Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD)

NCT05329649 · clinicaltrials.gov ↗

PHASE3

Phase

ACTIVE_NOT_RECRUITING

Status

13

Enrollment

INDUSTRY

Sponsor class

Conditions

Sickle Cell Disease
Hydroxyurea Failure
Hydroxyurea Intolerance
Hemoglobinopathies
Hematological Diseases

Interventions

BIOLOGICAL: CTX001

Sponsor

Vertex Pharmaceuticals Incorporated

Collaborators

CRISPR Therapeutics