Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
Raktažodžiai
Santrauka
apibūdinimas
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:
1. To determine if total hemoglobin levels increase above baseline in some subjects when the study drug is taken for 26 weeks.
2. To determine if fetal globin is increased above baseline levels in a proportion of subjects when the study drug is taken for 26 weeks.
3. 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.
Datos
| Paskutinį kartą patikrinta: | 02/28/2013 |
| Pirmasis pateikimas: | 07/11/2012 |
| Numatytas registravimas pateiktas: | 07/15/2012 |
| Pirmas paskelbtas: | 07/16/2012 |
| Paskutinis atnaujinimas pateiktas: | 03/12/2013 |
| Paskutinis atnaujinimas paskelbtas: | 03/13/2013 |
| Faktinė studijų pradžios data: | 04/30/2012 |
| Numatoma pirminio užbaigimo data: | 10/31/2012 |
| Numatoma studijų užbaigimo data: | 12/31/2012 |
Būklė ar liga
Intervencija / gydymas
Drug: Sodium 2,2 dimethylbutyrate
Fazė
Rankų grupės
| Ranka | Intervencija / gydymas |
|---|---|
| Experimental: Sodium 2,2 dimethylbutyrate A single dose (20 mg/kg/day) of study drug will be taken once per day by mouth. | Drug: Sodium 2,2 dimethylbutyrate Oral capsules, dose 20 mg/kg/day, once per day for 26 weeks |
Tinkamumo kriterijai
| Amžius, tinkami studijuoti | 16 Years Į 16 Years |
| Tinkamos studijoms lytys | All |
| Priima sveikus savanorius | Taip |
| Kriterijai | 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 |
Rezultatas
Pirminės rezultatų priemonės
1. To measure changes from baseline in total hemoglobin when HQK-1001 is administered orally for 26 weeks in subjects with beta thalassemia intermedia. [6 months]
Antrinės rezultatų priemonės
1. To measure the number of adverse events which occur with HQK-1001 treatment when given over 26 weeks in beta thalassemia intermedia. [6 months]
2. To measure changes from baseline in HbF during treatment with HQK-1001 for 26 weeks in beta thalassemia intermedia. [6 months]
