A Study to Evaluate the Effectiveness and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis

Titre officiel

A Phase 3, Double-Blind, Multicentre Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment Naïve Patients With Mayo Stage IIIb AL Amyloidosis

Sommaire:

L’amyloïdose à chaînes légères (AL) commence dans la moelle osseuse, où des protéines anormales se replient mal et créent des chaînes légères libres qui ne peuvent être décomposées. Ces chaînes légères libres se lient entre elles pour former des fibrilles amyloïdes qui s’accumulent dans l’espace extracellulaire des organes, touchant les reins, le cœur, le foie, la rate, le système nerveux et le tube digestif. L’objectif principal de cette étude est de déterminer si le CAEL-101 améliore la survie globale des patients atteints d’amyloïdose AL cardiaque.

Description de l'essai

Primary Outcome:

• Time from the date of randomization to date of death or end of study.
• Number of patients with treatment emergent adverse events as assessed by CTCAE v5.0

Secondary Outcome:

• Change in distance walked (in meters) during a six-minute walk test
• Quality of Life (QOL) by the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
• Quality of Life (QOL) by the Short Form-36 (SF-36) v2 Physical Component Score (PCS)
• Cardiac Improvement by Global Longitudinal Strain (GLS%)

This is a double-blind, randomized, multicentre international Phase 3 study of CAEL-101 combined with the standard of care (SoC) for plasma cell dyscrasia (PCD) versus placebo combined with standard of care PCD treatment in patients with Mayo stage IIIb AL amyloidosis that have not received prior treatment. The minimum planned treatment time for each patient will be at least 50 weeks or until the patient's death. It is planned that all patients will continue their double-blind treatment until the last patient completes at least 50 weeks of treatment.

Approximately 111 patients will be enrolled using a 2:1 randomization ratio of CAEL-101: placebo and will involve approximately 100 investigator sites.

The primary objective of this study is to assess the effects of CAEL-101 versus placebo on all-cause mortality.

Voir cet essai sur ClinicalTrials.gov