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Gene therapy Follow-Up: 15-Year watch for sickle cell patients

NCT ID NCT07373639

Disease control Sponsor: Beam Therapeutics Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 long-term follow-up study tracks 50 patients with sickle cell disease who received the experimental gene therapy BEAM-101. The goal is to monitor safety, side effects, and overall health for up to 15 years after treatment. Participants will have regular check-ups to see how well the therapy works over time.

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.

Participants

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Started

Dec 2025

Expected to finish

Feb 2043

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.

Who is studied

The study population consists of participants enrolled at sites from the parent study (BTX-AUT-001) who meet the criteria for long-term follow-up

Ages

14 to 37 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. Have received BEAM-101 in Study BTX-AUT-001 and are in the process of completing that study's end-of-study (EOS) visit. 2. Provide signed, written informed consent according to local institutional review board (IRB)/independent ethics committee (IEC) and institutional requirements. Exclusion Criteria: * There are no exclusion criteria for this study.

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Conditions

The condition(s) this trial relates to.

hemoglobinopathy inherited hemoglobinopathy sickle cell disease

As listed by the trial registrant

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

Hemoglobinopathy Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    17 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Boston Children's Hospital

    RECRUITING

    Boston, Massachusetts, 02215, United States

    Contact Email: •••••@•••••

  • Children's Healthcare of Atlanta - Aflac Cancer and Blood Disorders Center- Egleston

    NOT_YET_RECRUITING

    Atlanta, Georgia, 30329, United States

    Contact Email: •••••@•••••

  • Children's Hospital of Philadelphia

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact Email: •••••@•••••

  • Columbia University Irving Medical Center

    NOT_YET_RECRUITING

    New York, New York, 10032, United States

    Contact Email: •••••@•••••

  • Hackensack University Medical Center

    NOT_YET_RECRUITING

    Hackensack, New Jersey, 07601, United States

    Contact Email: •••••@•••••

  • Henry Ford Cancer Center

    NOT_YET_RECRUITING

    Detroit, Michigan, 48202, United States

    Contact Email: •••••@•••••

  • Mayo Clinic Florida

    NOT_YET_RECRUITING

    Jacksonville, Florida, 32224, United States

    Contact Email: •••••@•••••

  • Medical College of Wisconsin

    NOT_YET_RECRUITING

    Milwaukee, Wisconsin, 53226, United States

    Contact Email: •••••@•••••

  • Medical University of South Carolina

    NOT_YET_RECRUITING

    Charleston, South Carolina, 29425, United States

    Contact Email: •••••@•••••

  • Phoenix Children's Hospital

    NOT_YET_RECRUITING

    Phoenix, Arizona, 85016, United States

    Contact Email: •••••@•••••

  • St. Jude Children's Research Hospital

    NOT_YET_RECRUITING

    Memphis, Tennessee, 38105, United States

    Contact Email: •••••@•••••

  • The Children's Hospital at TriStar Centennial

    NOT_YET_RECRUITING

    Nashville, Tennessee, 37203, United States

    Contact Email: •••••@•••••

  • The Clevland Clinic Foundation

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

    Contact Email: •••••@•••••

  • University Hospitals Clevland Medical Center

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44106, United States

    Contact Email: •••••@•••••

  • University of Alabama at Birmingham

    NOT_YET_RECRUITING

    Birmingham, Alabama, 35233, United States

    Contact Email: •••••@•••••

  • University of Minnesota

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

    Contact Email: •••••@•••••

  • Washington University School of Medicine in St. Louis

    NOT_YET_RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Email: •••••@•••••

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