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CRISPR gene therapy aims to free kids from lifelong blood transfusions

NCT ID NCT05356195

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jul 02, 2026 · Updated 2 times

Summary

This phase 3 trial tests a single dose of CTX001, a CRISPR gene therapy made from the child's own blood stem cells, for children with transfusion-dependent beta-thalassemia. The goal is to help them become transfusion-free for at least 12 months. Sixteen children will receive the treatment after chemotherapy to prepare their bone marrow.

What this could mean

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

Active substance
CTX001 (Exagamglogene autotemcel) - a CRISPR gene therapy made from the patient's own blood stem cells
What this could lead to
If it works, this could allow children with severe beta-thalassemia to stop needing regular blood transfusions for at least a year or more.
What could go wrong
This is an early-phase study with only 16 participants, so results may not apply to everyone. The treatment requires strong chemotherapy beforehand, which carries serious risks like infection and infertility.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

16 people

The number who actually took part.

Started

May 2022

Expected to finish

Nov 2027

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

2 to 11 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 TDT as defined by: * Documented homozygous 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 * History of at least 100 mL/kilograms (kg)/year of packed RBC transfusions in the prior 24 months before signing of consent (or the last rescreening for patients going through repeat screening) or, for participants initiating transfusion therapy \<24 months before signing of consent, requirement for packed RBC transfusion at least every 3 to 4 weeks for ≥6 months * 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 hematopoietic stem cell transplant (HSCT) * Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications * Participants with sickle cell β-thalassemia variant * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator 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

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Hospital for Sick Children - Hematology

    Toronto, Canada

  • IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica

    Rome, Italy

  • St.Mary's Hospital - Children's Clinical Research Facility

    London, United Kingdom

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    Nashville, Tennessee, 37203, United States

  • University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology

    Düsseldorf, Germany

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