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COMPLETED Phase 1

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

NCT03432364 · View on ClinicalTrials.gov ↗

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.

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

FieldValue
Enrollment Target 5 participants
Start Date 2018-03-29
Est. Completion 2022-11-17
Phase Phase 1

Sponsor

Sangamo Therapeutics

10 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT03432364

The ClinicalTrials.gov registry entry for NCT03432364 describes a study currently listed as completed. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 5 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Sangamo Therapeutics, which has 10 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT03432364 reports 6 study locations spanning 5 distinct geographic areas — top geographies include California, Georgia, Massachusetts. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT03432364?

The interventions under investigation include: ST-400 Investigational product (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT03432364?

This trial is sponsored by Sangamo Therapeutics, which has 10 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

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.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial