Phase 2
N=5
A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)
Transfusion Dependent Beta-thalassemia
Bottom Line
View on ClinicalTrials.gov: NCT03432364 ↗Enrolled (actual)
5
Serious AEs
60.0%
Results posted
Dec 2023
Primary outcome: Primary: Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 Weeks After the ST-400 Infusion — 5; 2 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- ST-400 Investigational product (Genetic)
- Age
- Adult · 18+ yrs
- Sex
- All
- Sponsor
- Sangamo Therapeutics
- Primary completion
- Nov 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) up to 156 Weeks After the ST-400 Infusion |
5; 2 | — |
| SECONDARY Clinical Laboratory Measurement of Hemoglobin (Hb) Fractions (A and F in g/dL) |
-3.648; -3.227; -0.281; 1.025; 0.405; 0.502 | — |
| SECONDARY Clinical Laboratory Measurements of Percent (%) HbF |
12.83; 5.30; 4.35 | — |
| SECONDARY Annualized Frequency of Packed RBC Transfusions |
17.393; 21.518; 19.665 | — |
| SECONDARY Annualized Volume (mL) of Packed RBC Transfusions |
10295.984; 7453.191; 7068.788 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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 Criteria
- 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
Data sourced from ClinicalTrials.gov (NCT03432364). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.