Ignite Creation Date:
2025-12-24 @ 4:34 PM
Ignite Modification Date:
2026-01-07 @ 5:31 AM
Study NCT ID:
NCT01978366
Status:
TERMINATED
Last Update Posted:
2020-09-03
First Post:
2013-10-31
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open Label Extension Study of HT-100 in Patients With DMD
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'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 17}}, 'statusModule': {'whyStopped': 'Dosing stopped', 'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2013-10', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-07', 'dispFirstSubmitDate': '2019-07-23', 'completionDateStruct': {'date': '2016-04-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2020-08-31', 'studyFirstSubmitDate': '2013-10-31', 'dispFirstSubmitQcDate': '2019-07-23', 'studyFirstSubmitQcDate': '2013-10-31', 'dispFirstPostDateStruct': {'date': '2019-07-30', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2020-09-03', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2013-11-07', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-04-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability of administration of 6 months of chronic, oral, multiple doses of HT-100 to boys with DMD.', 'timeFrame': 'Months 2, 4, 6, 7', 'description': '* Target Safety profile by review of adverse events (AEs)\n* Physical examination findings\n* Clinical laboratory test results\n* Other diagnostic testing'}], 'secondaryOutcomes': [{'measure': 'Pharmacodynamic signals of HT-100 following chronic oral administration of multiple doses to boys with DMD.', 'timeFrame': 'Months 4, 6, 7', 'description': '* Pulmonary function\n* Motor function\n* Muscle composition\n* Biochemical and imaging markers'}, {'measure': 'Pharmacokinetic plasma profile of HT-100 following chronic oral administration of multiple doses to boys with DMD.', 'timeFrame': 'Months 4, 6', 'description': 'Halofuginone plasma concentrations'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Duchenne muscular dystrophy', 'halofuginone hydrobromide', 'anti-fibrotic', 'anti-inflammatory', 'muscle regeneration', 'protein synthesis inhibitor'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'http://www.akashirx.com/', 'label': 'Sponsor company website'}]}, 'descriptionModule': {'briefSummary': "This study is designed to provide 6-months continuous dosing with the study medication, called HT-100, on participants who successfully completed the predecessor study (HALO-DMD-01). The main purpose of this study is to assess chronic safety, tolerability, pharmacodynamic activity (testing the drug's effect on DMD) and population pharmacokinetics (measuring how much drug is in the bloodstream) in participants with a broad spectrum of Duchenne muscular dystrophy (DMD)."}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '6 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Completed both the single ascending dose (SAD) and multiple ascending dose (MAD) phases of predecessor study HALO-DMD-01\n* Maintained the same corticosteroid therapy from the predecessor study HALO-DMD-01\n* Ability to provide written informed consent\n* Ambulatory or non-ambulatory\n\nExclusion Criteria:\n\n* Recent, substantial change in use of cardiac medications or medications affecting muscle function\n* Clinically significant major disease, not related to DMD\n* Significantly compromised cardio-respiratory function\n* History of severe allergic or anaphylactic reactions\n* Prior treatment with another investigational product in past 6 months\n* Inability to undergo magnetic resonance imaging (MRI)\n* Current drug or alcohol abuse or prior treatment for abuse'}, 'identificationModule': {'nctId': 'NCT01978366', 'briefTitle': 'Open Label Extension Study of HT-100 in Patients With DMD', 'organization': {'class': 'INDUSTRY', 'fullName': 'Processa Pharmaceuticals'}, 'officialTitle': 'An Open Label Extension Study of HT-100 in Patients With Duchenne Muscular Dystrophy Who Have Completed Protocol HALO-DMD-01', 'orgStudyIdInfo': {'id': 'HALO-DMD-02'}, 'secondaryIdInfos': [{'id': 'HALO', 'type': 'OTHER', 'domain': 'Akashi Therapeutics'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1: HT-100 tablet, Dose 1', 'description': '• Multiple dose administration: Dose 1', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2: HT-100 tablet, Dose 2', 'description': '• Multiple dose administration: Dose 2', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 3: HT-100 tablet, Dose 3', 'description': '• Multiple dose administration: Dose 3', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 4: HT-100 tablet, Dose 4', 'description': '• Multiple dose administration: Dose 4', 'interventionNames': ['Drug: HT-100']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 5: HT-100 tablet, Dose 5', 'description': '• Multiple dose administration: Dose 5', 'interventionNames': ['Drug: HT-100']}], 'interventions': [{'name': 'HT-100', 'type': 'DRUG', 'otherNames': ['halofuginone hydrobromide delayed-release tablet'], 'description': 'May be administered in either fed or fasted state', 'armGroupLabels': ['Cohort 1: HT-100 tablet, Dose 1', 'Cohort 2: HT-100 tablet, Dose 2', 'Cohort 3: HT-100 tablet, Dose 3', 'Cohort 4: HT-100 tablet, Dose 4', 'Cohort 5: 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': 'AkashiTherapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Processa Pharmaceuticals', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}