Evaluating a single dose of CTX001 for treating transfusion-dependent beta-thalassemia and severe sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial is an open-label, single-dose study aimed at assessing the safety and efficacy of CTX001, which involves the use of CRISPR-Cas9 modified human hematopoietic stem and progenitor cells. Participants include individuals with transfusion-dependent beta-thalassemia or severe sickle cell disease who are eligible for autologous stem cell transplant. The study will monitor the participants for improvements in their condition and any potential side effects following treatment.

Why it matters

Who can join: the full rules

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.

Where it's running

• New York Presbyterian Hospital - Morgan Stanley Children's Hospital
  New York, New York, United States Recruiting
• Levine Children's Hospital - Hematology
  Charlotte, North Carolina, United States Recruiting
• TriStar Medical Group Children's Specialists - Pediatric Oncology
  Nashville, Tennessee, United States Recruiting
• University Hospital Dusseldorf - Department of Pediatric Oncology, Hematology and Clinical Immunology
  Düsseldorf, Germany Recruiting
• IRCSS Ospedale Pediatrico Bambino Gesu - Dipartimento di Onco-Ematologia e Terapia Cellulare e Genica
  Rome, Italy Recruiting
• King Faisal Specialist Hospital & Research Centre - Riyadh - Hematology
  Al Mathar Ash Shamali, Saudi Arabia Recruiting

Study contacts

• Study coordinator: Medical Information
• Email: medicalinfo@vrtx.com
• Phone: 6173416777

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Gene editing of your own blood stem cells to boost fetal hemoglobin for severe sickle cell disease and beta-thalassemia

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.