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

Sponsor

HemaQuest Pharmaceuticals Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT00790127

Collaborator

(none)

Enrollment

Locations

Arms

Duration (Months)

10.5

Patients Per Site

0.8

Patients Per Site Per Month

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Beta Thalassemia	Drug: HQK-1001 Drug: Placebo	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

21 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

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

Study Start Date :

Mar 1, 2009

Actual Primary Completion Date :

May 1, 2010

Actual Study Completion Date :

May 1, 2010

Arms and Interventions

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

Arm

Intervention/Treatment

Placebo Comparator: Placebo

Placebo

Drug: Placebo

Matching placebo capsules administered once a day, orally, for 56 days

Experimental: HQK-1001

HQK-1001

Drug: HQK-1001

HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days

Outcome Measures

Primary Outcome Measures

Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam [168 days]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Chronic Care Center	Beirut		Lebanon
2	Siriraj Hospital	Bangkok	Bangkoknoin District	Thailand	10700

Sponsors and Collaborators

HemaQuest Pharmaceuticals Inc.

Investigators

Principal Investigator: Noppadol Siritanaratkul, MD, Siriraj Hospital

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

, ,

ClinicalTrials.gov Identifier:

NCT00790127

Other Study ID Numbers:

HQP-2008-003b

First Posted:

Nov 13, 2008

Last Update Posted:

May 20, 2011

Last Verified:

May 1, 2011

Additional relevant MeSH terms:

Thalassemia

beta-Thalassemia

Study Results

No Results Posted as of May 20, 2011