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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02632786
• Date of registration: 09/12/2015
• Prospective Registration: Yes
• Primary sponsor: Prothena Therapeutics Ltd.
• Public title: The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis PRONTO
• Scientific 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
• Date of first enrolment: March 2016
• Target sample size: 129
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02632786
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Austria, France, Germany, Greece, Israel, Italy, Spain, United Kingdom, United States

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

AL Amyloidosis

Intervention(s)

Drug: NEOD001

Drug: Placebo

Primary Outcome(s)

Number of Participants With Cardiac Response and Non-Response [Time Frame: Baseline through 12 months of treatment]

Secondary Outcome(s)

NIS-LL Total Score [Time Frame: Baseline to 12 months of treatment]

6MWT Distance [Time Frame: Baseline to 12 months of treatment]

SF-36v2 PCS Score [Time Frame: Baseline to 12 months of treatment]

NT-proBNP Slope [Time Frame: Baseline through 12 months of treatment]

Number of Participants With Renal Best Response and Non-Response [Time Frame: Baseline through 12 months of treatment]

Hepatic Best Response [Time Frame: Baseline through 12 months of treatment]

Secondary ID(s)

NEOD001-201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 05/04/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02632786