Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon

• Status: Completed
• Trial end date: 2013-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Boston University
• Collaborator: HemaQuest Pharmaceuticals Inc.

Criteria

Inclusion Criteria:

• Diagnosis of beta thalassemia intermedia

• Ages 16-50 years

• Average total Hgb levels between 6.0 and 9.0 gm/dl within 30 days of initial dose of study drug

• Able to comply with all study procedures

• If female and of childbearing potential, must have a documented negative pregnancy test prior to entry and every 4 weeks

Exclusion Criteria:

• Red blood cell transfusions within 3 months prior to administration of study drug

• QT Segment corrected (QTc)> 450 msec

• Use of Erythropoiesis Stimulating Agents(ESAs)within 9 days of first dose

• Hydroxyurea treatment within 6 months of first study drug

• History of significant arrythmias, syncope, or resuscitation

• Alanine Transaminase (ALT)> 4x upper limit of normal

• Serum creatinine > 1.5 mg/dl

• Sse of iron chelating agents within 7 days of first dose

• Pulmonary hypertension requiring oxygen therapy