NCT03154047

Study in Subjects With Light Chain (AL) Amyloidosis

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)

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 80 (actual)
• Sponsor: Prothena Biosciences Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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 description

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).

Conditions

• AL Amyloidosis

Interventions

Type	Name	Description
DRUG	NEOD001	humanized monoclonal antibody

Timeline

• Start date: 2017-06-14
• Primary completion: 2018-05-30
• Completion: 2018-05-30
• First posted: 2017-05-15
• Last updated: 2019-03-26
• Results posted: 2018-12-12

Locations

34 sites across 10 countries: United States, Australia, Austria, France, Germany, Greece, Israel, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study