CRISPR stem cell therapy could end transfusions for sickle cell and thalassemia patients
NCT ID NCT05477563
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a one-time treatment called CTX001, which uses CRISPR gene editing to modify a patient's own stem cells to produce more fetal hemoglobin. The goal is to reduce or eliminate the need for blood transfusions in people with transfusion-dependent beta-thalassemia or severe sickle cell disease. The trial involves 26 participants and includes a conditioning step with chemotherapy before the stem cells are infused.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- CRISPR-edited stem cells (CTX001, also called exagamglogene autotemcel)
- What this could lead to
- If it works, this could free people with severe sickle cell disease or beta-thalassemia from needing regular blood transfusions or lifelong medication.
- What could go wrong
- This is a small, early-phase 3 study with only 26 participants. The treatment requires strong chemotherapy before the infusion, which can cause serious side effects. Long-term safety and effectiveness are still 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
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 26 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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Aug 2022
- Expected to finish
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Jun 2027
An estimate. End dates often move.
- Lead sponsor
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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
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12 to 35 years
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: * Participants with TDT and SCD: * Eligible for autologous stem cell transplant as per investigator's judgment. * Participants with TDT: * Diagnosis of TDT as defined by: * 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 * History of at least 100 milliliter (mL)/kilograms (kg)/year or 10 units/year of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening * Participants with SCD: * 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 Key Exclusion Criteria: * Participants with TDT and SCD: * 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) * Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator * Participants with TDT: * Participants with associated α-thalassemia and \>1 alpha deletion, or alpha multiplications * Participants with sickle cell β-thalassemia variant * Participants with SCD: * History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening Other protocol defined Inclusion/Exclusion criteria may apply.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
6 sites in 4 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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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
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IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
RECRUITINGRome, Italy
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King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
RECRUITINGAl Mathar Ash Shamali, Saudi Arabia
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Levine Children's Hospital - Hematology
RECRUITINGCharlotte, North Carolina, 28203, United States
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New York Presbyterian Hospital - Morgan Stanley Children's Hospital
RECRUITINGNew York, New York, 10032, United States
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TriStar Medical Group Children's Specialists - Pediatric Oncology
RECRUITINGNashville, Tennessee, 37203, United States
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University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
RECRUITINGDüsseldorf, Germany
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding common pain drugs reduce morphine needs in sickle cell crises?
- Gene editing offers hope for a One-Time sickle cell cure
- Can Blood-Type-Matched platelets make transfusions safer?
- Tiny biochip could reveal sickle cell severity
- Can a milder transplant cure sickle cell and thalassemia in adults?
- Can tiny tablets replace protein powder for kids with PKU?