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Sickle cell gene therapy trial halted after just 4 patients

NCT ID NCT04443907

Disease control Sponsor: Novartis Pharmaceuticals 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 Results may not be published yet.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 This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-phase trial tested a gene-edited stem cell product called OTQ923 in 4 people with severe sickle cell disease. The goal was to boost fetal hemoglobin to reduce painful crises and other complications. The study was terminated early, so we have limited data on safety and effectiveness.

What this could mean

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

Active substance
OTQ923 (genome-edited stem cells)
What this could lead to
If successful, this approach could point toward a one-time treatment that reduces painful crises and other severe complications of sickle cell disease.
What could go wrong
This was a very early, small trial (only 4 participants) that was terminated, so results are limited. Gene-editing therapies also carry risks like failed engraftment or unintended genetic changes.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

4 people

The number who actually took part.

Started

Aug 2020

Finished

Jan 2025

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

2 to 40 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.

Inclusion Criteria: 1. Male or female subjects age 2-40 years inclusive 2. Confirmed diagnosis of sickle cell disease with globin typing (e.g. HbSS, HbSC, HbS/β0-thalassemia or others) 3. Performance status \>70% (Karnofsky for subjects \>16 years of age and Lansky for subjects \<16 years of age) 4. At least one of the following indicators of disease severity as defined in the protocol - Vaso-occlusive pain crisis, Acute chest syndrome, Recurrent priapism, prior stroke, receive chronic transfusions, Red cell alloimmunization 5. Subjects, who have failed, not tolerated or refused hydroxyurea therapy. Exclusion Criteria: 1. Available matched related donor for HSCT 2. Clinically significant active infection 3. Seropositive for HIV or HTLV 4. Active known malignancy, myelodysplasia, abnormal cytogenetics or immunodeficiency 5. Prior HSCT or gene therapy 6. Known hepatic cirrhosis, bridging hepatic fibrosis or active hepatitis 7. Protocol defined iron overload 8. Cerebrovascular procedure within one year, including pial synangiosis for Moyamoya 9. Severe or progressive arteriopathy or cerebrovascular disease, including Moyamoya Other protocol defined inclusion/exclusion criteria may apply

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

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

Sickle cell disease

Contacts and locations

Locations

  • Memorial Sloan Kettering Cancer Ctr

    New York, New York, 10065, United States

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105-3678, United States

  • University of Chicago

    Chicago, Illinois, 60637, United States

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