Ignite Creation Date:
2025-12-25 @ 2:19 AM
Ignite Modification Date:
2025-12-26 @ 6:13 PM
Study NCT ID:
NCT00035334
Status:
COMPLETED
Last Update Posted:
2006-02-14
First Post:
2002-05-02
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009362', 'term': 'Neoplasm Metastasis'}, {'id': 'D000686', 'term': 'Amyloidosis'}, {'id': 'D001172', 'term': 'Arthritis, Rheumatoid'}, {'id': 'D009404', 'term': 'Nephrotic Syndrome'}, {'id': 'D007674', 'term': 'Kidney Diseases'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D010505', 'term': 'Familial Mediterranean Fever'}], 'ancestors': [{'id': 'D009385', 'term': 'Neoplastic Processes'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}, {'id': 'D057165', 'term': 'Proteostasis Deficiencies'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D001168', 'term': 'Arthritis'}, {'id': 'D007592', 'term': 'Joint Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D012216', 'term': 'Rheumatic Diseases'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D001327', 'term': 'Autoimmune Diseases'}, {'id': 'D007154', 'term': 'Immune System Diseases'}, {'id': 'D009401', 'term': 'Nephrosis'}, {'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': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D056660', 'term': 'Hereditary Autoinflammatory Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D004364', 'term': 'Pharmaceutical Preparations'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'DOUBLE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'count': 150}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2001-10'}, 'statusVerifiedDate': '2006-02', 'completionDateStruct': {'date': '2004-12'}, 'lastUpdateSubmitDate': '2006-02-13', 'studyFirstSubmitDate': '2002-05-02', 'studyFirstSubmitQcDate': '2002-05-02', 'lastUpdatePostDateStruct': {'date': '2006-02-14', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2002-05-03', 'type': 'ESTIMATED'}}, 'conditionsModule': {'keywords': ['Familial Mediterranean Fever', 'Amyloidosis', 'Secondary (AA) Amyloidosis', 'Nephrotic Syndrome'], 'conditions': ['Secondary (AA) Amyloidosis', 'Rheumatoid Arthritis', 'Nephrotic Syndrome', 'Familial Mediterranean Syndrome', 'Kidney Diseases', 'Gastrointestinal Diseases']}, 'referencesModule': {'references': [{'type': 'BACKGROUND', 'citation': 'Safety, Tolerability and Pharmacokinetic Profile of FibrillexTM (Anti-AA Amyloid Agent) in Healthy and Renal Impaired Subjects. Garceau D., Gurbindo C., Laurin J. Neurochem Inc. Reference: Proceedings from the IXth International Symposium on Amyloidosis , 2001 (Budapest, Hungary)'}, {'pmid': '17554116', 'type': 'DERIVED', 'citation': 'Dember LM, Hawkins PN, Hazenberg BP, Gorevic PD, Merlini G, Butrimiene I, Livneh A, Lesnyak O, Puechal X, Lachmann HJ, Obici L, Balshaw R, Garceau D, Hauck W, Skinner M; Eprodisate for AA Amyloidosis Trial Group. Eprodisate for the treatment of renal disease in AA amyloidosis. N Engl J Med. 2007 Jun 7;356(23):2349-60. doi: 10.1056/NEJMoa065644.'}]}, 'descriptionModule': {'briefSummary': 'The main objective of this study is to evaluate the safety and efficacy of NC-503 compared to placebo in patients with secondary (AA) amyloidosis using a composite assessment of clinical improvement/worsening of both renal and gastrointestinal functions.', 'detailedDescription': 'AA amyloidosis is associated with chronic inflammatory conditions (rheumatoid arthritis, ankylosing spondylitis, inflammatory bowel disease), chronic infection (tuberculosis, osteomyelitis), and Familial Mediterranean Fever. Rheumatoid arthritis is the major cause of AA amyloidosis in Western Europe and North America. The most common clinical feature of AA amyloidosis is renal dysfunction manifested as nephrotic-range proteinuria or renal insufficiency at the time of diagnosis. End-stage renal failure is the cause of death in 40-60% of cases. Gastrointestinal involvement is also frequent and is usually manifested as chronic diarrhea, body weight loss and malabsorption. Enlargement of the liver and spleen may also occur in some patients. The median survival time from diagnosis varies from 2 to 8 years depending on the stage of the disease at time of diagnosis. The goal of the current therapy in AA amyloidosis is the control of the associated disease. However, the current approaches for the treatment of AA amyloidosis are unspecific, toxic, invasive, and not sufficiently effective in many cases. NC-503 was specifically designed to compete with the naturally occurring sulfated GAGs for the binding to amyloidogenic precursor proteins, and to inhibit amyloid deposition into tissues. The proposed therapy with NC-503 is based on the prevention of the amyloid fibril formation. The objective of this clinical phase II/III study is to determine the efficacy and safety of NC-503 compared to a placebo in patients suffering from secondary (AA) amyloidosis by the assessment of clinical improvement/ worsening of both renal and gastrointestinal functions.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'PROTOCOL INCLUSION CRITERIA\n\n* Patients must be 18 years of age or older.\n* Males and females. If women of childbearing potential (i.e., not surgically sterilized or post-menopausal greater than one year) the patient must be using effective birth control.\n* Diagnosis of AA amyloidosis demonstrated by positive biopsy (Congo red staining) and immunohistochemistry or immunoelectron microscopy at screening visit. Tissue from previous biopsy can be used for confirmation of diagnosis, if available.\n* Persistent proteinuria defined as urinary protein excretion ? 1g/24h in two distinct 24-h urine collections at least 1 week apart within 3 months prior to study entry (baseline, Month 0 visit) without evidence of urinary tract infection or overt heart failure (NYHA class III or more); OR creatinine clearance ? 60 mL/min in two distinct measures at least 1 week apart within 3 months prior to study entry (baseline, Month 0 visit).\n* Creatinine clearance ? 20 mL/min AND serum creatinine ? 3 mg/dl within 3 months prior to study entry (baseline, Month 0 visit).\n* Written informed consent.\n\nPROTOCOL EXCLUSION CRITERIA\n\n* Evidence or suspicion of renal or renovascular diseases other than renal AA amyloidosis.\n* Presence of diabetes mellitus (Type I and II).\n* Evidence of a cause of potentially reversible reduced renal function, such as accelerated hypertension or drug nephrotoxicity.\n* AST, ALT, or ALP \\> 5 times the upper limit of normal, or total bilirubin 50% above upper limits of normal.\n* Presence of any other clinically significant diseases that could interfere with the interpretation of study results or compromise patient safety or any conditions that could reduce life expectancy to less than two years.\n* Use of an investigational drug within thirty days prior to the screening visit.\n* Active alcohol and/or drug abuse.\n* Initiation of or any changes in ACE inhibitor therapy within 3 months prior to the screening visit.\n* Initiation of or any changes in cytotoxic agents/colchicine therapy within 3 months prior to the screening visit.\n* Inability to provide legal consent.'}, 'identificationModule': {'nctId': 'NCT00035334', 'briefTitle': 'Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis', 'nctIdAliases': ['NCT00017667'], 'organization': {'class': 'INDUSTRY', 'fullName': 'Bellus Health Inc. - a GSK company'}, 'officialTitle': 'A Phase II/III Study of the Safety and Efficacy of NC-503 in Patients Suffering From Secondary (AA) Amyloidosis', 'orgStudyIdInfo': {'id': 'CL-503004'}}, 'armsInterventionsModule': {'interventions': [{'name': 'NC-503 (Anti-amyloidotic (AA) Agent)', 'type': 'DRUG'}]}, 'contactsLocationsModule': {'locations': [{'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'country': 'United States', 'facility': 'Indiana University School of Medicine, Department of Pathology and Laboratory Medicine,', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '02118', 'city': 'Boston', 'state': 'Massachusetts', 'country': 'United States', 'facility': 'Boston Medical Center, Renal Division', 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}, {'zip': '10029', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': 'Mount Sinai Medical Center', 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': 'FIN-18120', 'city': 'Heinola', 'country': 'Finland', 'facility': 'Rheumatism Foundation Hospital', 'geoPoint': {'lat': 61.20564, 'lon': 26.03811}}, {'zip': 'CEDEX 1', 'city': 'Le Mans', 'country': 'France', 'facility': 'Centre Hospitalier du Mans, Service de Rhumatologie', 'geoPoint': {'lat': 48.0021, 'lon': 0.20251}}, {'zip': 'CEDEX 59037', 'city': 'Lille', 'country': 'France', 'facility': 'Hôpital Claude Huriez, Service de médecine Interne, Clinique Médicale A', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'zip': '75679 CEDEX 14', 'city': 'Paris', 'country': 'France', 'facility': "Hôpital Cochin, Centre de Recherche et d'Explorations Fonctionnelles", 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '31048', 'city': 'Haifa', 'country': 'Israel', 'facility': 'Bnai Zion Medical Center', 'geoPoint': {'lat': 32.81303, 'lon': 34.99928}}, {'zip': '52621', 'city': 'Tel Litwinsky', 'country': 'Israel', 'facility': 'Heller Institute of Medical Research, Sheba Medical Center', 'geoPoint': {'lat': 32.05096, 'lon': 34.84588}}, {'zip': '27100', 'city': 'Pavia', 'country': 'Italy', 'facility': 'Italian Group for Systemic Amyloidosis, Biotechnology Research Laboratories, IRCCS Policlinico San Matteo, Internal Medicine and Medical Oncology', 'geoPoint': {'lat': 45.19205, 'lon': 9.15917}}, {'zip': '2001', 'city': 'Vilnius', 'country': 'Lithuania', 'facility': 'Vilnius University Hospital', 'geoPoint': {'lat': 54.68916, 'lon': 25.2798}}, {'zip': '9700 RB', 'city': 'Groningen', 'country': 'Netherlands', 'facility': 'University Hospital Groningen, Department of Medicine, Division of Rheumatology', 'geoPoint': {'lat': 53.21917, 'lon': 6.56667}}, {'zip': '02-632', 'city': 'Warsaw', 'country': 'Poland', 'facility': 'Instytut Reumatologiczny', 'geoPoint': {'lat': 52.22977, 'lon': 21.01178}}, {'zip': '53-137', 'city': 'Wroclaw', 'country': 'Poland', 'facility': 'Okregowy Szpital Kolejowy, Zaklad Reumatologii', 'geoPoint': {'lat': 51.10286, 'lon': 17.03006}}, {'zip': '115522', 'city': 'Moscow', 'country': 'Russia', 'facility': 'Institute of Rheumatology RAMS', 'geoPoint': {'lat': 55.75204, 'lon': 37.61781}}, {'zip': '320102', 'city': 'Yekaterinburg', 'country': 'Russia', 'facility': 'Regional Hospital No. 1', 'geoPoint': {'lat': 56.85733, 'lon': 60.61529}}, {'zip': '08916', 'city': 'Badalona', 'country': 'Spain', 'facility': 'Hospital Universitario Germans Trias I Pujol, Servicio de Reumatologia', 'geoPoint': {'lat': 41.45004, 'lon': 2.24741}}, {'zip': '08036', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Clinic I Provincial de Barcelona, Jefe del Departamento de Reumatologia', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': '08907', 'city': 'Llobregat', 'country': 'Spain', 'facility': 'Ciutad Sanitària y Universitària de Bellvitge, Servicio de Reumatologia, Hospitalet de Llobregat'}, {'zip': '28040', 'city': 'Madrid', 'country': 'Spain', 'facility': 'Hospital Clinico San Carlos de Madrid, Servicio de Reumatologia', 'geoPoint': {'lat': 40.4165, 'lon': -3.70256}}, {'city': 'Askaray, Istanbul, Turkey', 'country': 'Turkey (Türkiye)', 'facility': 'Cerrehpasa Tip Fakultesi', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}, {'zip': '34390 CAPA', 'city': 'Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology', 'geoPoint': {'lat': 41.01384, 'lon': 28.94966}}, {'zip': '81190', 'city': 'Uskudar, Altunizade, Istanbul', 'country': 'Turkey (Türkiye)', 'facility': 'Marmara University Medical School Hospital, Department of Rheumatology'}, {'zip': 'NW3 2PF', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Free and University College Medical School, Department of Medicine, National Amyloidosis Centre', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'G12 0YN', 'city': 'Scotland', 'country': 'United Kingdom', 'facility': 'Gartnavel General Hospital', 'geoPoint': {'lat': 53.8578, 'lon': -1.64198}}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Bellus Health Inc. - a GSK company', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'FDA Office of Orphan Products Development', 'class': 'FED'}]}}}