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

NCT ID: NCT02632786

Last Updated: 2019-04-05

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

129 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-03-31

Study Completion Date

2018-03-31

Brief Summary

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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.

Detailed Description

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Conditions

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AL Amyloidosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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NEOD001

Study Drug given IV every 28 days at 24mg/kg

Group Type EXPERIMENTAL

NEOD001

Intervention Type DRUG

NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates

Placebo

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Saline Bag

Interventions

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NEOD001

NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates

Intervention Type DRUG

Placebo

Saline Bag

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Prothena Biosciences Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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City of Hope

Duarte, California, United States

Site Status

Stanford Cancer Institute (SCI)

Stanford, California, United States

Site Status

Colorado Blood Cancer Institute

Denver, Colorado, United States

Site Status

Mayo Clinic

Jacksonville, Florida, United States

Site Status

University of Chicago Medicine

Chicago, Illinois, United States

Site Status

Indiana University Simon Cancer Center

Indianapolis, Indiana, United States

Site Status

Tufts Medical Center

Boston, Massachusetts, United States

Site Status

Boston University School of Medicine

Boston, Massachusetts, United States

Site Status

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status

Mayo Clinic - Minnesota

Rochester, Minnesota, United States

Site Status

Memorial Sloan-Kettering Cancer Center

New York, New York, United States

Site Status

Duke University Medical Center

Durham, North Carolina, United States

Site Status

The Cleveland Clinic

Cleveland, Ohio, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Hospital of the University of Pennsylvania

Philadelphia, Pennsylvania, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas; MD Anderson Cancer Center

Houston, Texas, United States

Site Status

University of Washington/Seattle Cancer Care Alliance

Seattle, Washington, United States

Site Status

Froedtert & Medical College of Wisconsin, Cancer Center - Froedtert Hospital

Milwaukee, Wisconsin, United States

Site Status

Westmead Hospital

Sydney, New South Wales, Australia

Site Status

The University of Queensland - Princess Alexandra Hospital (PAH)

Woolloongabba, Queensland, Australia

Site Status

Eastern Health (Box Hill Hospital)

Box Hill, Victoria, Australia

Site Status

Medizinische Universität Wien, Allgemeines Krankenhaus der Stadt Wien

Vienna, , Austria

Site Status

Hôpital Dupuytren - CHU Limoges

Limoges, , France

Site Status

Hôpital Pitié-Salpêtrière

Paris, , France

Site Status

Hopitaux Lyon Sud

Pierre-Bénite, , France

Site Status

CHU Rennes, Service de Medecine Interne

Rennes, , France

Site Status

Charite-Universitatsmedizin

Berlin, , Germany

Site Status

Universitätsklinikum Essen

Essen, , Germany

Site Status

Universitatsklinikum Hamburg-Eppendorf (UKE

Hamburg, , Germany

Site Status

Universitatsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Alexandra General Hospital of Athens

Athens, , Greece

Site Status

University Hospital of Patras

Pátrai, , Greece

Site Status

Hadassah University Medical Center

Jerusalem, , Israel

Site Status

Policlinica San Matteo

Pavia, , Italy

Site Status

Hospital Clinic de Barcelona

Barcelona, , Spain

Site Status

Hospital Universitario Puerta de Hierro - Majadahonda

Majadahonda, , Spain

Site Status

Centre for Clinical Haematology

Birmingham, , United Kingdom

Site Status

The Royal Free London NHS Foundation Trust - The Royal Free Hospital

London, , United Kingdom

Site Status

Southampton General Hospital

Southampton, , United Kingdom

Site Status

Countries

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

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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NEOD001-201

Identifier Type: -

Identifier Source: org_study_id

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