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Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon

NCT01642758 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 10

Last updated 2013-03-14

No results posted yet for this study

Summary

Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce production of beta globin, a major component of adult hemoglobin A, the protein which delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart failure, endocrine deficiencies, and eventually, many require chronic blood transfusions. There is no approved therapeutic for the deficiency of beta globin chains in beta thalassemia.

This trial will study an oral therapeutic which stimulates production of fetal globin, an alternate type which is produced by all humans, but is normally switched off in infancy. This type of globin can compensate for the missing protein in beta thalassemia.

Conditions

  • Beta Thalassemia Intermedia

Interventions

DRUG

Sodium 2,2 dimethylbutyrate

Oral capsules, dose 20 mg/kg/day, once per day for 26 weeks

Sponsors & Collaborators

  • HemaQuest Pharmaceuticals Inc.

    collaborator INDUSTRY

  • Boston University

    lead OTHER

Principal Investigators

  • Susan P Perrine, MD · Boston University

  • Adlette Inati, MD · Chronic Care Center and Rafik Hariri University Hospital, Beirut, Lebanon

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
16 Years
Max Age
50 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-05-31
Primary Completion
2012-11-30
Completion
2013-01-31

Countries

  • Lebanon

Study Locations

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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 NCT01642758 on ClinicalTrials.gov