At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 63 enrolled
Drug / intervention
CTX001biological
Likely dose
Not stated in record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sickle Cell Disease
In Brief
A Phase 3 clinical trial evaluating CTX001 for Sickle Cell Disease and 2 related conditions. Completed, enrolled 63 participants across 17 sites in 7 countries.
Detailed Summary
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesBelgium, Canada, France, Germany, Italy, United Kingdom, United States
CollaboratorsCRISPR Therapeutics
Timeline
Phase 3CompletedFinished
20192020202120222023202420252026
First PostedNov 2018
Enrollment StartNov 2018
Primary CompletionJul 2025
TodayJul 2026
First PostedNov 19, 2018
Enrollment StartNov 27, 2018
Primary CompletionJul 7, 2025
TodayJul 2, 2026
Enrollment to primary: 6.6 yearsPosted 7.6 years ago
Interventions
CTX001biological
Administered by IV infusion following myeloablative conditioning with busulfan.