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Trial record 1 of 1 for:    NCT02632786
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The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis (PRONTO)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Prothena Therapeutics Ltd. Identifier:
First received: December 9, 2015
Last updated: March 30, 2017
Last verified: March 2017
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.

Condition Intervention Phase
AL Amyloidosis Drug: NEOD001 Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2b, Randomized, Double-blind, Placebo-controlled Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis Who Have Persistent Cardiac Dysfunction

Resource links provided by NLM:

Further study details as provided by Prothena Therapeutics Ltd.:

Primary Outcome Measures:
  • Cardiac best response as measured by NT-proBNP [ Time Frame: Baseline to 12 months ]

Secondary Outcome Measures:
  • Change in Short Form-36 (SF-36) Questionnaire [ Time Frame: Baseline to 12 months ]
  • Change in 6MWT [ Time Frame: Baseline to 12 months ]
  • Renal best response as measured by proteinuria [ Time Frame: Baseline to 12 Months ]
  • Change in Neuropathy Impairment Score- Lower Limb (NIS-LL) score [ Time Frame: Baseline to 12 Months ]
  • Progression Free Survival [ Time Frame: Baseline to 12 Months ]
  • To assess the safety of single agent NEOD001 by assessing vital signs, electrocardiogram(ECG),laboratory tests and AEs [ Time Frame: Baseline to 12 months ]

Estimated Enrollment: 100
Study Start Date: March 2016
Estimated Study Completion Date: January 2018
Estimated Primary Completion Date: January 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NEOD001
Study Drug given IV every 28 days at 24mg/kg
Drug: NEOD001
NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates
Placebo Comparator: Placebo
Drug: Placebo
Saline Bag


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
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Please refer to this study by its identifier: NCT02632786

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Sponsors and Collaborators
Prothena Therapeutics Ltd.
  More Information

Responsible Party: Prothena Therapeutics Ltd. Identifier: NCT02632786     History of Changes
Other Study ID Numbers: NEOD001-201
Study First Received: December 9, 2015
Last Updated: March 30, 2017

Keywords provided by Prothena Therapeutics Ltd.:
plasma cell dyscrasia

Additional relevant MeSH terms:
Proteostasis Deficiencies
Metabolic Diseases processed this record on September 19, 2017