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CRISPR gene editing offers hope for thalassemia patients to ditch transfusions

NCT ID NCT03655678

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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study tested a single treatment using CRISPR gene editing to modify a person's own blood stem cells, aiming to help people with severe beta-thalassemia stop needing regular blood transfusions. 59 participants received the therapy and were monitored for safety and how long they could go without transfusions. The goal was to see if this one-time treatment could control the disease long-term.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

59 people

The number who actually took part.

Started

Sep 2018

Finished

Nov 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

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: * Diagnosis of transfusion-dependent β-thalassemia (TDT) as defined by 1. Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia/hemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning 2. History of at least 100 mL/kg/year or ≥10 units/year of packed RBC transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening * 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 * Prior allo-HSCT * Participants with associated α-thalassemia and \>1 alpha deletion or alpha multiplications * Participants with sickle cell beta thalassemia variant * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator * White blood cell (WBC) count \<3 × 10\^9/L or platelet count \<50 × 10\^9/L not related to hypersplenism Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major hematologic disorder hemoglobinopathy hereditary disease inherited hemoglobinopathy thalassemia

As listed by the trial registrant

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

Beta-thalassemia Genetic diseases, inborn Hematologic diseases Hemoglobinopathies Thalassemia

Contacts and locations

Locations

  • Ann & Robert Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • British Columbia Children's Hospital

    Vancouver, Canada

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Columbia University Medical Center (21+ years)

    New York, New York, 10032, United States

  • Imperial College Healthcare NHS Trust, Hammersmith Hospital

    London, United Kingdom

  • Lucile Packard Children's Hospital

    Palo Alto, California, 94304, United States

  • Ospedale Pediatrico Bambino Gesù, IRCCS

    Rome, Italy

  • Regensburg University Hospital, Clinic and Polyclinic for Paediatric and Adolescent Medicine

    Regensburg, Germany

  • The Children's Hospital at TriStar Centennial Medical Center/ Sarah Cannon Center for Blood Cancers

    Nashville, Tennessee, 37203, United States

  • The Hospital for Sick Children

    Toronto, Canada

  • University College London Hospitals NHS Foundation Trust

    London, United Kingdom

  • University Hospital Tübingen

    Tübingen, Germany

  • Universitätsklinikum Düsseldorf Hospital Duesseldorf

    Düsseldorf, Germany

More trials for these conditions

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