The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis (PRONTO)
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ClinicalTrials.gov Identifier: NCT02632786 |
Recruitment Status :
Completed
First Posted : December 17, 2015
Results First Posted : April 5, 2019
Last Update Posted : April 5, 2019
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Condition or disease | Intervention/treatment | Phase |
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AL Amyloidosis | Drug: NEOD001 Drug: Placebo | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 129 participants |
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 |
Study Start Date : | March 2016 |
Actual Primary Completion Date : | March 2018 |
Actual Study Completion Date : | March 2018 |
Arm | Intervention/treatment |
---|---|
Experimental: NEOD001
Study Drug given IV every 28 days at 24mg/kg
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Drug: NEOD001
NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates |
Placebo Comparator: Placebo
Placebo
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Drug: Placebo
Saline Bag |
- Number of Participants With Cardiac Response and Non-Response [ Time Frame: Baseline through 12 months of treatment ]N-terminal pro-brain natriuretic peptide (NT-proBNP ) best response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment. Cardiac best response, as assessed by NT-proBNP alone, is defined as the most favorable category among response (ie, decrease in NT-proBNP from baseline of >30% and >300 ng/L), stable (ie, neither response nor progression), and progression (ie, increase in NT-proBNP from baseline of >30% and >300 ng/L) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Non-response is defined as either stable or progression.
- SF-36v2 PCS Score [ Time Frame: Baseline to 12 months of treatment ]Change in Short Form-36 (SF-36 version 2) questionnaire Physical Component Summary [PCS] Score. PCS scores are calculated based on responses to specific Short Form-36 (version 2) questions using a weight scoring method. The lower the PCS score the more disability, the higher the score the less disability. A score of 50 is the mean in the US General Population and the standard deviation is 10. Minimum is 0 and maximum value is 100.
- 6MWT Distance [ Time Frame: Baseline to 12 months of treatment ]Change in 6 Minute Walk Test (6MWT) Distance (meters)
- Number of Participants With Renal Best Response and Non-Response [ Time Frame: Baseline through 12 months of treatment ]
Proteinuria and estimated Glomerular Filtration Rate (eGFR) response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment in subjects with renal involvement. Renal best response, as assessed by proteinuria, is defined as the most favorable category among response (ie, ≥30% decrease from baseline or <0.5 g/24 hours postbaseline result if subject does not meet criteria for progression), stable (ie, neither response nor progression), and progression (ie, ≥25% decrease in eGFR from baseline) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Assessments that qualify as both a response and progression are counted as progression.
Non-response is defined as either stable or progression.
- NIS-LL Total Score [ Time Frame: Baseline to 12 months of treatment ]Change in Neuropathy Impairment Score-Lower Limb (NIS-LL) Total Score in subjects with peripheral nerve involvement. NIS-LL is a scoring system graduated from 0 points to a maximum of 88 points (the absence of all motor, sensory, and reflex activity in the lower extremities). The scale is an additive of all deficits (64 potential points for muscle strength, 8 points for reflexes, and 16 points for sensory function) in the lower extremities. A score of 0 is normal and score of 88 is total impairment.
- NT-proBNP Slope [ Time Frame: Baseline through 12 months of treatment ]Rate of change in NT-proBNP (ng/L per infusion). Estimates of the intercept, slope, SE, and associated 95% CI for each treatment group, and the NEOD001 and placebo group difference comparisons are estimated using a general linear mixed effects model. The model fits a random intercept and slope for each subject and includes fixed effects for treatment group, time, treatment group by time interaction, IWRS stratification factors (hematologic response to first-line therapy: CR/VGPR, PR and NT-proBNP <1800 ng/L, ≥1800 ng/L), and an unstructured covariance structure to model the within-subject errors. Time is represented in months as a continuous variable and includes all scheduled time points, including baseline. The p-value is associated with the visit by treatment group interaction term.
- Hepatic Best Response [ Time Frame: Baseline through 12 months of treatment ]Alkaline Phosphatase response (Response or Non-Response [Stable, Progression]) from baseline through 12 months of treatment in subjects with hepatic involvement

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Age ≥18 years
- Confirmed diagnosis of systemic AL amyloidosis
- ≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response
- Cardiac involvement
- NT-proBNP ≥650
Exclusion Criteria:
- Non-AL amyloidosis
- Meets the International Myeloma Working Group (IMWG) definition of Multiple Myeloma
- NT-proBNP >5000
- Received Plasma cell directed chemotherapy within 6 months
- Received autologous stem cell transplant (ASCT) within 12 months

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02632786

Documents provided by Prothena Biosciences Ltd.:
Responsible Party: | Prothena Biosciences Ltd. |
ClinicalTrials.gov Identifier: | NCT02632786 |
Other Study ID Numbers: |
NEOD001-201 |
First Posted: | December 17, 2015 Key Record Dates |
Results First Posted: | April 5, 2019 |
Last Update Posted: | April 5, 2019 |
Last Verified: | April 2019 |
amyloidosis ntprobnp NEOD001 Prothena |
PRONTO amyloid plasma cell dyscrasia immunotherapy |
Immunoglobulin Light-chain Amyloidosis Amyloidosis Proteostasis Deficiencies Metabolic Diseases Neoplasms, Plasma Cell Neoplasms by Histologic Type |
Neoplasms Lymphoproliferative Disorders Immunoproliferative Disorders Immune System Diseases Paraproteinemias |