Status:
COMPLETED
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
Lead Sponsor:
Sangamo Therapeutics
Conditions:
Transfusion Dependent Beta-thalassemia
Eligibility:
All Genders
18-40 years
Phase:
PHASE1
PHASE2
Brief 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 inves...
Detailed Description
Once consented, study participants will progress through the following stages: * Screening: in-person visit at the study site to confirm eligibility for proceeding * Collection: autologous (self) blo...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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
Key Trial Info
Start Date :
March 29 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 17 2022
Estimated Enrollment :
5 Patients enrolled
Trial Details
Trial ID
NCT03432364
Start Date
March 29 2018
End Date
November 17 2022
Last Update
December 14 2023
Active Locations (6)
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1
University of California, Los Angeles
Los Angeles, California, United States, 90095-1678
2
UCSF Benioff Children's Hospital Oakland
Oakland, California, United States, 94609
3
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322
4
Dana-Farber Boston Children's Cancer and Blood Disorders Center
Boston, Massachusetts, United States, 02116