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NCT03432364 · ClinicalTrials.gov registry record · Phase 1
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
A Phase 1 study of Transfusion-dependent Beta-Thalassemia, sponsored by Sangamo Therapeutics.
- Completed
- Registry status
- Phase 1
- Development phase
- 5
- Enrollment target
- 6
- Study locations
NCT03432364: Completed Phase 1 study of Transfusion-dependent Beta-Thalassemia, sponsored by Sangamo Therapeutics.
NCT03432364 is a Phase 1 study of Transfusion-dependent Beta-Thalassemia that has completed, run by Sangamo Therapeutics. The registered enrollment target is 5 participants, below the 111-participant average among 3 other Transfusion-dependent Beta-Thalassemia trials with a reported enrollment target (95% lower). The trial reports 6 study locations across 5 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03432364, a Phase 1 study of Transfusion-dependent Beta-Thalassemia, has completed, sponsored by Sangamo Therapeutics.
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 5 participants
- Enrollment target
- 6
- Study locations
Study Summary
This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age. ST-400 is a type of investigational therapy that consists of gene edited cells. ST-400 is composed of the patient's own blood stem cells which are genetically modified in the laboratory using Sangamo's zinc finger nuclease (ZFN) technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene (which normally suppresses fetal hemoglobin production in erythrocytes). This process is intended to boost fetal hemoglobin (HbF), which can substitute for reduced or absent adult (defective) hemoglobin. ST-400 is then infused back into the patient after receiving conditioning chemotherapy to make room for the new cells in the bone marrow, with the aim of producing new erythrocytes with increased amounts of HbF. The primary objective is to understand safety and tolerability of ST-400, and secondary objectives are to assess the effects on HbF levels and transfusion requirements.
Primary Outcome
Safety and tolerability assessed by number of participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 weeks after the ST-400 infusion
Conditions Studied
Interventions
- GENETIC ST-400 Investigational product
Study Locations (6)
California
- University of California, Los Angeles - Los Angeles
- UCSF Benioff Children's Hospital Oakland - Oakland
Georgia
- Children's Healthcare of Atlanta - Atlanta
Massachusetts
- Dana-Farber Boston Children's Cancer and Blood Disorders Center - Boston
Minnesota
- University of Minnesota - Minneapolis
Pennsylvania
- Children's Hospital of Philadelphia - Philadelphia
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 5 participants |
| Start Date | 2018-03-29 |
| Est. Completion | 2022-11-17 |
| Phase | Phase 1 |
What the finished NCT03432364 record still lists
NCT03432364 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 5 participants, a relatively small participant target, below the 111-participant average among 3 other Transfusion-dependent Beta-Thalassemia trials with a reported enrollment target (95% lower).
The record links to 1 condition, with Transfusion-dependent Beta-Thalassemia appearing as the primary indexed condition, and to 1 intervention - of which ST-400 Investigational product is the first listed.
NCT03432364 lists 6 locations in 5 states (California, Georgia, Massachusetts).
Frequently Asked Questions
What is clinical trial NCT03432364 about?
NCT03432364 is a clinical study titled "A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)". This is a single-arm, multi-site, single-dose, Phase 1/2 study to assess ST-400 in 6 subjects with transfusion-dependent β-thalassemia (TDT) who are ≥18 and ≤40 years of age. ST-400 is a type of investigational therapy that consists of gene edited cells. ST-400 is composed of the patient's own blood...
What is the current status of trial NCT03432364?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 5 participants. The study started on 2018-03-29. Estimated completion is 2022-11-17.
What conditions does trial NCT03432364 study?
This clinical trial studies the following conditions: Transfusion-dependent Beta-Thalassemia.
What interventions are being tested in trial NCT03432364?
The interventions under investigation include: ST-400 Investigational product (GENETIC).
Who is sponsoring clinical trial NCT03432364?
This trial is sponsored by Sangamo Therapeutics, which has 25 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03432364 being conducted?
This trial has 6 study locations across California, Georgia, Massachusetts, Minnesota, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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