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

This study has been completed.
Sponsor:
Collaborator:
Mahidol University
Information provided by (Responsible Party):
Susan P. Perrine, Boston University
ClinicalTrials.gov Identifier:
NCT01609595
First received: April 25, 2012
Last updated: March 12, 2013
Last verified: March 2013

April 25, 2012
March 12, 2013
March 2012
November 2012   (final data collection date for primary outcome measure)
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 ] [ Designated as safety issue: No ]

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.

Same as current
Complete list of historical versions of study NCT01609595 on ClinicalTrials.gov Archive Site
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 ] [ Designated as safety issue: No ]
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.
Same as current
Not Provided
Not Provided
 
Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
An Open Label Academic Phase 2 Study of SDMB in Subjects in Thailand With Beta Thalassemia Intermedia

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.

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.
Interventional
Phase 2
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Beta Thalassemia Intermedia
Drug: sodium 2,2 dimethylbutyrate
Oral capsule, dose 20 mg/kg/day, daily, for 26 weeks
Other Name: HQK-1001, ST20
Experimental: Treatment
Study drug treatment
Intervention: Drug: sodium 2,2 dimethylbutyrate

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
10
December 2012
November 2012   (final data collection date for primary outcome measure)

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
Both
18 Years to 55 Years
No
Contact information is only displayed when the study is recruiting subjects
Thailand
 
NCT01609595
ST20-P2T, HQK-1001-Thal P2
Yes
Susan P. Perrine, Boston University
Boston University
Mahidol University
Principal Investigator: Suthat Fuchareon, MD Thalassemia Research Centre, Mahidol University
Boston University
March 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP