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
  Purpose

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.


Condition Intervention Phase
Beta Thalassemia Intermedia
Drug: sodium 2,2 dimethylbutyrate
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open Label Academic Phase 2 Study of SDMB in Subjects in Thailand With Beta Thalassemia Intermedia

Resource links provided by NLM:


Further study details as provided by Boston University:

Primary Outcome Measures:
  • 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.



Secondary Outcome Measures:
  • 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.


Enrollment: 10
Study Start Date: March 2012
Study Completion Date: December 2012
Primary Completion Date: November 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment
Study drug treatment
Drug: sodium 2,2 dimethylbutyrate
Oral capsule, dose 20 mg/kg/day, daily, for 26 weeks
Other Name: HQK-1001, ST20

Detailed 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.
  Eligibility

Ages Eligible for Study:   18 Years to 55 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01609595

Locations
Thailand
Mahidol University Thalassemia Research Centre
Nakhonpathom, Thailand
Sponsors and Collaborators
Boston University
Mahidol University
Investigators
Principal Investigator: Suthat Fuchareon, MD Thalassemia Research Centre, Mahidol University
  More Information

Publications:
Responsible Party: Susan P. Perrine, Sponsor Investigator, Boston University
ClinicalTrials.gov Identifier: NCT01609595     History of Changes
Other Study ID Numbers: ST20-P2T, HQK-1001-Thal P2
Study First Received: April 25, 2012
Last Updated: March 12, 2013
Health Authority: Thailand: Ministry of Public Health

Keywords provided by Boston University:
Thalassemia
Anemia
Fetal hemoglobin

Additional relevant MeSH terms:
Beta-Thalassemia
Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 01, 2014