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

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Brief Title

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

Official Title

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

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 Phase

Phase 2/Phase 3

Study Type

Interventional




Condition

Secondary (AA) Amyloidosis

Intervention

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


Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

150

Start Date

October 2001

Completion Date

December 2004


Eligibility Criteria

        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.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Finland

Location Countries

Finland

Administrative Informations


NCT ID

NCT00035334

Organization ID

CL-503004



Study Sponsor

Bellus Health Inc

Collaborators

 FDA Office of Orphan Products Development

Study Sponsor

, , 


Verification Date

February 2006