Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand — ST20-P2T  

Beta Thalassemia Intermedia Clinical Trial

Official title: An Open Label Academic Phase 2 Study of SDMB in Subjects in Thailand With Beta Thalassemia Intermedia

Status Completed

Clinical Trial Summary

Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells which carries oxygen throughout the body. Beta thalassemias cause progressively severe anemia, widespread organ damage, and often require blood transfusions. There is no FDA approved therapeutic to treat the underlying cause of beta thalassemia. Fetal hemoglobin is another type of endogenous hemoglobin which can replace the reduced beta globin protein, reduce the anemia, and even abolish transfusion requirements. This type of hemoglobin is normally suppressed in infancy.

Sodium 2,2 dimethylbutyrate (ST20, or HQK-1001) is a small molecule which stimulates production of fetal hemoglobin in nonhuman primates and in human patients in Phase I/II trials.

This is a Phase 2 open-label trial to evaluate the ability of this oral therapeutic to reduce anemia in patients with beta thalassemia intermedia, when administered once daily for 26 weeks. All participants will receive the study drug.

Clinical Trial Description

This trial will:

1. Determine the proportion of patients in which treatment with the study drug results in an increase in total hemoglobin by 1.5 g/dl above baseline levels when administered for 26 weeks in Thai patients with beta thalassemia intermedia, including Hemoglobin E beta thalassemia.

2. Determine the number and proportion of participants in whom treatment with the study drug results in an increase in fetal hemoglobin.

3. Determine the number of participants who have adverse events. ;

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta Thalassemia Intermedia
• Beta-Thalassemia
• Thalassemia

• NCT number: NCT01609595
• Study type: Interventional
• Source: Boston University
• Status: Completed
• Phase: Phase 2
• Start date: March 2012
• Completion date: December 2012