Clinical trial NCT02632786: The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: AL Amyloidosis
Keywords: amyloidosis
ntprobnp
NEOD001
Prothena
PRONTO
amyloid
plasma cell dyscrasia
immunotherapy
Lead sponsor: Prothena Biosciences Ltd.
Sponsor class: INDUSTRY
First posted: 2015-12-17
Enrollment: 129
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Number of Participants With Cardiac Response and Non-Response
timeFrame: Baseline through 12 months of treatment
Secondary outcomes:
measure: SF-36v2 PCS Score
timeFrame: Baseline to 12 months of treatment
measure: 6MWT Distance
timeFrame: Baseline to 12 months of treatment
measure: Number of Participants With Renal Best Response and Non-Response
timeFrame: Baseline through 12 months of treatment
measure: NIS-LL Total Score
timeFrame: Baseline to 12 months of treatment
measure: NT-proBNP Slope
timeFrame: Baseline through 12 months of treatment
measure: Hepatic Best Response
timeFrame: Baseline through 12 months of treatment
Other outcomes:
Locations:
city: Duarte
country: United States
facility: City of Hope
state: California
city: Stanford
country: United States
facility: Stanford Cancer Institute (SCI)
state: California
city: Denver
country: United States
facility: Colorado Blood Cancer Institute
state: Colorado
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Chicago
country: United States
facility: University of Chicago Medicine
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Simon Cancer Center
state: Indiana
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Boston University School of Medicine
state: Massachusetts
city: Detroit
country: United States
facility: Karmanos Cancer Institute
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic - Minnesota
state: Minnesota
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: The Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: Hospital of the University of Pennsylvania
state: Pennsylvania
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: University of Texas; MD Anderson Cancer Center
state: Texas
city: Seattle
country: United States
facility: University of Washington/Seattle Cancer Care Alliance
state: Washington
city: Milwaukee
country: United States
facility: Froedtert & Medical College of Wisconsin, Cancer Center - Froedtert Hospital
state: Wisconsin
city: Sydney
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Woolloongabba
country: Australia
facility: The University of Queensland - Princess Alexandra Hospital (PAH)
state: Queensland
city: Box Hill
country: Australia
facility: Eastern Health (Box Hill Hospital)
state: Victoria
city: Vienna
country: Austria
facility: Medizinische Universität Wien, Allgemeines Krankenhaus der Stadt Wien
city: Limoges
country: France
facility: Hôpital Dupuytren - CHU Limoges
city: Paris
country: France
facility: Hôpital Pitié-Salpêtrière
city: Pierre-Benite Cedex
country: France
facility: Hopitaux Lyon Sud
city: Rennes Cedex 2
country: France
facility: CHU Rennes, Service de Medecine Interne
city: Berlin
country: Germany
facility: Charite-Universitatsmedizin
city: Essen
country: Germany
facility: Universitätsklinikum Essen
city: Hamburg
country: Germany
facility: Universitatsklinikum Hamburg-Eppendorf (UKE
city: Heidelberg
country: Germany
facility: Universitatsklinikum Heidelberg
city: Athens
country: Greece
facility: Alexandra General Hospital of Athens
city: Patras
country: Greece
facility: University Hospital of Patras
city: Jerusalem
country: Israel
facility: Hadassah University Medical Center
city: Pavia
country: Italy
facility: Policlinica San Matteo
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Majadahonda
country: Spain
facility: Hospital Universitario Puerta de Hierro - Majadahonda
city: Birmingham
country: United Kingdom
facility: Centre for Clinical Haematology
city: London
country: United Kingdom
facility: The Royal Free London NHS Foundation Trust - The Royal Free Hospital
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
References: