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NCT04512235 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)
A Phase 3 study of AL Amyloidosis, sponsored by Alexion Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 281
- Enrollment target
- 20
- Study locations
NCT04512235: Active Phase 3 study of AL Amyloidosis, sponsored by Alexion Pharmaceuticals.
NCT04512235 is a Phase 3 study of AL Amyloidosis that is active but no longer recruiting, run by Alexion Pharmaceuticals. The registered enrollment target is 281 participants, below the 406-participant average among 15 other AL Amyloidosis trials with a reported enrollment target (31% lower). The trial reports 20 study locations across 12 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04512235, a Phase 3 study of AL Amyloidosis, is active but no longer recruiting, sponsored by Alexion Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 281 participants
- Enrollment target
- 20
- Study locations
Study 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 IIIa AL amyloidosis.
Primary Outcome
Time to all-cause mortality was defined as the number of weeks from the date of randomization to the date of death if it is on or before the end of PETP. Participants alive at the end of PETP (LPI+18 months) were censored at their last known alive date recorded within the PETP. CVHs were events adjudicated and confirmed as such by the Clinical Event Adjudication Committee (CEAC). Hypothesis testing with the Finkelstein-Schoenfeld test and treatment effect estimation with the win-ratio method bas
Conditions Studied
Interventions
- OTHER Placebo
- DRUG CAEL-101
- DRUG cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen
Study Locations (20)
California
- Research Site - Duarte
- Research Site - Palo Alto
- Research Site - San Francisco
Massachusetts
- Research Site - Boston
- Research Site - Boston
- Research Site - Boston
New York
- Research Site - New York
- Research Site - New York
- Research Site - Rochester
Florida
- Research Site - Jacksonville
- Research Site - Weston
North Carolina
- Research Site - Chapel Hill
- Research Site - Durham
Arizona
- Research Site - Scottsdale
Indiana
- Research Site - Indianapolis
Louisiana
- Research Site - New Orleans
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 281 participants |
| Start Date | 2020-11-03 |
| Est. Completion | 2027-04-08 |
| Phase | Phase 3 |
What the registry record for NCT04512235 still lists
NCT04512235 is an interventional study that assigns participants to a tested intervention. The registered 281 participants enrollment target is mid-sized for trials with a published cap, below the 406-participant average among 15 other AL Amyloidosis trials with a reported enrollment target (31% lower).
The record links to 1 condition, with AL Amyloidosis appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT04512235 names 20 study sites across 12 states, led by California, Massachusetts, New York.
Frequently Asked Questions
What is clinical trial NCT04512235 about?
NCT04512235 is a clinical study titled "A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIa AL Amyloidosis (CARES)". 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, sp...
What is the current status of trial NCT04512235?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 281 participants. The study started on 2020-11-03. Estimated completion is 2027-04-08.
What conditions does trial NCT04512235 study?
This clinical trial studies the following conditions: AL Amyloidosis.
What interventions are being tested in trial NCT04512235?
The interventions under investigation include: Placebo (OTHER), CAEL-101 (DRUG), cyclophosphamide, bortezomib, and dexamethasone (CyBorD) regimen (DRUG).
Who is sponsoring clinical trial NCT04512235?
This trial is sponsored by Alexion Pharmaceuticals, which has 162 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04512235 being conducted?
This trial has 20 study locations across Arizona, California, Florida, Indiana, Louisiana. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for AL Amyloidosis
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT04512235's enrollment target sits among peer trials
281 3rd of 15 higher than 13 of 15 other AL Amyloidosis trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other AL Amyloidosis trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
Comparing Dara-VCD Chemotherapy Plus Stem Cell Transplant to Dara-VCD Chemotherapy Alone for People Who Have Newly Diagnosed AL Amyloidosis
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-
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A Study to Evaluate the Efficacy and Safety of CAEL-101 in Patients With Mayo Stage IIIb AL Amyloidosis (CARES)
ACTIVE NOT RECRUITING · Phase 3
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-
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