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

May 18, 2011 updated by: HemaQuest Pharmaceuticals Inc.

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

21

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Lebanon
      • Beirut, Lebanon
        • Chronic Care Center
  • Thailand
    • Bangkoknoin District
      • Bangkok, Bangkoknoin District, Thailand, 10700
        • Siriraj Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 60 years (Child, Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Placebo Comparator: Placebo
Drug: Placebo
Matching placebo capsules administered once a day, orally, for 56 days
Experimental: HQK-1001
Drug: HQK-1001
HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam
Time Frame: 168 days
168 days

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

HemaQuest Pharmaceuticals Inc.

Investigators

  • Principal Investigator: Noppadol Siritanaratkul, MD, Siriraj Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

March 1, 2009

Primary Completion (Actual)

May 1, 2010

Study Completion (Actual)

May 1, 2010

Study Registration Dates

First Submitted

November 11, 2008

First Submitted That Met QC Criteria

November 12, 2008

First Posted (Estimate)

November 13, 2008

Study Record Updates

Last Update Posted (Estimate)

May 20, 2011

Last Update Submitted That Met QC Criteria

May 18, 2011

Last Verified

May 1, 2011

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HQP-2008-003b

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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