Ignite Creation Date:
2025-12-24 @ 10:52 PM
Ignite Modification Date:
2026-02-17 @ 11:00 PM
Study NCT ID:
NCT02329769
Status:
TERMINATED
Last Update Posted:
2017-12-08
First Post:
2014-12-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (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': 15}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2014-12', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-12', 'completionDateStruct': {'date': '2016-08-31', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-12-06', 'studyFirstSubmitDate': '2014-12-22', 'studyFirstSubmitQcDate': '2014-12-31', 'lastUpdatePostDateStruct': {'date': '2017-12-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2015-01-01', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2016-07-01', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Efficacy of PRO044 (composite of several measures)', 'timeFrame': 'After 48 weeks of treatment', 'description': 'Efficacy parameters:\n\nMuscle Function\n\n* 6 Minute Walk Distance (6MWD)\n* North Star Ambulatory Assessment\n* Timed tests (10-meter walk/run, rising from floor, stair climb)\n* DMD Functional Outcomes Questionnaire (DMD-FOS) -for ambulant subjects only\n* Egen Klassification - for non-ambulant subjects.\n\nMuscle strength\n\n* Pulmonary Function (Spirometry)\n* Handheld myometry.\n\nExploratory:\n\n* Performance Upper Limb (PUL).\n* Patient Reported Outcome measure (PROM).'}, {'measure': 'Safety and tolerability of PRO044 (treatement emergent adverse events)', 'timeFrame': 'After 48 weeks of treatment', 'description': 'Number of subjects with 1 or more treatement emergent adverse events following SC or IV PRO044 dosing'}], 'secondaryOutcomes': [{'measure': 'Assess the pharmacokinetics of PRO044 (composite of several measures)', 'timeFrame': 'After 48 weeks of treatment', 'description': 'Pharmacokinetic parameters:\n\n* t ½\n* AUC: 0-24h, 0-∞ (where applicable)\n* Cmax\n* tmax\n* CL (for IV subjects) or CL/F (for SC subjects)\n* PRO044 concentrations in muscle tissue.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['Duchenne muscular dystrophy', 'DMD', 'BioMarin', 'PRO044'], 'conditions': ['Duchenne Muscular Dystrophy']}, 'descriptionModule': {'briefSummary': 'The purpose of this study is to see whether PRO044 is safe and effective to use as medication for Duchenne Muscular Dystrophy (DMD) patients with a mutation around location 44 in the DNA for the dystrophin protein.', 'detailedDescription': 'A Phase II, open-label, extesion study. Following a Screening period of up to one month, subjects previously treated with PRO044, and eligible for enrolment in PRO044-CLIN-02, will be allocated to one of three groups to receive either 6 mg/kg or 9 mg/kg PRO044 weekly by IV infusion or 6 mg/kg weekly by SC injection for 48 weeks.\n\nSafety and tolerability, pharmacokinetics (PK), pharmacodynamic (PD) and efficacy assessments will be conducted at regular intervals throughout the study.'}, 'eligibilityModule': {'sex': 'MALE', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '20 Years', 'minimumAge': '9 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Subjects previously treated with PRO044.\n2. Continued use of glucocorticoids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain on steroids for the duration of the study. Changes to the dose regimen or cessation of glucocorticoids will be at the discretion of the Principle Investigator (PI) in consultation with the subject/parent and the Medical Monitor. If the subject is not on steroids, involvement in the study needs to be discussed with the medical monitor\n\nExclusion Criteria:\n\n1. Current, or history of, liver or renal disease.\n2. Acute illness within 4 weeks prior to the first dose of PRO044 (Week 1) which may interfere with the measurements.\n3. Severe cardiac myopathy which in the opinion of the Investigator prohibits participation in this study\n4. Need for daytime mechanical ventilation.\n5. Screening aPTT above the upper limit of normal (ULN).\n6. Screening platelet count below the lower limit of normal (LLN).\n7. Use of anticoagulants, antithrombotics or antiplatelet agents.\n8. Use of any investigational product within 6 months prior to the start of Screening for the study.\n9. Current or history of drug and/or alcohol abuse.'}, 'identificationModule': {'nctId': 'NCT02329769', 'briefTitle': 'Open Label, Extension Study of PRO044 in Duchenne Muscular Dystrophy (DMD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'BioMarin Pharmaceutical'}, 'officialTitle': 'A Phase II, Open Label, Extension Study to Assess the Effect of PRO044 in Patients With Duchenne Muscular Dystrophy', 'orgStudyIdInfo': {'id': 'PRO044-CLIN-02'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PRO044 SC 6 mg/kg', 'description': 'Weekly subcutaneous (SC) dosing with 6 mg/kg', 'interventionNames': ['Drug: PRO044 SC 6 mg/kg']}, {'type': 'EXPERIMENTAL', 'label': 'PRO044 IV 6 mg/kg', 'description': 'Weekly intravenous (IV) dosing with 6 mg/kg', 'interventionNames': ['Drug: PRO044 IV 6 mg/kg']}, {'type': 'EXPERIMENTAL', 'label': 'PRO044 SC 9 mg/kg', 'description': 'Weekly intravenous (IV) dosing with 9 mg/kg', 'interventionNames': ['Drug: PRO044 IV 9 mg/kg']}], 'interventions': [{'name': 'PRO044 SC 6 mg/kg', 'type': 'DRUG', 'armGroupLabels': ['PRO044 SC 6 mg/kg']}, {'name': 'PRO044 IV 6 mg/kg', 'type': 'DRUG', 'armGroupLabels': ['PRO044 IV 6 mg/kg']}, {'name': 'PRO044 IV 9 mg/kg', 'type': 'DRUG', 'armGroupLabels': ['PRO044 SC 9 mg/kg']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Leuven', 'country': 'Belgium', 'facility': 'UZ Leuven', 'geoPoint': {'lat': 50.87959, 'lon': 4.70093}}, {'city': 'Ferrara', 'country': 'Italy', 'facility': 'S.Anna Hospital', 'geoPoint': {'lat': 44.83804, 'lon': 11.62057}}, {'city': 'Roma', 'country': 'Italy', 'facility': 'Policlinico Universitario Agostino Gemelli', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'city': 'Leiden', 'country': 'Netherlands', 'facility': 'Leids Universitair Medisch Centrum', 'geoPoint': {'lat': 52.15833, 'lon': 4.49306}}, {'city': 'Gothenburg', 'country': 'Sweden', 'facility': 'Drottning Silvias Barn- ochungdomssjukhus', 'geoPoint': {'lat': 57.70716, 'lon': 11.96679}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'BioMarin Pharmaceutical', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}