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Thiotepa Conditioning for HSCT in Non-Malignant Disorders
NCT03513328Phase 2
Key facts
- Status
- Completed
- Start
- Jun 2018
- Primary completion
- Feb 2022
- Countries
- 1
- Sponsor
- University of Florida
- Modality
- Small Molecules
- Drugs
- thiotepa
- Conditions
- bone marrow failure syndrome (BMFS)thalassemiasickle cell disease (SCD)Diamond-Blackfan anemia (DBA)neutropenia
01
Overview
Phase 2, open-label trial of thiotepa in bone marrow failure syndrome (BMFS), thalassemia, sickle cell disease (SCD) and 13 more (4 arms), sponsored by University of Florida. Primary endpoint: Minimum Effective Dose of Thiotepa at Day 42. Started Jun 2018; status: completed.
02
Design
4 arms- Study design
- Non-randomizedOpen-label
Group A (Low Risk) - 5 mg/kg
Thiotepa 5 mg/kg + Busulfan/Fludarabine/rATG
Group A (Low Risk) - 10 mg/kg
Thiotepa 10 mg/kg + Busulfan/Fludarabine/rATG
Group B (High Risk) - 5 mg/kg
Thiotepa 5 mg/kg + Busulfan/Fludarabine/rATG
Group B (High Risk) - 10 mg/kg
Thiotepa 10 mg/kg + Busulfan/Fludarabine/rATG
03
Who it enrols
Patients with various non-malignant disorders (e.g., hemoglobinopathies, bone marrow failure) requiring allogeneic HSCT. Must have adequate organ function and a suitable donor.
04
Endpoints
Primary
Minimum Effective Dose of Thiotepa
Secondary
Percentage of Subjects With Graft Rejection/Failure
Disease-Free Survival
Overall Survival
Transplant-related Mortality
Grade 2-4 Acute Graft-versus-host Disease
Chronic Graft-versus-host Disease
Transplant-related Complications
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