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CRISPR gene therapy: a Long-Term safety check for blood disease patients

NCT ID NCT04208529

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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 12, 2026 · Updated 2 times

Summary

This study follows 160 children and adults with beta-thalassemia or sickle cell disease who received a one-time treatment of their own CRISPR-edited stem cells (CTX001). The goal is to monitor long-term safety, including any new cancers or blood disorders, and to see how well the therapy works over time. Participants join after completing earlier studies, and there are no new exclusion criteria.

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

About 160 people

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

Started

Jan 2021

Expected to finish

Sep 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

2 years and older

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: * Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form * Participants must have received CTX001 infusion in a parent study Exclusion Criteria: * There are no exclusion criteria

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Conditions

The condition(s) this trial relates to.

beta thalassemia beta-thalassemia major hematologic disorder hemoglobinopathy hereditary disease inherited hemoglobinopathy sickle cell disease 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 Sickle cell anemia Sickle cell disease Thalassemia

How to take part

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

  1. The places running it

    20 sites in 6 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. 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

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago - Hematology

    Chicago, Illinois, 60611, United States

  • Center for Pediatric Clinical Studies (CPCS)

    Klinik Für Kinder- Und Jugendmedizin, Germany

  • Great Ormond Street Hospital for Children

    London, United Kingdom

  • Hammersmith Hospital - Haematology Dept

    London, United Kingdom

  • Herbert Irving Pavilion - Hematology

    New York, New York, 10032, United States

  • Hopital Universitaire des Enfants Reine Fabiola (HUDERF) - Hematology

    Brussels, Belgium

  • Hospital for Sick Children - Hematology

    Toronto, Canada

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

    Rome, Italy

  • Levine Children's Hospital - Hematology

    Charlotte, North Carolina, 28203, United States

  • Lucile Packard Children's Hospital

    Palo Alto, California, 94304, United States

  • Methodist Healthcare System of San Antonio, Methodist Hospital, Methodist Children's Hospital

    San Antonio, Texas, 78229, United States

  • New York Presbyterian Hospital - Morgan Stanley Children's Hospital

    New York, New York, 10032, United States

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

    Regensburg, Germany

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • St. Paul's Hospital - Hematology

    Vancouver, Canada

  • The Children's Hospital of Philadelphia - Hematology

    Philadelphia, Pennsylvania, 19104, United States

  • Toronto General Hospital - Hematology

    Toronto, Canada

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    Nashville, Tennessee, 37203, United States

  • University College London Hospital NHS Foundation - Main

    London, United Kingdom

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

    Düsseldorf, Germany

More trials for these conditions

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