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

• ClinicalTrials.gov Identifier: NCT00790127
• Recruitment Status: Completed
• First Posted: November 13, 2008
• Last Update Posted: May 20, 2011
• Sponsor: HemaQuest Pharmaceuticals Inc.
• Information provided by: HemaQuest Pharmaceuticals Inc.

Tracking Information

• First Submitted Date: November 11, 2008
• First Posted Date: November 13, 2008
• Last Update Posted Date: May 20, 2011
• Study Start Date: March 2009
• Actual Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: November 12, 2008): Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam [ Time Frame: 168 days ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 12, 2008)

• Pharmacokinetics assessed by plasma drug concentration levels [ Time Frame: 140 days ]
• Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin [ Time Frame: 168 days ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
• 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
• Brief 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
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 1; Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Beta Thalassemia

Intervention

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

Study Arms

• Placebo Comparator: Placebo
  Placebo
  Intervention: Drug: Placebo
• Experimental: HQK-1001
  HQK-1001
  Intervention: Drug: HQK-1001

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: May 17, 2011): 21
• Original Estimated Enrollment (submitted: November 12, 2008): 56
• Actual Study Completion Date: May 2010
• Actual Primary Completion Date: May 2010 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 60 Years (Child, Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Lebanon, Thailand
• Removed Location Countries: United States

Administrative Information

• NCT Number: NCT00790127
• Other Study ID Numbers: HQP-2008-003b
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Susan Perrine, M.D. Chief Scientific Officer, VP Clinical Affairs, HemaQuest Pharmaceuticals Inc.
• Original Responsible Party: Same as current
• Current Study Sponsor: HemaQuest Pharmaceuticals Inc.
• Original Study Sponsor: Same as current
• Collaborators: Not Provided

Investigators

• Principal Investigator: Noppadol Siritanaratkul, MD; Siriraj Hospital

• PRS Account: HemaQuest Pharmaceuticals Inc.
• Verification Date: May 2011