data de início: 30 de agosto de 2021
conclusão estimada: junho de 2027
Ultima atualização: 21 de fevereiro de 2024
fase de desenvolvimento:
Fase 3
tamanho / inscrição: 150
descrição do estudo: 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 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.
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.
desfechos primários:
- Time to all-cause mortality for the Double Blind Phase
Comparison of time to all-cause mortality for birtamimab and placebo control.
- Time from the first dose of study drug until the pre-defined number of events (all-cause mortality) have been reached.
resultados secundários:
- 6MWT distance for the Double Blind Phase
Mês 9
- Physical Component Summary (PCS) score of the Short Form-36, version 2 for the Double Blind Phase
Mês 9
critério de inclusão:
• Idades elegíveis: 18+
• Sexos elegíveis: todos
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
Diagnóstico confirmado de amiloidose AL
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
Chave
critério de exclusão: Criteria for Double-blind Phase:
Amiloidose não AL
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
Estenose valvar grave (por exemplo, estenose aórtica ou mitral com área valvar <1.0 cm2) ou doença cardíaca congênita grave
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
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