NCT02632786 · REGISTRY CONDITION SEARCH

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

Found through the registry condition index, not selected as a treatment recommendation. This discovery collection was restricted to studies with posted results.

Phase: PHASE2
Status at capture: COMPLETED
Registry last update: 2019-04-05

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.

Open the original ClinicalTrials.gov record → · Download preserved record

What did the study report?

Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.

flow

Participant flow

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baseline

Baseline characteristics

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outcome · POSTED

Number of Participants With Cardiac Response and Non-Response

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outcome · POSTED

SF-36v2 PCS Score

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outcome · POSTED

6MWT Distance

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outcome · POSTED

Number of Participants With Renal Best Response and Non-Response

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outcome · POSTED

NIS-LL Total Score

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outcome · POSTED

NT-proBNP Slope

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outcome · POSTED

Hepatic Best Response

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adverse events

Adverse events

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more info

Limitations, agreements, and source contact

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Who could take part

eligibility 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
healthy Volunteers: false
minimum Age: 18 Years
sex: ALL
std Ages: ADULT
OLDER_ADULT

Treatment arms and interventions

arm Groups

description
Study Drug given IV every 28 days at 24mg/kg
intervention Names
Drug: NEOD001
label
NEOD001
type
EXPERIMENTAL
description
Placebo
intervention Names
Drug: Placebo
label
Placebo
type
PLACEBO_COMPARATOR

interventions

arm Group Labels
NEOD001
description
NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates
name
NEOD001
type
DRUG
arm Group Labels
Placebo
description
Saline Bag
name
Placebo
type
DRUG

Study design

allocation

RANDOMIZED

intervention Model

PARALLEL

masking Info

masking: QUADRUPLE
who Masked: PARTICIPANT
CARE_PROVIDER
INVESTIGATOR
OUTCOMES_ASSESSOR

primary Purpose

TREATMENT

Enrollment

count: 129
type: ACTUAL

Registered outcome plans (not posted results)

primary Outcomes

description
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.
measure
Number of Participants With Cardiac Response and Non-Response
time Frame
Baseline through 12 months of treatment

secondary Outcomes

description
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.
measure
SF-36v2 PCS Score
time Frame
Baseline to 12 months of treatment
description
Change in 6 Minute Walk Test (6MWT) Distance (meters)
measure
6MWT Distance
time Frame
Baseline to 12 months of treatment
description
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.
measure
Number of Participants With Renal Best Response and Non-Response
time Frame
Baseline through 12 months of treatment
description
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.
measure
NIS-LL Total Score
time Frame
Baseline to 12 months of treatment
description
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.
measure
NT-proBNP Slope
time Frame
Baseline through 12 months of treatment
description
Alkaline Phosphatase response (Response or Non-Response \[Stable, Progression\]) from baseline through 12 months of treatment in subjects with hepatic involvement
measure
Hepatic Best Response
time Frame
Baseline through 12 months of treatment

Full study description

brief 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.

Source references

Source notices and limitations

Discovery and provenance

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source url
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Preserved source evidence · Not medical advice