Clinical Trial: Phase 1/2, Open Label, Dose Escalation Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase 1/2, Open Label, Dose Escalation Study of Intravenous Administration of Single Agent NEOD001 in Subjects With Light Chain (AL) Amyloidosis

Brief Summary: Dose escalation study to determine the maximum tolerated dose of NEOD001 in approximately 30 subjects with AL amyloidosis. Expansion phase to evaluate safety, efficacy and pharmacokinetics of NEOD001 in 25 additional subjects at the maximum tolerated dose.

Detailed Summary:

The purpose of the dose escalation phase of the study is to determine the maximum tolerated dose/Phase 2 recommended dose of NEOD001 when given as a single agent intravenously in approximately 30 subjects with AL amyloidosis.

The purpose of the expansion phase of the study is to evaluate the safety, preliminary efficacy and pharmacokinetics of single agent NEOD001 at the maximum tolerated dose/Phase 2 recommended dose in approximately 25 additional evaluable subjects.


Sponsor: Prothena Therapeutics Ltd.

Current Primary Outcome:

  • Safety and tolerability [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Adverse event profile
    • Dose limiting toxicity and maximum tolerated dose
  • Maximum tolerated dose [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Adverse event profile
    • Dose Limiting Toxicity and maximum tolerated dose


Original Primary Outcome:

  • Safety and tolerability [ Time Frame: 28 day cycles ]
    • Adverse event profile
    • Dose limiting toxicity and maximum tolerated dose
  • Maximum tolerated dose [ Time Frame: 28 day cycles ]
    • Adverse event profile
    • Dose Limiting Toxicity and maximum tolerated dose


Current Secondary Outcome:

  • Pharmacokinetics [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
  • Immunogenicity [ Time Frame: 28 day cycles, up to 1 year; beyond 1 year with approval of Sponsor and Investigator ]
    • Measurement of anti-NEOD001 antibodies


Original Secondary Outcome:

  • Pharmacokinetics [ Time Frame: 28 day cycles ]
    • Pharmacokinetic parameters including Cmax, Tmax, AUC, Cav, Cmin, t½, CL, and Vz
  • Immunogenicity [ Time Frame: 28 day cycles ]
    • Measurement of anti-NEOD001 antibodies


Information By: Prothena Therapeutics Ltd.

Dates:
Date Received: October 11, 2012
Date Started: April 2013
Date Completion: September 2016
Last Updated: October 10, 2015
Last Verified: October 2015