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

NCT ID: NCT00790127

Last Updated: 2011-05-20

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 21 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-03-31
• Study Completion Date: 2010-05-31

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

Conditions

Beta Thalassemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

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

HQK-1001

HQK-1001

Group Type: EXPERIMENTAL

HQK-1001

Intervention Type: DRUG

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

Interventions

HQK-1001

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

Intervention Type: DRUG

Placebo

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

Intervention Type: DRUG

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

• Minimum Eligible Age: 12 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

HemaQuest Pharmaceuticals Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

HemaQuest Pharmaceuticals Inc.

Principal Investigators

Noppadol Siritanaratkul, MD

Role: PRINCIPAL_INVESTIGATOR

Siriraj Hospital

Locations

Chronic Care Center

Beirut, , Lebanon

Siriraj Hospital

Bangkok, Bangkoknoin District, Thailand

Countries

Lebanon; Thailand

References

Foong WC, Loh CK, Ho JJ, Lau DS. Foetal haemoglobin inducers for reducing blood transfusion in non-transfusion-dependent beta-thalassaemias. Cochrane Database Syst Rev. 2023 Jan 13;1(1):CD013767. doi: 10.1002/14651858.CD013767.pub2.

Reference Type: DERIVED

PMID: 36637054 (View on PubMed)

Other Identifiers

HQP-2008-003b

Identifier Type: -

Identifier Source: org_study_id