Ignite Creation Date:
2025-12-25 @ 4:02 AM
Ignite Modification Date:
2026-04-09 @ 1:46 PM
Study NCT ID:
NCT02525302
Status:
TERMINATED
Last Update Posted:
2019-03-12
First Post:
2015-07-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C010176', 'term': 'halofuginone'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'whyStopped': 'Dosing stopped', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2015-05'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-03', 'completionDateStruct': {'date': '2016-12-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-03-08', 'studyFirstSubmitDate': '2015-07-18', 'studyFirstSubmitQcDate': '2015-08-14', 'lastUpdatePostDateStruct': {'date': '2019-03-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-08-17', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-12-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Pharmacokinetics peak plasma concentration (Cmax)', 'timeFrame': 'Pre-dose and 2-4 hour post-dose'}], 'primaryOutcomes': [{'measure': 'Number of adverse events by severity and relationship', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Dose reduction or modification due to upper GI or other adverse events', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Trial discontinuations due to upper GI or other AEs', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Vital signs (Number of subjects with clinically significant changes)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes'}, {'measure': 'Laboratory values (Number of subjects with clinically significant changes)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes.'}, {'measure': 'Electrocardiograms', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes in QT interval'}, {'measure': 'Echocardiograms', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes in left ventricular ejection fraction, end systolic and diastolic interventricular septal thickness, left ventricular posterior wall thickness'}, {'measure': 'Cardiovascular Magnetic Resonance', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant change in diagnostic interpretation'}], 'secondaryOutcomes': [{'measure': 'Cardiovascular Magnetic Resonance', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Circumferential strain and myocardial fibrotic areas'}, {'measure': 'Pulmonary function testing (Number of subjects with clinically significant changes)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes.'}, {'measure': 'Motor function measure (MFM) scale', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Performance of upper limb (PUL) scale', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Biomarkers of extracellular matrix turnover (Number of subjects with clinically significant changes)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes.'}, {'measure': 'Quantitative muscle testing (QMT) scores', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Timed function tests (TFTs)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Motor Function Measure (MFM)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': 'Upper extremity function (proximal, mid-range, and distal) by Performance of Upper Limb (PUL)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}, {'measure': '9-hole peg test', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Assessment of upper limb function and dexterity'}, {'measure': 'Tip pinch and key pinch tests (Number of subjects with clinically significant changes)', 'timeFrame': 'Every 6 months from enrollment for up to 3 years', 'description': 'Number of subjects with clinically significant changes.'}, {'measure': 'Electrical impedance myography (EIM) score', 'timeFrame': 'Every 6 months from enrollment for up to 3 years'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['DMD', 'neuromuscular', 'Duchenne'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.akashirx.com/', 'label': 'Sponsor company website'}]}, 'descriptionModule': {'briefSummary': 'This study, HALO-DMD-03, is a follow-on study to HALO-DMD-01 and HALO-DMD-02, and allows continued open-label access to HT-100 for subjects who have completed these studies. HALO-DMD-03 will provide safety and strength and function data on continuous long-term dosing. Data from this study will be used to inform the safety, tolerability, and dose selection for a future trial of HT-100 in boys with Duchenne Muscular Dystrophy (DMD).', 'detailedDescription': "As a follow-on study to the initial clinical studies of HT-100 in DMD (Protocols HALO-DMD-01 and HALO-DMD-02), this open-label study is designed to provide data on continuous long-term dosing. Subjects will be entered into the study without cessation of dosing, in a staggered fashion, into the same cohort assignment they had in the predecessor studies. Up to 30 subjects who have completed dosing in HALO-DMD-02 will be offered the opportunity to continue on the same dose regimen until market approval of HT-100 or termination of the study by the Sponsor. Reasons for termination could include, among others, safety concerns or lack of efficacy, based on analysis of combined data from all HT-100 studies. Safety data from subjects approaching the end the HALO-DMD-02 participation will be individually reviewed by the Medical Monitor and the subject's physician (Principal Investigator \\[PI\\]). If the Medical Monitor and the PI agree there are no clinically significant safety signals (absence of clinically significant laboratory or clinical abnormalities to date), the subject will be considered eligible and offered continuation of dosing. To avoid an interruption in dosing, subjects will immediately be screened for participation and enrolled upon completing the predecessor trial, HALO-DMD-02. Participation is in this study HALO-DMD-03 is optional. Safety and pharmacodynamics (PD) monitoring will continue throughout the subject's study participation. Dose reduction/modification might occur or individual subjects' participation in the trial may be discontinued if any Adverse Events (AEs) suggest that HT-100 is not sufficiently well tolerated."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Completed both previous studies HALO-DMD-01 and HALO-DMD-02\n2. Ability to provide written informed consent\n3. Ability to understand and follow site and protocol instruction for the entire duration of the study\n\nExclusion Criteria:\n\nAnswering yes to any of the following make the subject NOT eligible to participate in the study.\n\n1. Clinically significant major disease not related to DMD that would make it not safe to be in the study or affect ability to follow the protocol\n2. History of severe allergic or anaphylactic reactions\n3. Recent report of drug/alcohol abuse'}, 'identificationModule': {'nctId': 'NCT02525302', 'briefTitle': 'HT-100 Long-term Study in DMD Patients Who Completed HALO-DMD-02', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akashi Therapeutics'}, 'officialTitle': 'HT-100 Long-term Safety and Pharmacodynamics in Patients With DMD Who Have Completed Protocols HALO-DMD-01 and HALO-DMD-02', 'orgStudyIdInfo': {'id': 'HALO-DMD-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 1', 'description': 'HT-100 multiple dose administration (dose 1).', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 2', 'description': 'HT-100 multiple dose administration (dose 1).', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 3', 'description': 'HT-100 multiple dose administration (dose 1).', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 4', 'description': 'HT-100 multiple dose administration (dose 1).', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 5', 'description': 'HT-100 multiple dose administration (dose 1).', 'interventionNames': ['Drug: HT-100']}], 'interventions': [{'name': 'HT-100', 'type': 'DRUG', 'otherNames': ['halofuginone hydrobromide'], 'description': "HT-100 is Akashi Therapeutics' proprietary delayed-release formulation of halofuginone hydrobromide, a small molecule therapeutic with anti-fibrotic properties. May be administered in either fed or fasted state. Not mutation specific.", 'armGroupLabels': ['Cohort 1: HT-100 tablet, Dose 1', 'Cohort 1: HT-100 tablet, Dose 2', 'Cohort 1: HT-100 tablet, Dose 3', 'Cohort 1: HT-100 tablet, Dose 4', 'Cohort 1: HT-100 tablet, Dose 5']}]}, 'contactsLocationsModule': {'locations': [{'zip': '95817', 'city': 'Sacramento', 'state': 'California', 'country': 'United States', 'facility': 'University of California, Davis Medical Center', 'geoPoint': {'lat': 38.58157, 'lon': -121.4944}}, {'zip': '21205', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Kennedy Krieger Institute, Johns Hopkins School of Medicine', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'country': 'United States', 'facility': 'Washington University School of Medicine', 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'country': 'United States', 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'country': 'United States', 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}], 'overallOfficials': [{'name': 'Diana M Escolar, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Askashi Therapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akashi Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}