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

• Status: Completed
• Trial end date: 2004-12-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Bellus Health Inc
• Collaborator: FDA Office of Orphan Products Development

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.