AL Amyloidosis Clinical Trial

A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis

Summary

A Phase 3 study to evaluate the efficacy and safety of birtamimab plus standard of care compared to placebo plus standard of care in Mayo Stage IV patients with AL amyloidosis.

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Full Description

This is a Phase 3 multicenter, global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of birtamimab in Mayo Stage IV patients with AL amyloidosis (i.e., Double-blind Phase), followed by a long-term, open-label extension (i.e., Open-label Extension [OLE] Phase). The primary objective of the Double-blind Phase is to evaluate the efficacy of birtamimab by assessing time to all-cause mortality. All patients will receive bortezomib-containing chemotherapy regimen as standard of care.

For the Double-blind Phase of the study, approximately 220 newly diagnosed Mayo Stage IV patients with AL amyloidosis will be enrolled and randomized in a 2:1 ratio to birtamimab or placebo. Subjects will remain on study until study completion, when the pre-defined number of events (all-cause mortality) have been reached.

After completion of the Double-blind Phase, eligible subjects may enter the optional OLE Phase, in which all subjects will receive open-label birtamimab treatment, regardless of Double-blind Phase randomized treatment assignment. Treatment in the OLE Phase will continue for an additional 24 months or until birtamimab is commercially available in a subject's country of residence, whichever occurs first (in accordance with country-specific regulations). The primary objective of the OLE Phase is to evaluate the long-term safety of birtamimab plus standard of care in Mayo Stage IV subjects with AL amyloidosis.

View Eligibility Criteria

Eligibility Criteria

Key Inclusion Criteria for Double-blind Phase:

Aged ≥18 years and legal age of consent according to local regulations
Newly diagnosed and AL amyloidosis treatment-naïve with cardiac involvement
Confirmed diagnosis of AL amyloidosis
Confirmed Mayo Stage IV AL Amyloidosis as defined by NT-proBNP ≥1800 pg/mL and Troponin-T ≥0.025 ng/mL or high sensitivity cardiac troponin T≥40ng/L and dFLC ≥18 mg/dL
Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly

Inclusion Criteria for Open-label (OLE) Phase:

Must not have discontinued treatment in Double-blind Phase
WOCBP must have a negative pregnancy test and must agree to use highly effective contraception through 90 days following last study drug administration
Male subjects must be surgically sterile or agree to use highly effective contraception through 90 days following last study drug administration
Ability to understand and willingness to sign an ICF prior to initiating the OLE Phase

Key Exclusion Criteria for Double-blind Phase:

Non-AL amyloidosis
NT-proBNP >8500 pg/mL
Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma except for malignancy biomarker of involved/uninvolved serum free light chain ratio ≥100
Subject is eligible for and plans to undergo ASCT or organ transplant during the study
Myocardial infarction, uncontrolled angina, severe uncontrolled ventricular arrhythmias, or ECG evidence of acute ischemia, within 6 months prior to the Month 1-Day 1 Visit
Severe valvular stenosis (e.g., aortic or mitral stenosis with a valve area <1.0 cm2) or severe congenital heart disease
ECG evidence of acute ischemia or active conduction system abnormalities
Prior treatment with hematopoietic growth factors, transfusions of blood or blood products within 1 week of Month 1-Day 1
Prior radiotherapy within 4 weeks of Month 1-Day 1
Prior treatment with plasma cell-directed chemotherapy, birtamimab, daratumumab, 11- 1F4, anti-serum amyloid P antibody, doxycycline for amyloid, or other investigational treatment directed at amyloid
Waldenström's macroglobulinemia and/or immunoglobulin M monoclonal gammopathy

Exclusion Criteria for OLE Phase:

Any medical condition or clinically significant abnormality on physical, neurological, laboratory, vital signs, or ECG examination that precludes treatment with birtamimab or participation in the study, in the medical judgment of the Investigator
Symptomatic orthostatic hypotension that in the medical judgment of the Investigator would interfere with subject's ability to safely receive treatment or complete study assessments
History of Grade ≥3 infusion-related AEs during the Double-blind Phase or hypersensitivity to birtamimab
Unable or unwilling to adhere to the study-specified procedures and restrictions
Planning to use any other investigational treatment during the study

Study is for people with:

AL Amyloidosis

Phase:

Phase 3

Estimated Enrollment:

220

Study ID:

NCT04973137

Recruitment Status:

Recruiting

Sponsor:

Prothena Biosciences Ltd.

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There are 124 Locations for this study

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St. Francis Hospital
Hartford Connecticut, 06105, United States
Yale Cancer Center
North Haven Connecticut, 06473, United States
Smilow Cancer Hospital Care Center at Trumbull
Trumbull Connecticut, 06611, United States
MedStar Georgetown University Hospital
Washington District of Columbia, 20007, United States
Cleveland Clinic Florida
Weston Florida, 33331, United States
Goshen Center for Cancer Care
Goshen Indiana, 46526, United States
Johns Hopkins University
Bethesda Maryland, 20814, United States
Boston University School of Med.
Boston Massachusetts, 02118, United States
Dana-Farber Cancer Institute
Boston Massachusetts, 02215, United States
University of Michigan Rogel Cancer Center
Ann Arbor Michigan, 48109, United States
Barbara Ann Karmanos Cancer Institute
Detroit Michigan, 48201, United States
Mayo Clinic - Rochester
Rochester Minnesota, 55905, United States
University of Nebraska Medical Center
Omaha Nebraska, 68105, United States
Hackensack University Medical Center
Hackensack New Jersey, 07601, United States
Roswell Park Comprehensive Cancer Center
Buffalo New York, 14263, United States
Perlmutter Cancer Center - 38th Street
New York New York, 10016, United States
New York University Langone Hospital - Long Island
New York New York, 11501, United States
Duke Clinical Research Institute
Durham North Carolina, 27705, United States
Wake Forest Baptist Health - Comprehensive Cancer Center
Winston-Salem North Carolina, 27157, United States
Cleveland Clinic Main Campus
Cleveland Ohio, 44195, United States
The Ohio State University College of Medicine
Columbus Ohio, 43210, United States
University of Tennessee Medical Center
Knoxville Tennessee, 37920, United States
Vanderbilt - Ingram Cancer Center - Henry-Joyce Cancer Clinic
Nashville Tennessee, 37232, United States
Baylor University Medical Center
Dallas Texas, 75246, United States
University of Washington
Seattle Washington, 98109, United States
Froedtert and Medical College of Wisconsin
Milwaukee Wisconsin, 53226, United States
SESLHD: St George Hospital
Kogarah New South Wales, 2217, Australia
Icon Cancer Centre - Wesley
Auchenflower Queensland, 4066, Australia
Icon Cancer Center
South Brisbane Queensland, 4101, Australia
Royal Adelaide Hospital
Adelaide South Australia, 5000, Australia
Box Hill Hospital
Box Hill Victoria, 3128, Australia
Royal Perth Hospital
Perth Western Australia, 6000, Australia
Paracelsus Medical University
Salzburg , 5020, Austria
Paracelsus Medical University
Salzburg , , Austria
Medizinische Universität
Vienna , , Austria
Medizinische Universität Wien
Wien , , Austria More Info
Hermine Agis, MD
Contact
Hermine Agis
Principal Investigator
Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan
Oostende , 8400, Belgium
AZ Sint-Jan Brugge-Oostende AV
Oostende , , Belgium
Centre Hospitalier Universitaire Universite Catholique de Louvain - Site Godinne
Yvoir , 5530, Belgium
CHU UCL Namur (Site Godinne)
Yvoir , , Belgium
Tom Baker Cancer Centre
Calgary Alberta, T2N4N, Canada
Royal Victoria Hospital
Montréal Quebec, H3A1A, Canada
Fakultní Nemocnice Brno
Brno Jihormoravsky Kraj, 62500, Czechia
Fakultni nemocnice ostrava
Ostrava-Poruba Moravian-Silesian, 70852, Czechia
Všeobecná fakultní nemocnice v Praze
Praha Prague, 12808, Czechia
Odense Universitetshospital
Odense Syddanmark, 5000, Denmark
Hôpital Bretonneau
Tours Cedex 9 Centre, 37044, France
Hôpital Haut-Lévêque
Pessac Gironde, 33604, France
Hopital Necker
Paris Cedex 15 Ill-de-France, 75743, France
Pitie-Salpêtrière Hospital
Paris Ill-de-France, 75013, France
Hôpitaux de Brabois
Vandœuvre-lès-Nancy Lorraine, 54511, France
Hopital Rangueil
Toulouse Cedex 9 Midi-Pyrenees, 31059, France
Hôpital Claude Huriez
Lille Nord, 59037, France
CHU Nantes
Nantes Pays De La Loire, 44093, France
Centre Hospitalier Universitaire Poitiers
Poitiers Vienne, 86021, France
CHU de Bordeaux
Bordeaux , , France
Hôpitaux Universitaires Henri Mondor
Créteil , 94000, France
Hopital Henri Mondor, Creteil
Créteil , , France
Centre Hospitalier Universitaire Limoges
Limoges , 87000, France
Hôpital Necker-Enfants Malades
Paris , , France
Universitätsklinikum Heidelberg
Heidelberg Baden-Wuerttemberg, 69120, Germany
Uniklinik Köln
Köln Baden-Württemberg, 50937, Germany
University Medicine Mainz
Mainz Rheinland-Pfalz, 55131, Germany
Charité Campus Mitte
Berlin , 10117, Germany
Alexandra General Hospital
Athens Attica, 11528, Greece
Theagenio Anti-Cancer Hospital of Thessaloniki
Thessaloníki Central Macedonia, 54007, Greece
University Hospital of Patras
Patra Peloponnese, 26504, Greece
Pécsi Tudományegyetem Klinikai Központ
Pécs Baranya, 7622, Hungary
Szent-Györgyi Albert Klinikai Központ
Szeged Csongrad, 6725, Hungary
Debreceni Egyetem Klinikai Központ
Debrecen Hajdu-Bihar County, 4032, Hungary
Semmelweis Egyetem - I. sz. Belgyógyászati Klinika
Budapest , 1087, Hungary
Dél-pesti Centrumkórház - Országos Hematológiai és Infektológiai Intézet
Budapest , 1097, Hungary
Cork University Hospital
Cork Munster, T12 D, Ireland
Cancer Clinical Trials and Research Unit, Beaumont Hospital
Dublin , , Ireland
Bnai Zion Medical Center
Haifa Haifa District, 31048, Israel
Rambam Medical Center
Haifa Haifa District, 31096, Israel
Hadassah Medical Center
Jerusalem Jerusalem District, 91120, Israel
Rabin Medical Center - Beilinson Hospital
Ashkelon Southern District, 78306, Israel
Samson Assuta Ashdod Hospital
Ashdod , 77476, Israel
Barzilai Medical Center
Ashkelon , 78306, Israel
Carmel Medical Center
Haifa , 34362, Israel
Azienda Ospedaliero Universitaria Ospedali Riuniti Di Ancona
Ancona , 60020, Italy
Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi
Bologna , 40138, Italy
ASST Spedali Civili
Brescia , 25123, Italy
Azienda Ospedaliero Universitaria Policlinico Gaspare Rodolico - San Marco
Catania , 95125, Italy
Fondazione IRCCS Policlinico San Matteo
Pavia , 27100, Italy
Azienda Ospedaliero-Universitaria Pisana
Pisa , 56124, Italy
Umberto I - Policlinico di Roma
Roma , 00161, Italy
Kyoto Kuramaguchi Medical Center
Kita Kyoto, , Japan
Shinshu University Hospital
Matsumoto Nagano, 390-0, Japan
Jichi medical university hospital
Shimotsuke Tochigi, , Japan
Tokushima University Hospital
Kuramoto Tokushima, , Japan
Red Cross Medical Center
Shibuya-ku Tokyo, 150-8, Japan
Nagoya City University Hospital
Aichi , , Japan
Fukushima Medical University Hospital
Fukushima , 960-1, Japan
Keimyung University Dongsan Hospital
Daegu Gyeongsangbugdo, 42601, Korea, Republic of
Chonnam National University Hwasun Hospital
Gwangju Jeollanam-do, 58128, Korea, Republic of
Severance Hospital
Seoul Seoul Teugbyeols, 120-7, Korea, Republic of
Pusan National University Hospital
Busan , 49241, Korea, Republic of
Kosin University Gospel Hospital
Busan , 60270, Korea, Republic of
Gachon University Gil Medical Center
Incheon , 21565, Korea, Republic of
Seoul National University Hospital
Seoul , 03080, Korea, Republic of
Hanyang University Seoul Hospital
Seoul , 04763, Korea, Republic of
Samsung Medical Center
Seoul , 06351, Korea, Republic of
The Catholic University of Korea - Seoul St. Mary's Hospital
Seoul , 06591, Korea, Republic of
Maastricht Universitair Medisch Centrum
Maastricht Limburg, 6229H, Netherlands
Universitair Medisch Centrum Groningen
Groningen , 9713G, Netherlands
Klinika Hematologii i Transplantologii
Gdańsk Pomorskie, 80-21, Poland
Hospital de Braga
Braga , 4710-, Portugal
Centro Hospitalar e Universitário de Coimbra
Coimbra , 3000-, Portugal
Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria
Lisboa , 1649-, Portugal
Hospital Universitario Son Espases
Palma De Mallorca Balearic Islands, 07120, Spain
Hospital Son Llatzer
Palma De Mallorca Balearic Islands, 07198, Spain
Institut Català d'Oncologia - Hospital Germans Trias i Pujol
Badalona Barcelona, 08916, Spain
University Hospital Marqués de Valdecilla
Santander Cantabria, 39008, Spain
Hospital Universitario Puerta de Hierro - Majadahonda
Majadahonda Madrid, 28222, Spain
Clinica Universidad de Navarra -Madrid
Pamplona Navarra, , Spain
Clinica Universidad de Navarra - Pamplona
Pamplona Navarre, 31008, Spain
Hospital Universitario de Canarias
Tenerife Santa Cruz De Tenerife, 38320, Spain
Hospital Universitari Vall d'hebron
Barcelona , 08035, Spain
Hospital Clinic of Barcelona
Barcelona , 08036, Spain
Institut Català d'Oncologia - Hospital Duran i Reynals
Barcelona , 08908, Spain
Hospital Josep Trueta ICO Girona
Girona , 17007, Spain
Hospital Ramon y Cajal
Madrid , 28034, Spain
Complejo Asistencial Universitario de Salamanca - Hospital Clínico
Salamanca , 37007, Spain
Hospital Universitari i Politècnic la Fe
Valencia , 46026, Spain
Hospital Universitario 12 de Octubre
Vila-real , 1254, Spain
National Taiwan University Hospital
Taipei City Taipei, 10047, Taiwan
Kaohsiung Chang Gung Memorial Hospital
Kaohsiung , 833, Taiwan
Taipei Veterans General Hospital
Taipei , 11217, Taiwan
Ankara UMF
Ankara , 06100, Turkey
Gazi University
Ankara , 06500, Turkey
Istanbul University Cerrahpasa Medical Faculty
Istanbul , 34320, Turkey
Ege Universitesi Tip Fakultesi
İzmir , 35100, Turkey
University Hospitals Birmingham NHS Foundation Trust - Queen Elizabeth Hospital
Birmingham England, B152S, United Kingdom
The Newcastle Upon Tyne Hospitals NHS Foundation Trust
High Heaton England, NE77D, United Kingdom
Barts Health NHS Trust
London England, EC1A7, United Kingdom
University College London Hospitals
London England, NW12P, United Kingdom
Manchester University NHS Foundation Trust
Manchester England, M139W, United Kingdom
Nottingham University Hospitals NHS Trust
Nottingham England, NG51P, United Kingdom
Belfast City Hospital
Belfast , , United Kingdom

How clear is this clinincal trial information?

Study is for people with:

AL Amyloidosis

Phase:

Phase 3

Estimated Enrollment:

220

Study ID:

NCT04973137

Recruitment Status:

Recruiting

Sponsor:


Prothena Biosciences Ltd.

How clear is this clinincal trial information?

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