to produce fetal hemoglobin, which can reduce the symptoms of Sickle Cell Disease. This approach is different from traditional treatments that mainly focus on managing symptoms rather than addressing the underlying genetic cause.¹ ² ³ ⁴ ⁵

Eligibility Criteria

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.

Inclusion Criteria

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

Exclusion Criteria

I have a perfect match for a stem cell donor and no active infections.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single infusion of CTX001 through a central venous catheter

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months up to 2 years

Regular visits (in-person and virtual)

Treatment Details

Interventions

CTX001

Trial OverviewThe 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.

Participant Groups

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.

CTX001 is already approved in European Union, United States for the following indications: