Ignite Creation Date:
2025-12-25 @ 5:08 AM
Ignite Modification Date:
2025-12-26 @ 4:12 AM
Study NCT ID:
NCT00790127
Status:
COMPLETED
Last Update Posted:
2011-05-20
First Post:
2008-11-11
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['United States']}, 'conditionBrowseModule': {'meshes': [{'id': 'D017086', 'term': 'beta-Thalassemia'}], 'ancestors': [{'id': 'D013789', 'term': 'Thalassemia'}, {'id': 'D000745', 'term': 'Anemia, Hemolytic, Congenital'}, {'id': 'D000743', 'term': 'Anemia, Hemolytic'}, {'id': 'D000740', 'term': 'Anemia'}, {'id': 'D006402', 'term': 'Hematologic Diseases'}, {'id': 'D006425', 'term': 'Hemic and Lymphatic Diseases'}, {'id': 'D006453', 'term': 'Hemoglobinopathies'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C074677', 'term': '2,2-dimethylbutyric acid'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 21}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2009-03'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2011-05', 'dispFirstSubmitDate': '2011-05-13', 'completionDateStruct': {'date': '2010-05', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2011-05-18', 'studyFirstSubmitDate': '2008-11-11', 'dispFirstSubmitQcDate': '2011-05-13', 'studyFirstSubmitQcDate': '2008-11-12', 'dispFirstPostDateStruct': {'date': '2011-05-18', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2011-05-20', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2008-11-13', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2010-05', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety as assessed by (1) adverse events, (2) laboratory values (3) vital signs (4) physical exam', 'timeFrame': '168 days'}], 'secondaryOutcomes': [{'measure': 'Pharmacokinetics assessed by plasma drug concentration levels', 'timeFrame': '140 days'}, {'measure': 'Pharmacodynamics assessed by red blood cell production and induction of fetal hemoglobin', 'timeFrame': '168 days'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Beta Thalassemia']}, 'referencesModule': {'references': [{'pmid': '36637054', 'type': 'DERIVED', 'citation': '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.'}]}, 'descriptionModule': {'briefSummary': '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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Clinical diagnosis of beta thalassemia intermedia or hemoglobin E beta thalassemia\n* Average of the initial two hemoglobin levels of less than 10 g/dL within ≥ 30 days prior to randomization.\n* Age ≥ 12 and ≤ 60 years\n* Able and willing to give informed consent or assent (if less than 18 years of age) and comply with all study procedures\n* 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\n\nExclusion Criteria:\n\n* Spleen palpable ≥ 2 cm below the left costal margin\n* Pulmonary hypertension requiring oxygen therapy\n* QTc \\> 450 msec on screening ECG\n* Infection with hepatitis C, hepatitis B requiring therapy\n* Known infection with HIV\n* Red blood cell (RBC) transfusions within 2 months prior to administration of study medication with stable hemoglobin levels\n* Fever greater than 38.5°C in the week prior to administration of study medication\n* ALT \\> 4x upper limit of normal (ULN)\n* Baseline elevation of CPK value prior to randomization\n* Treatment with hydroxyurea within 2 months prior to administration of study medication\n* Cancer diagnosis within 5 years of randomization (except for non-melanoma cutaneous malignancies)\n* Serum creatinine \\> 1.5 mg/dl\n* Received investigational systemic therapy within 30 days prior to randomization\n* Currently pregnant or breast feeding a child\n* Subject history of clinically significant arrhythmias or syncope\n* Known current drug or alcohol abuse'}, 'identificationModule': {'nctId': 'NCT00790127', 'briefTitle': 'Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia', 'organization': {'class': 'INDUSTRY', 'fullName': 'HemaQuest Pharmaceuticals Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'HQP-2008-003b'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo', 'interventionNames': ['Drug: Placebo']}, {'type': 'EXPERIMENTAL', 'label': 'HQK-1001', 'description': 'HQK-1001', 'interventionNames': ['Drug: HQK-1001']}], 'interventions': [{'name': 'HQK-1001', 'type': 'DRUG', 'description': 'HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days', 'armGroupLabels': ['HQK-1001']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Matching placebo capsules administered once a day, orally, for 56 days', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Beirut', 'country': 'Lebanon', 'facility': 'Chronic Care Center', 'geoPoint': {'lat': 33.89332, 'lon': 35.50157}}, {'zip': '10700', 'city': 'Bangkok', 'state': 'Bangkoknoin District', 'country': 'Thailand', 'facility': 'Siriraj Hospital', 'geoPoint': {'lat': 13.75398, 'lon': 100.50144}}], 'overallOfficials': [{'name': 'Noppadol Siritanaratkul, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Siriraj Hospital'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'HemaQuest Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'oldNameTitle': 'Susan Perrine, M.D. Chief Scientific Officer, VP Clinical Affairs', 'oldOrganization': 'HemaQuest Pharmaceuticals Inc.'}}}}