Status:
RECRUITING
Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Collaborating Sponsors:
CRISPR Therapeutics
Conditions:
Sickle Cell Disease
Hydroxyurea Failure
Eligibility:
All Genders
2-11 years
Phase:
PHASE3
Brief Summary
This is a single-dose, open-label study in pediatric participants with severe SCD and hydroxyurea (HU) failure or intolerance. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9...
Eligibility Criteria
Inclusion
- Key
- 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
- 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.
Key Trial Info
Start Date :
May 2 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 31 2026
Estimated Enrollment :
15 Patients enrolled
Trial Details
Trial ID
NCT05329649
Start Date
May 2 2022
End Date
May 31 2026
Last Update
October 15 2025
Active Locations (7)
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1
Levine Children's Hospital - Hematology
Charlotte, North Carolina, United States, 28203
2
The Children's Hospital of Philadelphia - Hematology
Philadelphia, Pennsylvania, United States, 19104
3
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
4
TriStar Medical Group Children's Specialists - Pediatric Oncology
Nashville, Tennessee, United States, 37203