CAEL-101 for Amyloidosis

Recruiting in Palo Alto (17 mi)

+327 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Alexion

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing CAEL-101, a special medicine, in patients with severe AL amyloidosis. The goal is to see if it helps clear harmful protein deposits from their organs and improves their survival. The study will compare CAEL-101 with standard treatments.

Eligibility Criteria

This trial is for patients with Mayo Stage IIIa AL Amyloidosis, a condition where abnormal proteins build up in organs. Participants must have heart involvement, measurable hematologic disease, and agree to use effective contraception. Those with other amyloidosis types or prior treatments for AL amyloidosis (except limited CyBorD regimen) are excluded.

Inclusion Criteria

Patients must be at least 18 years of age

My diagnosis of amyloidosis was confirmed with specific tests on tissue samples.

Women of childbearing potential (WOCBP) must have a negative pregnancy test during Screening and must agree to use highly effective contraception from Screening to at least 5 months following the last study drug administration or 12 months following the last dose of her PCD therapy, whichever is longer

+5 more

Exclusion Criteria

I have POEMS syndrome or multiple myeloma with specific symptoms or test results.

My condition is not AL amyloidosis.

I have been treated for AL amyloidosis or multiple myeloma, but only briefly used a specific treatment regimen.

+1 more

Participant Groups

The study tests CAEL-101's effectiveness and safety against placebo when added to the standard CyBorD treatment regimen. CAEL-101 is an antibody designed to clear out harmful protein deposits from tissues in patients with advanced cardiac AL Amyloidosis.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: CAEL-101 combined with SoC plasma cell dyscrasiaExperimental Treatment2 Interventions

The study is divided into 2 parts, the Primary Study and the Open-Label Extension Study. CAEL-101 is administered as an intravenous (IV) infusion over approximately 2 hours. It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months.

Group II: Placebo combined with SoC plasma cell dyscrasiaPlacebo Group2 Interventions

Patients randomized to receive placebo will receive 0.9% normal saline in an equivalent volume to a CAEL-101 infusion (approximately 250 cc). It is planned that all patients will continue their double-blind treatment until the last patient is randomized in the study plus 18 months.