CAEL-101 for Amyloidosis

Recruiting in Palo Alto (17 mi)

+317 other locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Alexion Pharmaceuticals

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?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.

Eligibility Criteria

This trial is for adults with stage IIIb AL amyloidosis, a condition where abnormal proteins build up in organs. Participants must be planning their first treatment with CyBorD regimen, have measurable hematologic disease, and cardiac involvement without prior therapy for AL amyloidosis or multiple myeloma. Women of childbearing potential and men must agree to use contraception.

Inclusion Criteria

I have heart issues due to AL amyloidosis, confirmed by tests.

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

I am a man who is either surgically sterile or will use effective birth control and not donate sperm during and up to 5 or 12 months after the study, depending on my treatment.

+4 more

Exclusion Criteria

I have a type of amyloidosis that is not AL amyloidosis.

My blood pressure is very low or drops significantly when I stand up, despite treatment.

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

+1 more

Participant Groups

The study tests CAEL-101's effectiveness and safety against placebo when added to the standard CyBorD regimen (cyclophosphamide, bortezomib, dexamethasone) in patients with advanced AL amyloidosis. It aims to see if CAEL-101 can improve survival by removing misfolded proteins from organs.

2Treatment groups

Experimental Treatment

Placebo Group

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

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. The study is divided into 2 parts, the Primary Evaluation Treatment period part and the Open-Label Extension period of Study.

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.