Ignite Creation Date:
2024-05-06 @ 3:02 PM
Last Modification Date:
2024-10-26 @ 1:42 PM
Study NCT ID:
NCT04504825
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2023-11-30
First Post:
2020-07-20
Brief Title:
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis
Sponsor:
Alexion Pharmaceuticals Inc
Organization:
Alexion Pharmaceuticals Inc
Study Overview
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 IIIb AL Amyloidosis
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 reduces cardiovascular related hospitalizations and it is safe and well tolerated in patients with stage IIIb AL amyloidosis
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 reduces cardiovascular related hospitalizations and it is safe and well tolerated in patients with stage IIIb 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 IIIb PCD treatment-naïve AL amyloidosis patients The primary evaluation treatment period PETP part of the study will stop when the last patient is randomized in the PETP plus 18 months Approximately 124 patients will be enrolled using a 21 randomization ratio Stratification will be based on geographic region across investigator sites The primary endpoint is a composite endpoint of all-cause mortality and frequency of cardiovascular hospitalizations Patients in both study intervention groups will be followed from randomization until death from any cause heart transplant left wall assist device LVAD implantation or until the end of study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None