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

Gene-Editing breakthrough? new therapy aims to stop sickle cell pain crises

NCT ID NCT05456880

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026 · Updated 2 times

Summary

This early-stage trial is testing a new treatment called BEAM-101 for people with severe sickle cell disease. The therapy uses a patient's own blood stem cells, which are gene-edited in a lab to produce healthy red blood cells. After a short course of chemotherapy, the edited cells are infused back into the patient. The goal is to reduce or eliminate the painful blockages (vaso-occlusive crises) that are a hallmark of the disease. The study is currently enrolling 15 participants aged 12 to 35.

What this could mean

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

Active substance
BEAM-101 (a gene-edited stem cell therapy made from the patient's own blood cells)
What this could lead to
If it works, this could provide a long-term reduction in severe pain crises for people with sickle cell disease, potentially reducing the need for frequent hospital visits.
What could go wrong
This is an early, small trial (15 people) testing safety and effectiveness. The treatment requires strong chemotherapy before the infusion, which carries serious risks like infection and infertility. It may not work for everyone.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

About 15 people

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

Started

Aug 2022

Expected to finish

Feb 2028

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 Include: 1. Age ≥12 years to ≤35 years 2. Documented diagnosis of sickle cell disease with βS/βS, βS/β0, or βS/β+ genotypes. 3. Severe SCD defined by the occurrence of at least 4 severe VOCs in the 24 months prior to screening despite receiving hydroxyurea or other supportive care measures Key Exclusion Criteria Include: 1. HbF levels \>20%, obtained at the time of screening on or off hydroxyurea therapy 2. Previous receipt of an autologous or allogeneic HSCT or solid organ transplantation 3. Available and willing matched sibling donor 4. Definitive diagnosis of moyamoya syndrome based on screening brain MRA 5. History of overt stroke

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

As listed by the trial registrant

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

Sickle cell disease

Contacts and locations

Locations

  • Boston Children's Hospital

    Boston, Massachusetts, 02215, United States

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

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Henry Ford Cancer Center

    Detroit, Michigan, 48202, United States

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224, United States

  • Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Memorial Sloan Kettering Cancer Center

    New York, New York, 10065, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • St Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • The Children's Hospital at TriStar Centennial

    Nashville, Tennessee, 37203, United States

  • The Cleveland Clinic Foundation

    Cleveland, Ohio, 44195, United States

  • University Hospitals Cleveland Medical Center

    Cleveland, Ohio, 44106, United States

  • University of Alabama at Birmingham

    Birmingham, Alabama, 35233, United States

  • University of Miami

    Miami, Florida, 33136-1005, United States

  • University of Minnesota

    Minneapolis, Minnesota, 55455, United States

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110, United States

More trials for these conditions

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