The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis

• Status: Completed
• Trial end date: 2018-03-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a global, multicenter, Phase 2b, randomized, double-blind, placebo-controlled, two-arm, parallel-group efficacy and safety study of NEOD001 as a single agent administered intravenously in adults with AL amyloidosis who had a hematologic response to previous treatment for their amyloidosis (e.g., chemotherapy, autologous stem cell transplant [ASCT]) and have persistent cardiac dysfunction.

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

Details

• Lead Sponsor: Prothena Biosciences Ltd.; Prothena Therapeutics Ltd.

Criteria

Inclusion Criteria:

1. Age ≥18 years

2. Confirmed diagnosis of systemic AL amyloidosis

3. ≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response

4. Cardiac involvement

5. NT-proBNP ≥650

Exclusion Criteria:

1. Non-AL amyloidosis

2. Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma

3. NT-proBNP >5000

4. Received Plasma cell directed chemotherapy within 6 months

5. Received autologous stem cell transplant (ASCT) within 12 months