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Gene-edited stem cells: a lasting fix for sickle cell?

NCT ID NCT06155500

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 This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Jul 29, 2026 · Updated 3 times

Summary

This study follows 4 people with sickle cell disease who previously received a one-time treatment of their own gene-edited stem cells (OTQ923). Researchers are checking for delayed side effects, such as new cancers or blood disorders, and measuring how long fetal hemoglobin production lasts. The goal is to see if the treatment remains safe and effective over time.

What this could mean

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

Active substance
OTQ923 (gene-edited stem cells)
What this could lead to
If successful, this could show that a single treatment with gene-edited stem cells provides lasting control of sickle cell disease symptoms.
What could go wrong
This is a very small, early-phase follow-up study with only 4 participants, so results may not apply broadly. Long-term risks like new cancers or blood disorders are still being checked.

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

Apr 2024

Expected to finish

Jan 2039

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

18 to 100 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. Patients must have received gene therapy treatment from the parent treatment protocol (CADPT03A12101). 2. Patients must provide informed consent prior to their entry into this study. Exclusion Criteria: 1\. Completion of less than 1 year of safety follow-up in the treatment protocol (CADPT03A12101).

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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 Childrens Research Hospital

    Memphis, Tennessee, 38105, United States

  • University of Chicago

    Chicago, Illinois, 60637, United States

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