Ignite Creation Date:
2025-12-24 @ 10:48 PM
Ignite Modification Date:
2025-12-25 @ 8:18 PM
Study NCT ID:
NCT01322269
Status:
COMPLETED
Last Update Posted:
2013-06-19
First Post:
2011-03-22
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
A Study of HQK-1001 in Patients With Sickle Cell Disease
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000755', 'term': 'Anemia, Sickle Cell'}], 'ancestors': [{'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': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 52}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2011-04'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2013-06', 'dispFirstSubmitDate': '2013-06-11', 'lastUpdateSubmitDate': '2013-06-11', 'studyFirstSubmitDate': '2011-03-22', 'dispFirstSubmitQcDate': '2013-06-11', 'studyFirstSubmitQcDate': '2011-03-23', 'dispFirstPostDateStruct': {'date': '2013-06-19', 'type': 'ESTIMATED'}, 'lastUpdatePostDateStruct': {'date': '2013-06-19', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-03-24', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2012-03', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety', 'timeFrame': 'Day 1 through Week 30', 'description': 'Physical exams, vital signs, clinical laboratory safety assessments, ECG and adverse event monitoring.'}], 'secondaryOutcomes': [{'measure': 'Fetal hemoglobin levels', 'timeFrame': 'Day 1 and Weeks 4, 8, 12, 16, 20, 25, 26 and 30'}, {'measure': 'Incidence of sickle cell crisis events', 'timeFrame': 'Day 1 through Week 30'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'conditions': ['Sickle Cell Disease', 'Sickle Cell Anemia', 'Sickle Cell Disorders', 'Hemoglobin S Disease', 'Sickling Disorder Due to Hemoglobin S']}, 'referencesModule': {'references': [{'pmid': '23828223', 'type': 'DERIVED', 'citation': 'Kutlar A, Reid ME, Inati A, Taher AT, Abboud MR, El-Beshlawy A, Buchanan GR, Smith H, Ataga KI, Perrine SP, Ghalie RG. A dose-escalation phase IIa study of 2,2-dimethylbutyrate (HQK-1001), an oral fetal globin inducer, in sickle cell disease. Am J Hematol. 2013 Nov;88(11):E255-60. doi: 10.1002/ajh.23533. Epub 2013 Oct 3.'}]}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to evaluate the safety and tolerability of three dose levels of HQK-1001 administered once daily for 26 weeks in subjects with sickle cell disease.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '60 Years', 'minimumAge': '12 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Established diagnosis of SCD\n* Males and females between 12 and 60 years of age, inclusive\n* At least 3 episodes of a SCD-related crisis or complication in the 3 years prior to screening OR 1 episode of acute chest syndrome in the 5 years prior to screening\n* If receiving hydroxyurea, must be receiving a stable dose for at least 6 months prior to screening\n* If hydroxyurea treatment has been discontinued, at least 3 months have elapsed since last dose\n* If transfusion in the 4 months prior to screening, then HbA level \\< 20% at screening\n* Average of the initial two HbF levels ≥ 2.0 % within ≤ 7 days prior to the initial dose of HQK-1001. The two must be obtained ≥ 24 hours apart\n* Ability to swallow tablets\n* Able and willing to give informed consent and assent (if applicable)\n* If subject is a woman of child-bearing potential (WCBP), she must have a negative serum pregnancy test within 7 days of first dose of HQK-1001\n* If a subject is a WCBP, she must agree to use an effective form of contraception within 7 days of the initial dose of HQK-1001 and for one month after HQK-1001 discontinuation\n* Sexually active male subjects (with WCBP partners) must agree to use latex condoms or ensure that their partner(s) use an effective form of contraception\n* In the view of the Investigator, subject is able and willing to comply with necessary study procedures\n\nExclusion Criteria:\n\n* More than 4 hospitalizations for acute sickle cell related events in the previous 12 months prior to screening\n* Pulmonary hypertension requiring oxygen therapy\n* QTc \\> 450 msec (male) or 470 msec (female) on screening ECG (QT corrected by Fridericia's formula)\n* Assigned to a regular transfusion program\n* Use of erythropoiesis stimulating agents within 90 days of screening\n* ALT \\> 3x upper limit of normal (ULN)\n* Serum creatinine \\> 1.2 mg/dL\n* A serious, concurrent illness that would limit ability to complete or comply with the study requirements\n* An acute vaso-occlusive event within 3 weeks prior to screening\n* Creatine phosphokinase (CK) \\> 20% above the ULN\n* An acute illness (e.g., febrile, GI, respiratory) within 72 hours prior to screening\n* History of syncope, clinically significant dysrhythmias or resuscitation from sudden death\n* Chronic opiate use, which, in the view of the Investigator, could confound evaluation of an investigational drug\n* Current abuse of alcohol or drugs\n* Received another investigational agent within 4 weeks or 5 half-lives, whichever is longer, prior to screening\n* Currently pregnant or breast feeding a child\n* Known infection with HIV-1\n* Infection with hepatitis B or hepatitis C, such that patients are currently on therapy or will be placed on therapy during the trial"}, 'identificationModule': {'nctId': 'NCT01322269', 'briefTitle': 'A Study of HQK-1001 in Patients With Sickle Cell Disease', 'organization': {'class': 'INDUSTRY', 'fullName': 'HemaQuest Pharmaceuticals Inc.'}, 'officialTitle': 'A Randomized, Open-Label, Multi-Dose Study of HQK-1001 in Subjects With Sickle Cell Disease', 'orgStudyIdInfo': {'id': 'HQP 1001-SCD-006'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HQK-1001 (30 mg/kg)', 'interventionNames': ['Drug: HQK-1001']}, {'type': 'EXPERIMENTAL', 'label': 'HQK-1001 (40 mg/kg)', 'interventionNames': ['Drug: HQK-1001']}, {'type': 'EXPERIMENTAL', 'label': 'HQK-1001 (50 mg/kg)', 'interventionNames': ['Drug: HQK-1001']}], 'interventions': [{'name': 'HQK-1001', 'type': 'DRUG', 'description': 'HQK-1001 tablets, once daily for daily 26 weeks', 'armGroupLabels': ['HQK-1001 (30 mg/kg)', 'HQK-1001 (40 mg/kg)', 'HQK-1001 (50 mg/kg)']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "Children's Hospital and Research Center - Oakland", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '33101', 'city': 'Miami', 'state': 'Florida', 'country': 'United States', 'facility': 'University of Miami Miller School of Medicine - Dept of Pediatrics', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '30912', 'city': 'Augusta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Georgia Health Sciences University - Adult SIckle Cell Center', 'geoPoint': {'lat': 33.47097, 'lon': -81.97484}}, {'zip': '60612', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': 'University of Illinois at Chicago - Dept of Pediatrics', 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '71103', 'city': 'Shreveport', 'state': 'Louisiana', 'country': 'United States', 'facility': 'LSU Health Sciences Center - Feist Weiller Cancer Center', 'geoPoint': {'lat': 32.52515, 'lon': -93.75018}}, {'zip': '02111', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Tufts Medical Center', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '27599', 'city': 'Chapel Hill', 'state': 'North Carolina', 'country': 'United States', 'facility': 'University of North Carolina at Chapel Hill - Comprehensive Sickle Cell Program', 'geoPoint': {'lat': 35.9132, 'lon': -79.05584}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '75390-9063', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Univerisity of Texas Southwestern Medical Center at Dallas - Pediatric Hematology Oncology', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': 'MSG 1X8', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'The Hospital for Sick Children', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': 'MSG 2C4', 'city': 'Toronto', 'state': 'Ontario', 'country': 'Canada', 'facility': 'University Health Network Toronto General Hospital', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'city': 'Cairo', 'country': 'Egypt', 'facility': 'Abu El Reesh Pediatric University Hospital', 'geoPoint': {'lat': 30.06263, 'lon': 31.24967}}, {'city': 'Mona', 'state': 'Kingston', 'country': 'Jamaica', 'facility': 'University of the West Indies - Sickle Cell Unit'}, {'city': 'Beirut', 'country': 'Lebanon', 'facility': 'American University of Beirut Medical Center', 'geoPoint': {'lat': 33.89332, 'lon': 35.50157}}, {'city': 'Beirut', 'country': 'Lebanon', 'facility': 'Rafik Hariri University Hospital', 'geoPoint': {'lat': 33.89332, 'lon': 35.50157}}, {'city': 'Hazmiyeh', 'country': 'Lebanon', 'facility': 'Chronic Care Center', 'geoPoint': {'lat': 33.85534, 'lon': 35.53589}}], 'overallOfficials': [{'name': 'Richard Ghalie, MD, MBA', 'role': 'STUDY_DIRECTOR', 'affiliation': 'HemaQuest Pharmaceuticals Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'HemaQuest Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}