Ignite Creation Date:
2025-12-25 @ 2:37 AM
Ignite Modification Date:
2026-03-02 @ 8:18 AM
Study NCT ID:
NCT03614234
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-10-08
First Post:
2018-07-30
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24', 'removedCountries': ['Spain']}, 'conditionBrowseModule': {'meshes': [{'id': 'D000795', 'term': 'Fabry Disease'}, {'id': 'D011507', 'term': 'Proteinuria'}], 'ancestors': [{'id': 'D013106', 'term': 'Sphingolipidoses'}, {'id': 'D020140', 'term': 'Lysosomal Storage Diseases, Nervous System'}, {'id': 'D020739', 'term': 'Brain Diseases, Metabolic, Inborn'}, {'id': 'D001928', 'term': 'Brain Diseases, Metabolic'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D059345', 'term': 'Cerebral Small Vessel Diseases'}, {'id': 'D002561', 'term': 'Cerebrovascular Disorders'}, {'id': 'D014652', 'term': 'Vascular Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008064', 'term': 'Lipidoses'}, {'id': 'D008052', 'term': 'Lipid Metabolism, Inborn Errors'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D052439', 'term': 'Lipid Metabolism Disorders'}, {'id': 'D014555', 'term': 'Urination Disorders'}, {'id': 'D014570', 'term': 'Urologic Diseases'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D020924', 'term': 'Urological Manifestations'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'Open-label extension study'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 29}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2018-11-13', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2026-03-28', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-07', 'studyFirstSubmitDate': '2018-07-30', 'studyFirstSubmitQcDate': '2018-08-02', 'lastUpdatePostDateStruct': {'date': '2024-10-08', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-08-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-12-31', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluation of treatment-related adverse events', 'timeFrame': 'Throughout the study, 364 weeks', 'description': 'CTCAE v4.03'}], 'secondaryOutcomes': [{'measure': 'Kidney function 1', 'timeFrame': 'Every 6 months throughout the duration of the study, 364 weeks', 'description': 'Estimated glomerular filtration rate (eGFRCKD-EPI)'}, {'measure': 'Cardiac assessment', 'timeFrame': 'Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364', 'description': 'Left Ventricular Mass Index (g/m2) by echocardiogram and cardiac function stress test'}, {'measure': 'Biomarkers for Fabry disease', 'timeFrame': 'Every 6 months throughout the duration of the study, 364 weeks', 'description': 'Plasma Lyso-Gb3 and Gb3'}, {'measure': 'Kidney function 2', 'timeFrame': 'Every 6 months throughout the duration of the study, 364 weeks', 'description': 'Protein/Creatinine ratio (UPCR), spot urine test'}, {'measure': 'Clinical assessment', 'timeFrame': 'Every four weeks throughout the duration of the study, 364 weeks', 'description': 'Record of pain medication and pre-medication use'}, {'measure': 'Pain assessment', 'timeFrame': 'Every 6 months throughout the duration of the study, 364 weeks', 'description': 'Short form Brief Pain Inventory (BPI)'}, {'measure': 'Symptom assessment', 'timeFrame': 'Once a year throughout the study at weeks 52, 104, 152, 200, 256, 312 and end of study week 364', 'description': 'Mainz Severity Score Index (MSSI)'}, {'measure': 'Quality of life assessment', 'timeFrame': 'Every 6 months throughout the duration of the study, 364 weeks', 'description': 'Quality of life (EQ-5D-5L)'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Glomerular filtration rate', 'Proteinuria', 'PRX-102', 'pegunigalsidase alfa', 'Fabry disease'], 'conditions': ['Fabry Disease']}, 'descriptionModule': {'briefSummary': 'The objective of CLI-06657AA1-03 (formerly PB-102-F51) is to evaluate the long-term safety, tolerability, and efficacy of 2 mg/kg pegunigalsidase alfa administered intravenously every four weeks in adult Fabry patients who have successfully completed PB-102-F50.', 'detailedDescription': 'This is an open-label study to assess the long-term safety and efficacy of pegunigalsidase alfa treatment of 2.0 mg/kg administered intravenously every 4 weeks. The duration of treatment will be until pegunigalsidase alfa is commercially available to the patient, or at the discretion of the Sponsor.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n1. Completion of study PB-102-F50.\n2. The patient signs informed consent.\n3. Female patients and male patients whose co-partners are of child-bearing potential agree to use a medically accepted, effective contraception method. These include combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal) supplemented with a barrier method (preferably male condom), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable) supplemented with a barrier method (preferably male condom), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomised partner, or sexual abstinence. Contraception should be used for 2 weeks after treatment termination.\n\nExclusion Criteria:\n\nPresence of any medical, emotional, behavioral, or psychological condition that, in the judgment of the Investigator, would interfere with patient compliance with the requirements of the study.'}, 'identificationModule': {'nctId': 'NCT03614234', 'briefTitle': 'Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients', 'organization': {'class': 'INDUSTRY', 'fullName': 'Chiesi Farmaceutici S.p.A.'}, 'officialTitle': 'Open Label Extension Study to Evaluate the Long-term Safety and Efficacy of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Administered by Intravenous Infusion Every 4 Weeks in Adult Patients With Fabry Disease', 'orgStudyIdInfo': {'id': 'CLI-06657AA1-03'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Experimental open label', 'description': 'Pegunigalsidase alfa', 'interventionNames': ['Drug: pegunigalsidase alfa']}], 'interventions': [{'name': 'pegunigalsidase alfa', 'type': 'DRUG', 'otherNames': ['PRX-102'], 'description': 'Recombinant human alpha galactosidase A', 'armGroupLabels': ['Experimental open label']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'UAB Medicine', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '30307', 'city': 'Atlanta', 'state': 'Georgia', 'country': 'United States', 'facility': 'Emory University School of Medicine', 'geoPoint': {'lat': 33.749, 'lon': -84.38798}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa Hospitals and Clinica', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '49525', 'city': 'Grand Rapids', 'state': 'Michigan', 'country': 'United States', 'facility': 'Infusion Associates', 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '75235', 'city': 'Dallas', 'state': 'Texas', 'country': 'United States', 'facility': 'Renal Disease Research Institute, LLC', 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '84112', 'city': 'Salt Lake City', 'state': 'Utah', 'country': 'United States', 'facility': 'University of Utah Hospitals & Clinics', 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '22030', 'city': 'Fairfax', 'state': 'Virginia', 'country': 'United States', 'facility': 'O & O Alpan', 'geoPoint': {'lat': 38.84622, 'lon': -77.30637}}, {'zip': '2650', 'city': 'Edegem', 'country': 'Belgium', 'facility': 'UZ Antwerpen', 'geoPoint': {'lat': 51.15662, 'lon': 4.44504}}, {'zip': '128 08', 'city': 'Prague', 'country': 'Czechia', 'facility': 'Fakultní poliklinika Všeobecné fakultní nemocnice v Praze', 'geoPoint': {'lat': 50.08804, 'lon': 14.42076}}, {'zip': '2100', 'city': 'Copenhagen', 'country': 'Denmark', 'facility': 'Medical Endocrinology PE 2132, Rigshospitalet', 'geoPoint': {'lat': 55.67594, 'lon': 12.56553}}, {'zip': '80131', 'city': 'Napoli', 'state': 'Via Pansini', 'country': 'Italy', 'facility': 'Azienda Ospedaliera Universitaria "Federico II"', 'geoPoint': {'lat': 40.87618, 'lon': 14.5195}}, {'zip': '5021', 'city': 'Bergen', 'country': 'Norway', 'facility': 'Helse Bergen HF Haukeland Universitetssykehus', 'geoPoint': {'lat': 60.39299, 'lon': 5.32415}}, {'zip': 'CB2 0QQ', 'city': 'Cambridge', 'country': 'United Kingdom', 'facility': "Addenbrooke's Hospital", 'geoPoint': {'lat': 52.2, 'lon': 0.11667}}, {'city': 'London', 'country': 'United Kingdom', 'facility': 'The Royal Free Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Chiesi Farmaceutici S.p.A.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}