A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis
ClinicalTrials.gov ID: NCT04512235
Sponsor: Alexion
Information provided by: Alexion (Responsible Party)
Last Update Posted: 2023-08-07
Brief Summary:
AL (or light chain) amyloidosis begins in the bone marrow where abnormal proteins misfold and create free light chains that cannot be broken down. These free light chains bind together to form amyloid fibrils that build up in the extracellular space of organs, affecting the kidneys, heart, liver, spleen, nervous system and digestive tract.
The primary purpose of this study is to determine whether CAEL-101, a monoclonal antibody that removes AL amyloid deposits from tissues and organs, improves overall survival and it is safe and well tolerated in patients with stage IIIa AL amyloidosis.
Detailed Description:
This is a double-blind, randomized, multicenter international Phase 3 study of CAEL-101 combined with standard of care (SoC) plasma cell dyscrasia (PCD) treatment versus placebo combined with SoC PCD treatment in Mayo stage IIIa PCD treatment-naïve AL amyloidosis patients. As this is an event-driven study, the study will enroll until at least 79 deaths have been observed.
Approximately 267 patients will be enrolled using a 2:1 randomization ratio. An interim analysis (IA) may also be performed when at least 75% of the events have been observed. Patients in both study intervention groups will be followed from randomization until death from any cause or until the end of study.
OFFICIAL TITLE
A Phase 3, Double-Blind, Multicenter 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 IIIa AL Amyloidosis
INTERVENTION / TREATMENT
Drug: CAEL-101
Other: Placebo
Drug: cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
| Category | Value |
|---|---|
| Study Start (Actual) | 2020-11-12 |
| Primary Completion (Estimated) | 2025-03-15 |
| Study Completion (Estimated) | 2025-03-15 |
| Enrollment (Estimated) | 267 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers |
CAEL101-302
|