Study of the Safety and Efficacy of NC-503 in Secondary (AA) Amyloidosis

Phase 2

Completed

• Conditions: Rheumatoid Arthritis; Familial Mediterranean Syndrome; Nephrotic Syndrome; Kidney Diseases; Gastrointestinal Diseases; Secondary (AA) Amyloidosis

• Registration Number: NCT00035334
• Lead Sponsor: Bellus Health Inc. - a GSK company

Brief Summary

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.

Detailed Description

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.

Study Timeline

• Study Start: 2001-10
• Study First Submitted: 2002-05-02
• Study First Submitted QC: 2002-05-02
• Study First Posted: 2002-05-03
• Study Completion: 2004-12
• Status Verified: 2006-02
• Last Update Submitted: 2006-02-13
• Last Update Posted: 2006-02-14

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Trial Locations

Locations (26)

Instytut Reumatologiczny; 🇵🇱 Warszawa, Poland

Regional Hospital No. 1; 🇷🇺 Yekaterinburg, Russian Federation

Indiana University School of Medicine, Department of Pathology and Laboratory Medicine,; 🇺🇸 Indianapolis, Indiana, United States

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Centre Hospitalier du Mans, Service de Rhumatologie; 🇫🇷 Le Mans, France

Hôpital Claude Huriez, Service de médecine Interne, Clinique Médicale A; 🇫🇷 Lille, France

Rheumatism Foundation Hospital; 🇫🇮 Heinola, Finland

Mount Sinai Medical Center; 🇺🇸 New York, New York, United States

Bnai Zion Medical Center; 🇮🇱 Haifa, Israel

Vilnius University Hospital; 🇱🇹 Vilnius, Lithuania

Heller Institute of Medical Research, Sheba Medical Center; 🇮🇱 Tel Hashomer, Israel

Gartnavel General Hospital; 🇬🇧 Scotland, United Kingdom

Okregowy Szpital Kolejowy, Zaklad Reumatologii; 🇵🇱 Wroclaw, Poland

Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology; 🇹🇷 Istanbul, Turkey

Marmara University Medical School Hospital, Department of Rheumatology; 🇹🇷 Uskudar, Altunizade, Istanbul, Turkey

Ciutad Sanitària y Universitària de Bellvitge, Servicio de Reumatologia, Hospitalet de Llobregat; 🇪🇸 Llobregat, Spain

Institute of Rheumatology RAMS; 🇷🇺 Moscow, Russian Federation

Hospital Universitario Germans Trias I Pujol, Servicio de Reumatologia; 🇪🇸 Badalona, Spain

Hospital Clinico San Carlos de Madrid, Servicio de Reumatologia; 🇪🇸 Madrid, Spain

Cerrehpasa Tip Fakultesi; 🇹🇷 Askaray, Istanbul, Turkey, Turkey

Royal Free and University College Medical School, Department of Medicine, National Amyloidosis Centre; 🇬🇧 London, United Kingdom

Hospital Clinic I Provincial de Barcelona, Jefe del Departamento de Reumatologia; 🇪🇸 Barcelona, Spain

Boston Medical Center, Renal Division; 🇺🇸 Boston, Massachusetts, United States

Italian Group for Systemic Amyloidosis, Biotechnology Research Laboratories, IRCCS Policlinico San Matteo, Internal Medicine and Medical Oncology; 🇮🇹 Pavia, Italy

University Hospital Groningen, Department of Medicine, Division of Rheumatology; 🇳🇱 Groningen, Netherlands

Hôpital Cochin, Centre de Recherche et d'Explorations Fonctionnelles; 🇫🇷 Paris, France