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CRISPR stem cell therapy shows promise for kids with sickle cell

NCT ID NCT05329649

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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-edited stem cell therapy, in 13 children with severe sickle cell disease who have not responded to standard treatments. The goal is to see if it can prevent severe pain crises for at least a year. Participants receive their own modified stem cells after chemotherapy to prepare the body.

What this could mean

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

Active substance
CTX001 (CRISPR-edited stem cells)
What this could lead to
If it works, this could offer children with severe sickle cell disease a long-term reduction in painful crises and hospital stays.
What could go wrong
This is a small, early-phase study (13 kids) and requires intensive chemotherapy before the infusion, which carries serious risks. Long-term effects are unknown.

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

13 people

The number who actually took part.

Started

May 2022

Expected to finish

Jun 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 severe SCD as defined by: * Documented SCD genotypes * History of at least two severe VOCs events per year for the previous two years prior to enrollment * Hydroxyurea (HU) failure unless HU intolerant * Eligible for autologous stem cell transplant as per investigators judgment Key Exclusion Criteria: * A willing and healthy 10/10 human leukocyte antigen (HLA)-matched related donor * Prior hematopoietic stem cell transplant (HSCT). * Clinically significant and active bacterial, viral, fungal, or parasitic infection Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

hematologic disorder hemoglobinopathy inherited hemoglobinopathy sickle cell disease

As listed by the trial registrant

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

Hematological diseases Hemoglobinopathies Hydroxyurea failure Hydroxyurea intolerance Sickle cell disease

Contacts and locations

Locations

  • 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

  • St. Jude Children's Research Hospital

    Memphis, Tennessee, 38105, United States

  • St.Mary's Hospital - Haematology Dept

    London, United Kingdom

  • The Children's Hospital of Philadelphia - Hematology

    Philadelphia, Pennsylvania, 19104, United States

  • TriStar Medical Group Children's Specialists - Pediatric Oncology

    Nashville, Tennessee, 37203, United States

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

    Düsseldorf, Germany

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