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

NCT00035334 | Completed Phase 2

• 1 other identifier: CL-503004
• Study Type: interventional
• Target: 150
• Locations: 12 countries
• Sites: 26

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.

Conditions

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

Keywords

Familial Mediterranean Fever; Amyloidosis; Secondary (AA) Amyloidosis; Nephrotic Syndrome

Interventions

NC-503 (Anti-amyloidotic (AA) Agent) DRUG

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must be 18 years of age or older.
• 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.
• 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.
• 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).
• Creatinine clearance ? 20 mL/min AND serum creatinine ? 3 mg/dl within 3 months prior to study entry (baseline, Month 0 visit).
• Written informed consent.

You may not qualify if:

• Evidence or suspicion of renal or renovascular diseases other than renal AA amyloidosis.
• Presence of diabetes mellitus (Type I and II).
• Evidence of a cause of potentially reversible reduced renal function, such as accelerated hypertension or drug nephrotoxicity.
• AST, ALT, or ALP \> 5 times the upper limit of normal, or total bilirubin 50% above upper limits of normal.
• 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.
• Use of an investigational drug within thirty days prior to the screening visit.
• Active alcohol and/or drug abuse.
• Initiation of or any changes in ACE inhibitor therapy within 3 months prior to the screening visit.
• Initiation of or any changes in cytotoxic agents/colchicine therapy within 3 months prior to the screening visit.
• Inability to provide legal consent.

Sponsors & Collaborators

• Bellus Health Inc. - a GSK company: lead
• FDA Office of Orphan Products Development: collaborator

Study Sites (26)

Indiana University School of Medicine, Department of Pathology and Laboratory Medicine,

Indianapolis, Indiana, 46202, United States

Location

Boston Medical Center, Renal Division

Boston, Massachusetts, 02118, United States

Location

Mayo Clinic

Rochester, Minnesota, 55905, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Rheumatism Foundation Hospital

Heinola, FIN-18120, Finland

Location

Centre Hospitalier du Mans, Service de Rhumatologie

Le Mans, CEDEX 1, France

Location

Hôpital Claude Huriez, Service de médecine Interne, Clinique Médicale A

Lille, CEDEX 59037, France

Location

Hôpital Cochin, Centre de Recherche et d'Explorations Fonctionnelles

Paris, 75679 CEDEX 14, France

Location

Bnai Zion Medical Center

Haifa, 31048, Israel

Location

Heller Institute of Medical Research, Sheba Medical Center

Tel Litwinsky, 52621, Israel

Location

Italian Group for Systemic Amyloidosis, Biotechnology Research Laboratories, IRCCS Policlinico San Matteo, Internal Medicine and Medical Oncology

Pavia, 27100, Italy

Location

Vilnius University Hospital

Vilnius, 2001, Lithuania

Location

University Hospital Groningen, Department of Medicine, Division of Rheumatology

Groningen, 9700 RB, Netherlands

Location

Instytut Reumatologiczny

Warsaw, 02-632, Poland

Location

Okregowy Szpital Kolejowy, Zaklad Reumatologii

Wroclaw, 53-137, Poland

Location

Institute of Rheumatology RAMS

Moscow, 115522, Russia

Location

Regional Hospital No. 1

Yekaterinburg, 320102, Russia

Location

Hospital Universitario Germans Trias I Pujol, Servicio de Reumatologia

Badalona, 08916, Spain

Location

Hospital Clinic I Provincial de Barcelona, Jefe del Departamento de Reumatologia

Barcelona, 08036, Spain

Location

Ciutad Sanitària y Universitària de Bellvitge, Servicio de Reumatologia, Hospitalet de Llobregat

Llobregat, 08907, Spain

Location

Hospital Clinico San Carlos de Madrid, Servicio de Reumatologia

Madrid, 28040, Spain

Location

Cerrehpasa Tip Fakultesi

Askaray, Istanbul, Turkey, Turkey (Türkiye)

Location

Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology

Istanbul, 34390 CAPA, Turkey (Türkiye)

Location

Marmara University Medical School Hospital, Department of Rheumatology

Uskudar, Altunizade, Istanbul, 81190, Turkey (Türkiye)

Location

Royal Free and University College Medical School, Department of Medicine, National Amyloidosis Centre

London, NW3 2PF, United Kingdom

Location

Gartnavel General Hospital

Scotland, G12 0YN, United Kingdom

Location

Related Publications (2)

• 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)

  BACKGROUND

• 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.

  PMID: 17554116 DERIVED

MeSH Terms

Conditions

Neoplasm Metastasis; Amyloidosis; Arthritis, Rheumatoid; Nephrotic Syndrome; Kidney Diseases; Gastrointestinal Diseases; Familial Mediterranean Fever

Interventions

Pharmaceutical Preparations

Condition Hierarchy (Ancestors)

Neoplastic Processes; Neoplasms; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Arthritis; Joint Diseases; Musculoskeletal Diseases; Rheumatic Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Nephrosis; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Digestive System Diseases; Hereditary Autoinflammatory Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: May 2, 2002
• First Posted: May 3, 2002
• Study Start: October 1, 2001
• Study Completion: December 1, 2004
• Last Updated: February 14, 2006

Record last verified: 2006-02

Locations

LI (1); NL (1); TU (3); GB (2); US (4); FI (1); PL (2); ES (4); FR (3); IL (2); RU (2); IT (1)