Skip to main content
OpenTrials
Completed

NCT Number: NCT01642758

Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon

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.

Completed

Looking for future studies?

Notify Me

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

16 year–50 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Chronic Care Center

Beirut, Lebanon

About this study

This is a trial of an experimental oral medicine which stimulates production of fetal hemoglobin, an innate type of hemoglobin which is normally made but is suppressed in infancy. Fetal globin (HbF) can perform the function of the missing beta globin and reduce anemia in beta thalassemia, when it is produced in higher amounts than normal.

In this trial, 10 patients with beta thalassemia intermedia in Lebanon will all receive the study drug for 6 months at a dose which has been previously shown to be safe in normal volunteers and in beta thalassemia and sickle cell patients and to stimulate fetal globin production in many, when given for brief periods. The purpose of this trial is the following:

  • To determine if total hemoglobin levels increase above baseline in some subjects when the study drug is taken for 26 weeks.
  • To determine if fetal globin is increased above baseline levels in a proportion of subjects when the study drug is taken for 26 weeks.
  • To determine the number of adverse events which occur with 26 weeks of administration of the study drug in beta thalassemia intermedia subjects.

After a screening period, the subjects will take the study drug at home once a day. They will be seen once every 4 weeks for examinations and laboratory tests during the dosing period and for 4 weeks afterwards.

This trial will provide an important step in evaluating a potential treatment for patients with beta thalassemia intermedia, that can be used around the world, if it is effective and safe.

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
  • 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

Treatment and study plan

Sodium 2,2 dimethylbutyrate

Drug

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

Other names: ST20

Primary outcomes

  1. To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia.

    Time frame: 6 months

    Baseline hemoglobin levels will be determined in each subject and averaged from levels obtained on a screening visit and on day one of the study, before any drug is taken. Hemoglobin levels will then be analyzed every 4 weeks during 26 weeks of taking the study drug and for 4 weeks after the dosing is completed. Changes from baseline will be determined.

Secondary outcomes

  1. To measure the number of adverse events which occur with HQK-1001 treatment when given over 26 weeks in beta thalassemia intermedia.

    Time frame: 6 months

    Adverse events which occur during HQK-1001 administration for 26 weeks will be recorded every 4 weeks.

  2. To measure changes from baseline in HbF during treatment with HQK-1001 for 26 weeks in beta thalassemia intermedia.

    Time frame: 6 months

    Levels of HbF will be averaged from a screening visit and day 1 of the study, prior to any drug treatment. HbF levels will then be measured every 4 weeks during treatment and for 4 weeks after the treatment, and compared to each subject's baseline value. The number of subjects in which an increase in HbF develops above individuals' average baseline value will be obtained.

Sponsors and collaborators

Lead sponsor

Boston University

Other

Collaborators

  • HemaQuest Pharmaceuticals Inc.

Registry information

Official study title

An Open-Label Phase 2 Study of HQK-1001 in Subjects With Beta Thalassemia Intermedia

Acronym: LB-04-THAL

Important dates

Study start
2012
Primary completion
2012
Study completion
2013
First posted
Jul 17, 2012
Registry last updated
Mar 14, 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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.