Clinical Trial: Study in Subjects With Light Chain (AL) Amyloidosis

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: A Phase 2b Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of NEOD001 in Subjects With Light Chain (AL) Amyloidosis Who Were Previously Enrolled in Study NEOD001-201 (PRONTO)

Brief Summary: The objective of this study is to evaluate the long-term safety and efficacy of NEOD001 in subjects with AL amyloidosis who have completed Study NEOD001-201.

Detailed Summary: Global, multicenter, Phase 2b, open-label extension study of subjects with AL amyloidosis who had a hematologic response to first-line treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and completed Study NEOD001-201. Subjects in this study may receive concomitant chemotherapy. Subject screening will occur during the 28 days prior to the first administration of study drug, which may overlap with the last visit in Study NEOD001-201. If all eligibility requirements are met, the subject will be enrolled and Screening assessments will be completed. Study visits will occur every 28 days based on scheduling from Month 1 Day 1. A ±5-day window is allowed for visits starting after Month 1. Subjects who discontinue study drug before the End of Study Visit (EOS) should have an Early Treatment Discontinuation Visit 30 (±5) days after their final administration of study drug. Each subject's study participation may be up to 38 months or until the study is terminated, whichever occurs first. The study consists of a Screening Phase (1 month), Treatment Phase (36 months), and EOS Visit (30 [±5] days after the last dose).
Sponsor: Prothena Therapeutics Ltd.

Current Primary Outcome: Long term safety and tolerability as assessed by vital signs, 12-lead ECGs, routine clinical laboratory assessments, and Adverse Events [ Time Frame: baseline up to 38 months ]

Safety will be assessed through changes in vital signs, 12-lead ECGs, laboratory test results, and summaries of AEs. All TEAEs and SAEs will be summarized.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Cardiac best response as measured by NT-proBNP [ Time Frame: baseline up to 38 months ]
    Response rates will be summarized including 95% confidence intervals
  • Change in Short Form-36 (SF-36) questionnaire [ Time Frame: baseline up to 38 months ]
    Change from baseline will be analyzed using linear mixed model for repeated measurements (MMRM)
  • Change in 6MWT [ Time Frame: baseline up to 38 months ]
    The 6 MWT distance change from baseline will be analyzed using a Wilcoxon signed rank test
  • Renal best response rate as measured by proteinuria [ Time Frame: baseline up to 38 months ]
    Response rates will be summarized including 95% confidence intervals. The distributions of time-to-event endpoints will be summarized using the Kaplan-Meier method
  • Change from baseline in Neuropathy Impairment Score-Lower Limbs (NIS-LL) total score [ Time Frame: baseline up to 38 months ]
    Change from baseline in NIS-LL will be summarized including 95% confidence intervals. The distributions of time-to-event endpoints will be summarized using the Kaplan-Meier method
  • Hepatic response [ Time Frame: baseline up to 38 months ]
    Response rates will be summarized including 95% confidence intervals


Original Secondary Outcome: Same as current

Information By: Prothena Therapeutics Ltd.

Dates:
Date Received: April 28, 2017
Date Started: June 30, 2017
Date Completion: September 30, 2020
Last Updated: May 12, 2017
Last Verified: May 2017