At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 59 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 of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects With Transfusion-Dependent β-Thalassemia
In Brief
A Phase 3 clinical trial evaluating CTX001 for Beta-Thalassemia and 4 related conditions. Completed, enrolled 59 participants across 14 sites in 5 countries.
Detailed Summary
This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in participans with transfusion-dependent β-thalassemia (TDT). 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--
ConditionsBeta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
CountriesCanada, Germany, Italy, United Kingdom, United States
CollaboratorsCRISPR Therapeutics
Timeline
Phase 3CompletedFinished
20192020202120222023202420252026
First PostedAug 2018
Enrollment StartSep 2018
Primary CompletionNov 2025
TodayJul 2026
First PostedAug 31, 2018
Enrollment StartSep 14, 2018
Primary CompletionNov 13, 2025
TodayJul 2, 2026
Enrollment to primary: 7.2 yearsPosted 7.8 years ago
Interventions
CTX001biological
Administered by IV infusion following myeloablative conditioning with busulfan