This could be good news for thalassemia intermedia and Hemoglobin E patients:
HQK-1001 Clinical Trials
Phase 1 Clinical Trials
In January 2008, HemaQuest began a Phase 1 trial of its first drug candidate, HQK-1001, which is initially being developed to treat the hemoglobin disorders, sickle cell disease and beta thalassemia. The first trial administered HQK-1001 to 32 healthy volunteers to evaluate doses that are safe and produce therapeutic drug levels. In the initial trial, normal subjects were treated with increasing single doses of HQK-1001 ranging from 2 to 20 mg/kg. All dose levels were well-tolerated, and there was no difference in the number or severity of side effects between HQK-1001 and placebo.
A multi-dose trial began in March 2008, in which doses of 5, 10 and 15 mg/kg were given once daily for 14 days and tested for safety, pharmacokinetics and potential pharmacodynamic effects in healthy volunteers. In this trial, the drug was also found to be well-tolerated, with no difference in side effects between those receiving placebo and HQK-1001. In addition, the trial demonstrated pharmacodynamic effects documented by increases in reticulocytes, a measure of red blood cell production. Plasma drug concentrations within the therapeutic range (as demonstrated in laboratory studies) were achieved in subjects receiving HQK-1001 at doses of 10 and 15 mg/kg.
Proof of Concept Clinical Trials
HemaQuest is now testing HQK-1001 in patients with sickle cell disease patients the United States and other countries. HemaQuest expects to have data from these clinical studies in the first half of 2011.
Sickle Cell Trial – Projected Enrollment Start Date: April 2010
Number and Title: HQP-1001-SCD-006: A Randomized, Open-Label, Multi-Dose Study of HQK 1001 in Patients with Sickle Cell Disease
This study is designed to test HQK-1001 at three dose levels to determine if it is safe in patients with sickle cell disease. Additionally, the trial will test the three doses of HQK-1001 to look for certain markers that may indicate if the drug is having an effect on this disease. This trial is being conducted in medical centers in the United States and other countries.
Criteria for participation:
The following criteria (along with other criteria) must be evaluated by the trial physician:
Male or female patients from ages 12 to 60 years old with sickle cell disease
Patients must have had at least three episodes of a sickle cell crisis in the three years prior to the study OR at least one episode of acute chest syndrome over the 5 years prior to the study
A fetal hemoglobin (HbF) level greater than or equal to 2%
If receiving Hydroxyurea, the patient must have been on a stable dose for 6 months prior to the study
Patients may not be eligible if they have the following medical conditions:
Severe pulmonary hypertension
Significant electrocardiogram abnormalities
Blood transfusions within 4 months
More than 4 sickle cell events requiring hospitalization with the last 12 months
Further criteria that may include or exclude patient will be assessed by the physician
For information about this trial email: info@hemaquest.com.
More information can be found at www.clinicaltrials.gov
Sickle Cell Trial – Completed
Number and Title: HQP-2008-004: A Randomized, Blinded, Placebo-controlled, Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacokinetics of HQK 1001 in Subjects with Sickle Cell Disease
Protocol HQP-2008-004 is designed to test HQK-1001 to determine if it is safe in patients with sickle cell disease or sickle beta thalassemia. Additionally, the trial is designed to test different doses of HQK-1001 and to look for certain markers that may indicate if the drug is having an effect in these diseases. This trial is being conducted in medical centers in the United States and Jamaica.
Criteria for participation:
The following criteria (along with other criteria) must be evaluated by the trial physician:
Male or female patients from ages 12 to 60 years old with sickle cell disease or sickle beta thalassemia
Patients must have had an average of 1 episode of sickle cell crisis for the last three years OR one episode of acute chest syndrome within the last 5 years
A fetal hemoglobin (HbF) level greater than or equal to 2%
Patients may not be eligible if they have the following medical conditions:
Severe pulmonary hypertension
Significant electrocardiogram abnormalities
Transfusions within 3 months
More than 4 sickle cell events requiring hospitalization with the last 12 months
Further criteria that may include or exclude patient will be assessed by the physician
For information about this trial email: info@hemaquest.com.
More information can be found at www.clinicaltrials.gov
Thalassemia Clinical Trial – Completed
Number and Title: HQ:-2008-003: A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects with Beta Thalassemia Intermedia, including Hemoglobin E Beta Thalassemia
Protocol HQP-2008-003 is designed to test HQK-1001 to determine if it is safe in patients with thalassemia intermedia or hemoglobin E beta thalassemia. Additionally, the trial is designed to test different doses of HQK-1001 and to look for certain markers that may indicate if the drug is having an effect in these diseases. This trial is being conducted in medical centers in Bangkok, Thailand, and Beirut, Lebanon.
Criteria for participation:
The following criteria (along with other criteria) must be evaluated by the trial physician:
Female and male patients from the ages of 12 to 60 years old with thalassemia intermedia or hemoglobin E beta thalassemia
Baseline hemoglobin levels less than 10 g/dL
Patients may not be eligible if they have the following medical conditions:
Greatly enlarged spleen
Severe pulmonary hypertension
Significant electrocardiogram abnormalities
Transfusions within 3 months
Recent fevers
Certain medications
Clinically significant laboratory abnormalities
Further criteria that may include or exclude patient will be assessed by the physician
http://www.hemaquest.com/clinical/trials_HQK-1001.aspThe thal intermedia/hg E study has been completed. Does anyone know the results? Are they yet available?
Sharmin