Den här översättningen är inte klar ännu. Den här sidan är just nu på engelska.

Gå till den engelska sidan

Cure My Disease

Don't miss your cure!

 Log in / Sign up

Sickle cell gene therapy trial pulled before it even started

NCT ID NCT05951205

Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled This study
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study was designed to test a single dose of exa-cel, a gene-edited cell therapy, in people with severe sickle cell disease who have the HbSC genotype. Participants would have received their own modified stem cells after chemotherapy. However, the trial was withdrawn before any patients were enrolled, so no data on safety or effectiveness were collected.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Exa-cel (also called Exagamglogene autotemcel or CTX001), a gene-edited cell therapy made from the patient's own blood stem cells
What this could lead to
If it worked, this could provide a one-time treatment to reduce or eliminate sickle cell crises for people with the HbSC genotype.
What could go wrong
The trial was withdrawn before enrolling anyone, so no results exist. Gene-editing therapies can have serious side effects, including infertility and blood cancers, and require intensive chemotherapy beforehand.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Expected to start

Jul 2027

An estimate. Start dates often move.

Expected to finish

Dec 2033

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 35 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * Participants with documented βS/βC (HbSC) genotype * Participants must be eligible for autologous stem cell transplant as per investigator's judgment Key Exclusion Criteria: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement * Participants with prior hematopoietic stem cell transplant (HSCT) * Treatment with regular RBC transfusions that, in the opinion of the investigator, cannot be interrupted after engraftment. Other protocol defined Inclusion/Exclusion criteria may apply.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Sickle cell disease are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

sickle cell disease sickle cell-hemoglobin c disease syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

More trials for these conditions

Other studies related to the condition(s) this trial covers.