A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
Completed · Phase 1/Phase 2
Conditions studied: Transfusion Dependent Beta-thalassemia
In brief
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.
Key facts
- Study ID
- NCT03432364
- Run by
- Sangamo Therapeutics
- People needed
- 5
- Starts
- 2018-03-29
- Expected to finish
- 2022-11-17
- Last updated by the study team
- 2023-12-14
Who can join
Age: 18 and older, up to 40. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Informed Consent
- Clinical diagnosis of TDT with ≥ 8 documented RBC transfusion events per year on an annualized basis in the 2-years prior to screening
- Confirmed beta-thalassemia diagnosis by molecular genetic testing
- Clinically stable and eligible to receive conditioning chemotherapy
- Able and willing to use an effective method of contraception from the signing of the informed consent and for one year following ST-400 infusion.
You may not qualify if…
- Previous history of autologous or allogeneic blood stem cell transplantation or solid organ transplantation
- Pregnant or breastfeeding female
- Medical contraindication to mobilization, apheresis, or conditioning
- Significant liver, lung, heart, or kidney dysfunction
- Diagnosis of HIV or evidence of active HBV or HCV
- History of significant bleeding disorder or uncontrolled seizures
- History of active malignancy in past 5 years (non-melanoma skin cancer or cervical cancer in situ permitted) any history of hematologic malignancy, or family history of cancer predisposition syndrome without negative testing result in the study candidate.
- Currently participating in another clinical trial using an investigational study medication, or recent participation in such a trial
- Previous treatment with gene therapy
Where it is running
- University of California, Los Angeles — Los Angeles, California, United States
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
- Children's Healthcare of Atlanta — Atlanta, Georgia, United States
- Dana-Farber Boston Children's Cancer and Blood Disorders Center — Boston, Massachusetts, United States
- University of Minnesota — Minneapolis, Minnesota, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
Full record on ClinicalTrials.gov
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