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Experimental antibody trial for rare heart disease halted early

NCT ID NCT04973137

Disease control Sponsor: Prothena Biosciences Ltd. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This phase 3 study tested an experimental drug called birtamimab in 208 people with advanced AL amyloidosis, a rare disease where abnormal proteins damage organs like the heart. Participants received either birtamimab or a placebo, plus standard chemotherapy. The goal was to see if birtamimab could improve survival. However, the study was terminated early, so the results are limited.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
birtamimab (an investigational antibody given by IV every 28 days)
What this could lead to
If it worked, birtamimab could help control a rare, serious disease that affects the heart and other organs, potentially improving survival.
What could go wrong
The trial was terminated early, so we don't have full results. It's unclear if birtamimab is effective or safe for this very advanced stage of disease.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

208 people

The number who actually took part.

Started

Aug 2021

Finished

Jun 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

AL amyloidosis amyloidosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Light chain (AL) amyloidosis

Contacts and locations

Locations

  • AZ Sint-Jan Brugge-Oostende AV

    Ostend, Belgium

  • Alexandra General Hospital

    Athens, Attica, 11528, Greece

  • Algemeen Ziekenhuis Sint-Jan Brugge-Oostende - Campus Sint-Jan

    Ostend, Oostende, 8400, Belgium

  • Ankara UMF

    Ankara, Ankara, 06100, Turkey (Türkiye)

  • Azienda Ospedaliero-Universitaria Pisana

    Pisa, 56124, Italy

  • Azienda Ospedaliero-Universitaria di Bologna Policlinico Sant Orsola-Malpighi

    Bologna, Bologna, 40138, Italy

  • Barbara Ann Karmanos Cancer Institute

    Detroit, Michigan, 48201, United States

  • Barts Health NHS Trust

    London, England, EC1A7BE, United Kingdom

  • Barzilai Medical Center

    Ashkelon, 7830604, Israel

  • Bnai Zion Medical Center

    Haifa, Haifa District, 31048, Israel

  • Boston University School of Med.

    Boston, Massachusetts, 02118, United States

  • CHU Nantes

    Nantes, Pays de la Loire Region, 44093, France

  • CHU UCL Namur (Site Godinne)

    Yvoir, Belgium

  • CHU de Bordeaux

    Bordeaux, France

  • Cancer Clinical Trials and Research Unit, Beaumont Hospital

    Dublin, Ireland

  • Carmel Medical Center

    Haifa, 3436212, Israel

  • Centre Hospitalier Universitaire Poitiers

    Poitiers, Vienne, 86021, France

  • Centre Hospitalier Universitaire Universite Catholique de Louvain - Site Godinne

    Yvoir, Yvoir, 5530, Belgium

  • Centro Hospitalar Universitário Lisboa Norte - Hospital De Santa Maria

    Lisbon, Lisbon District, 1649-028, Portugal

  • Chonnam National University Hwasun Hospital

    Gwangju, Jeollanam-do, 58128, South Korea

  • Cleveland Clinic Florida

    Weston, Florida, 33331, United States

  • Cleveland Clinic Main Campus

    Cleveland, Ohio, 44195, United States

  • Clinica Universidad de Navarra - Pamplona

    Pamplona, Navarre, 31008, Spain

  • Clinica Universidad de Navarra -Madrid

    Pamplona, Navarre, Spain

  • Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Complejo Asistencial Universitario de Salamanca - Hospital Clínico

    Salamanca, Salamanca, 37007, Spain

  • Cork University Hospital

    Cork, Munster, T12 DFK4, Ireland

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Duke Clinical Research Institute

    Durham, North Carolina, 27705, United States

  • Ege Universitesi Tip Fakultesi

    Izmir, İzmir, 35100, Turkey (Türkiye)

  • Fakultni nemocnice ostrava

    Ostrava-Poruba, Moravian-Silesian, 70852, Czechia

  • Froedtert and Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Fukushima Medical University Hospital

    Fukushima, Fukushima, 960-1247, Japan

  • Gachon University Gil Medical Center

    Incheon, 21565, South Korea

  • Gazi University

    Ankara, Ankara, 06500, Turkey (Türkiye)

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Hadassah Medical Center

    Jerusalem, Jerusalem, 9112001, Israel

  • Hopital Henri Mondor, Creteil

    Créteil, France

  • Hopital Necker

    Paris, Ill-de-France, 75743, France

  • Hopital Rangueil

    Toulouse, Midi-Pyrenees, 31059, France

  • Hospital Clinic of Barcelona

    Barcelona, Barcelona, 08036, Spain

  • Hospital Josep Trueta ICO Girona

    Girona, Girona, 17007, Spain

  • Hospital Ramon y Cajal

    Madrid, Madrid, 28034, Spain

  • Hospital Universitario 12 de Octubre

    Vila-real, 1254, Spain

  • Hospital Universitario Son Espases

    Palma de Mallorca, Balearic Islands, 07120, Spain

  • Hospital Universitario de Canarias

    Santa Cruz de Tenerife, Santa Cruz de Tenerife, 38320, Spain

  • Hospital de Braga

    Braga, Braga District, 4710-243, Portugal

  • Hôpital Bretonneau

    Tours, Centre-Val de Loire, 37044, France

  • Hôpital Claude Huriez

    Lille, Nord, 59037, France

  • Hôpital Haut-Lévêque

    Pessac, Gironde, 33604, France

  • Hôpital Necker-Enfants Malades

    Paris, France

  • Hôpitaux Universitaires Henri Mondor

    Créteil, Créteil, 94000, France

  • Hôpitaux de Brabois

    Vandœuvre-lès-Nancy, Lorraine, 54511, France

  • Institut Català d'Oncologia - Hospital Duran i Reynals

    Barcelona, Barcelona, 08908, Spain

  • Institut Català d'Oncologia - Hospital Germans Trias i Pujol

    Badalona, Barcelona, 08916, Spain

  • Istanbul University Cerrahpasa Medical Faculty

    Istanbul, Istanbul, 34320, Turkey (Türkiye)

  • Jichi medical university hospital

    Shimotsuke, Tochigi, Japan

  • Johns Hopkins University

    Bethesda, Maryland, 20814-1422, United States

  • Kaohsiung Chang Gung Memorial Hospital

    Kaohsiung City, Kaohsiung, 833, Taiwan

  • Keimyung University Dongsan Hospital

    Daegu, Gyeongsangbuk-do, 42601, South Korea

  • Klinika Hematologii i Transplantologii

    Gdansk, Pomeranian Voivodeship, 80-214, Poland

  • Kosin University Gospel Hospital

    Busan, 602702, South Korea

  • Kyoto Kuramaguchi Medical Center

    Kita, Kyoto, Japan

  • Maastricht Universitair Medisch Centrum

    Maastricht, Limburg, 6229HX, Netherlands

  • Manchester University NHS Foundation Trust

    Manchester, England, M139WL, United Kingdom

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905, United States

  • MedStar Georgetown University Hospital

    Washington D.C., District of Columbia, 20007, United States

  • Medizinische Universität

    Vienna, Austria

  • Medizinische Universität Wien

    Vienna, Austria

  • Nagoya City University Hospital

    Aichi, Japan

  • National Taiwan University Hospital

    Taipei City, Taipei, 10047, Taiwan

  • New York University Langone Hospital - Long Island

    New York, New York, 11501, United States

  • Odense Universitetshospital

    Odense, Region Syddanmark, 5000, Denmark

  • Paracelsus Medical University

    Salzburg, State of Salzburg, 5020, Austria

  • Paracelsus Medical University

    Salzburg, Austria

  • Perlmutter Cancer Center - 38th Street

    New York, New York, 10016, United States

  • Pitie-Salpêtrière Hospital

    Paris, Ill-de-France, 75013, France

  • Pusan National University Hospital

    Busan, 49241, South Korea

  • Rabin Medical Center - Beilinson Hospital

    Ashkelon, Southern District, 7830604, Israel

  • Rambam Medical Center

    Haifa, Haifa District, 3109601, Israel

  • Red Cross Medical Center

    Shibuya-ku, Tokyo, 150-8935, Japan

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263, United States

  • Royal Perth Hospital

    Perth, Western Australia, 6000, Australia

  • Royal Victoria Hospital

    Montreal, Quebec, H3A1A1, Canada

  • SESLHD: St George Hospital

    Kogarah, New South Wales, 2217, Australia

  • Samsung Medical Center

    Seoul, Seoul, 06351, South Korea

  • Semmelweis Egyetem - I. sz. Belgyógyászati Klinika

    Budapest, Budapest, 1087, Hungary

  • Seoul National University Hospital

    Seoul, 03080, South Korea

  • Severance Hospital

    Seoul, Seoul Teugbyeols, 120-752, South Korea

  • Shinshu University Hospital

    Matsumoto, Nagano, 390-0802, Japan

  • Smilow Cancer Hospital Care Center at Trumbull

    Trumbull, Connecticut, 06611, United States

  • Taipei Veterans General Hospital

    Taipei, Taipei, 11217, Taiwan

  • The Catholic University of Korea - Seoul St. Mary's Hospital

    Seoul, 06591, South Korea

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    High Heaton, England, NE77DN, United Kingdom

  • The Ohio State University College of Medicine

    Columbus, Ohio, 43210, United States

  • Theagenio Anti-Cancer Hospital of Thessaloniki

    Thessaloniki, Central Macedonia, 54007, Greece

  • Tom Baker Cancer Centre

    Calgary, Alberta, T2N4N2, Canada

  • Uniklinik Köln

    Köln, Baden-Wurttemberg, 50937, Germany

  • Universitair Medisch Centrum Groningen

    Groningen, Provincie Groningen, 9713GZ, Netherlands

  • University College London Hospitals

    London, England, NW12PG, United Kingdom

  • University Hospital Marqués de Valdecilla

    Santander, Cantabria, 39008, Spain

  • University Hospital of Patras

    Patra, Peloponnese, 26504, Greece

  • University Medicine Mainz

    Mainz, Rhineland-Palatinate, 55131, Germany

  • University of Nebraska Medical Center

    Omaha, Nebraska, 68105, United States

  • University of Tennessee Medical Center

    Knoxville, Tennessee, 37920, United States

  • University of Washington

    Seattle, Washington, 98109, United States

  • Universitätsklinikum Heidelberg

    Heidelberg, Baden-Wurttemberg, 69120, Germany

  • Vanderbilt - Ingram Cancer Center - Henry-Joyce Cancer Clinic

    Nashville, Tennessee, 37232, United States

  • Všeobecná fakultní nemocnice v Praze

    Prague, Prague, 12808, Czechia

  • Yale Cancer Center

    North Haven, Connecticut, 06473, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.