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Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand

NCT01609595 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2013-03-13

No results posted yet for this study

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.

Conditions

  • Beta Thalassemia Intermedia

Interventions

DRUG

sodium 2,2 dimethylbutyrate

Oral capsule, dose 20 mg/kg/day, daily, for 26 weeks

Sponsors & Collaborators

  • Mahidol University

    collaborator OTHER

  • Boston University

    lead OTHER

Principal Investigators

  • Suthat Fuchareon, MD · Thalassemia Research Centre, Mahidol University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
55 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-03-31
Primary Completion
2012-11-30
Completion
2012-12-31

Countries

  • Thailand

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01609595 on ClinicalTrials.gov