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

February 13, 2006 updated by: Bellus Health Inc

A Phase II/III Study of the Safety and Efficacy of NC-503 in Patients Suffering From Secondary (AA) Amyloidosis

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment

150

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Finland
      • Heinola, Finland, FIN-18120
        • Rheumatism Foundation Hospital
  • France
      • Le Mans, France, CEDEX 1
        • Centre Hospitalier du Mans, Service de Rhumatologie
      • Lille, France, CEDEX 59037
        • Hôpital Claude Huriez, Service de médecine Interne, Clinique Médicale A
      • Paris, France, 75679 CEDEX 14
        • Hôpital Cochin, Centre de Recherche et d'Explorations Fonctionnelles
  • Israel
      • Haifa, Israel, 31048
        • Bnai Zion Medical Center
      • Tel Hashomer, Israel, 52621
        • Heller Institute of Medical Research, Sheba Medical Center
  • Italy
      • Pavia, Italy, 27100
        • Italian Group for Systemic Amyloidosis, Biotechnology Research Laboratories, IRCCS Policlinico San Matteo, Internal Medicine and Medical Oncology
  • Lithuania
      • Vilnius, Lithuania, 2001
        • Vilnius University Hospital
  • Netherlands
      • Groningen, Netherlands, 9700 RB
        • University Hospital Groningen, Department of Medicine, Division of Rheumatology
  • Poland
      • Warszawa, Poland, 02-632
        • Instytut Reumatologiczny
      • Wroclaw, Poland, 53-137
        • Okregowy Szpital Kolejowy, Zaklad Reumatologii
  • Russian Federation
      • Moscow, Russian Federation, 115522
        • Institute of Rheumatology RAMS
      • Yekaterinburg, Russian Federation, 320102
        • Regional Hospital No. 1
  • Spain
      • Badalona, Spain, 08916
        • Hospital Universitario Germans Trias I Pujol, Servicio de Reumatologia
      • Barcelona, Spain, 08036
        • Hospital Clinic I Provincial de Barcelona, Jefe del Departamento de Reumatologia
      • Llobregat, Spain, 08907
        • Ciutad Sanitària y Universitària de Bellvitge, Servicio de Reumatologia, Hospitalet de Llobregat
      • Madrid, Spain, 28040
        • Hospital Clinico San Carlos de Madrid, Servicio de Reumatologia
  • Turkey
      • Askaray, Istanbul, Turkey, Turkey
        • Cerrehpasa Tip Fakultesi
      • Istanbul, Turkey, 34390 CAPA
        • Istanbul Faculty of Medicine, Department of Internal Medicine, Division of Rheumatology
      • Uskudar, Altunizade, Istanbul, Turkey, 81190
        • Marmara University Medical School Hospital, Department of Rheumatology
  • United Kingdom
      • London, United Kingdom, NW3 2PF
        • Royal Free and University College Medical School, Department of Medicine, National Amyloidosis Centre
      • Scotland, United Kingdom, G12 0YN
        • Gartnavel General Hospital
  • United States
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University School of Medicine, Department of Pathology and Laboratory Medicine,
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Boston Medical Center, Renal Division
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Mayo Clinic
    • New York
      • New York, New York, United States, 10029
        • Mount Sinai Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

PROTOCOL INCLUSION CRITERIA

  • 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.

PROTOCOL EXCLUSION CRITERIA

  • 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bellus Health Inc

Collaborators

FDA Office of Orphan Products Development

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

October 1, 2001

Study Completion

December 1, 2004

Study Registration Dates

First Submitted

May 2, 2002

First Submitted That Met QC Criteria

May 2, 2002

First Posted (Estimate)

May 3, 2002

Study Record Updates

Last Update Posted (Estimate)

February 14, 2006

Last Update Submitted That Met QC Criteria

February 13, 2006

Last Verified

February 1, 2006

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CL-503004

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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