Purpose

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

Condition

Eligibility

Eligible Ages
Over 18 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • 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 >0.03 ng/mL and dFLC ≥18 mg/dL - Planned first-line chemotherapy contains bortezomib administered subcutaneously weekly.

Exclusion Criteria

  • Non-AL amyloidosis. - NT-proBNP >8500 pg/mL. - Meets the International Myeloma Working Group (IMWG) definition of multiple myeloma - 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

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Birtamimab plus Standard of Care Chemotherapy
Intravenous administration of 24 mg/kg birtamimab every 28 days. Drug: Standard of Care Chemotherapy. Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD) according to the institutional standard of care. Initiation of daratumumab at randomization allowed at the discretion of the investigator
  • Drug: Birtamimab
    Intravenous administration of 24 mg/kg birtamimab every 28 days
  • Drug: Standard of Care Chemotherapy
    Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD)) according to the institutional standard of care
Placebo Comparator
Placebo plus Standard of Care Chemotherapy
Intravenous 0.9% Saline administration as a placebo every 28 days. Drug: Standard of Care Chemotherapy. Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD) according to the institutional standard of care. Initiation of daratumumab at randomization allowed at the discretion of the investigator
  • Other: Placebo
    Intravenous 0.9% Saline administration as a placebo every 28 days
  • Drug: Standard of Care Chemotherapy
    Bortezomib-containing chemotherapy regimen (e.g. cyclophosphamide, bortezomib, and dexamethasone (CyBorD)) according to the institutional standard of care

Recruiting Locations

Boston University School of Med.
Boston, Massachusetts 02118

More Details

Status
Recruiting
Sponsor
Prothena Biosciences Ltd.

Study Contact

Valerie Palma
650-837-8550
AFFIRM-ALClinicalTrial@prothena.com

Detailed 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. The primary objective 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. Approximately 150 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.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.