Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia

Beta Thalassemia Clinical Trial

Official title:

A Multi-National, Blinded, Placebo-Controlled, Dose Escalation Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of HQK-1001 in Subjects With Beta Thalassemia Intermedia, Including Hemoglobin E Beta Thalassemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00790127
• Other study ID #: HQP-2008-003b
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: November 11, 2008
• Last updated: May 18, 2011
• Start date: March 2009
• Est. completion date: May 2010

Study information

• Verified date: May 2011
• Source: HemaQuest Pharmaceuticals Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationThailand: Food and Drug AdministrationThailand: Ethical CommitteeLebanon: Ministry of Public HealthLebanon: Institutional Review Board
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered daily for 8 weeks in subjects with beta thalassemia intermedia

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: May 2010
• Est. primary completion date: May 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 12 Years to 60 Years

Eligibility

Inclusion Criteria:

• Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia

• Average of the initial two hemoglobin levels of less than 10 g/dL within = 30 days prior to randomization.

• Age = 12 and = 60 years

• Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures

• If female and of childbearing potential, must have a documented negative pregnancy test at Day -1 and must agree to use one or more locally medically accepted methods of contraception in the month prior to randomization, through the entire study, and for 4 weeks after the last dosing of study medication

Exclusion Criteria:

• Spleen palpable = 2 cm below the left costal margin

• Pulmonary hypertension requiring oxygen therapy

• QTc > 450 msec on screening ECG

• Infection with hepatitis C, hepatitis B requiring therapy

• Known infection with HIV

• Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels

• Fever greater than 38.5°C in the week prior to administration of study medication

• ALT > 4x upper limit of normal (ULN)

• Baseline elevation of CPK value prior to randomization

• Treatment with hydroxyurea within 2 months prior to administration of study medication

• Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies)

• Serum creatinine > 1.5 mg/dl

• Received investigational systemic therapy within 30 days prior to randomization

• Currently pregnant or breast feeding a child

• Subject history of clinically significant arrhythmias or syncope

• Known current drug or alcohol abuse

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Beta Thalassemia
• Beta-Thalassemia
• Thalassemia

Intervention

Drug:
• HQK-1001
HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days
• Placebo
Matching placebo capsules administered once a day, orally, for 56 days

Locations

Country	Name	City	State
Lebanon	Chronic Care Center	Beirut
Thailand	Siriraj Hospital	Bangkok	Bangkoknoin District

Sponsors (1)

Lead Sponsor	Collaborator
HemaQuest Pharmaceuticals Inc.

Countries where clinical trial is conducted

Lebanon,  Thailand, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam		168 days	Yes
Secondary	Pharmacokinetics assessed by plasma drug concentration levels		140 days	No
Secondary	Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin		168 days	No