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NCT Number: NCT01609595

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

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.

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Key information

Conditions

Beta Thalassemia Intermedia Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Thalassemia beta-Thalassemia

Age range

18 year–55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Mahidol University Thalassemia Research Centre

Nakhonpathom, Thailand

About this study

This trial will:

  • 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.
  • Determine the number and proportion of participants in whom treatment with the study drug results in an increase in fetal hemoglobin.
  • Determine the number of participants who have adverse events.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Diagnosis of Beta Thalassemia Intermedia
  • Splenectomized
  • Average of two Hgb levels between 6.0 and 9.0 g/dl

Exclusion criteria

  • Red blood cell transfusion within 3 months of study drug initiation
  • Enlarged spleen
  • Use of hydroxyurea within 6 months
  • QT Segment corrected (QTc)> 450 msec (men) or 470 msec (women) on screening ECG
  • Use of iron chelating agents within 7 days of first dose
  • Alanine Transaminase(ALT)> 4 times the upper limit of normal
  • Use of erythropoiesis stimulating agents (ESAs) within 90 days of first dose
  • serum creatinine > 1.5 mg/dL

Treatment and study plan

Sodium 2,2 dimethylbutyrate

Drug

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

Other names: HQK-1001, ST20

Primary outcomes

  1. The number of participants in which an increase in total hemoglobin of at least 1.5 g/dl above average baseline occurs with study drug treatment.

    Time frame: Within 30 weeks, including 26 weeks of dosing with the study drug

    Baseline hemoglobin levels will be determined by averaging 2 values prior to administration of the study drug.

    The number of participants in which an increase in total hemoglobin of at least 1.5 g/dL above baseline occurs will be determined.

Secondary outcomes

  1. The proportion of participants in which an increase in fetal hemoglobin occurs above the subjects' averaged baseline levels.

    Time frame: Within 30 weeks, including 26 weeks of study drug administration

    Tests of fetal hemoglobin will be obtained at two times prior to administration of the study drug and will be averaged. Laboratory tests of fetal hemoglobin will be assessed monthly during the study drug administration for 26 weeks, as a biomarker of drug activity.

Sponsors and collaborators

Lead sponsor

Boston University

Other

Collaborators

  • Mahidol University

Registry information

Official study title

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

Acronym: ST20-P2T

Important dates

Study start
2012
Primary completion
2012
Study completion
2012
First posted
Jun 1, 2012
Registry last updated
Mar 13, 2013

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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