At a glance
ClinicalIndex Comparison Record- ✓AL amyloidosis stage IIIa (European Modification of 2004 Mayo Clinic Staging) with NT-proBNP >650 ng/L at screening
- ✓Measurable hematologic disease: dFLC >4 mg/dL, or iFLC >4 mg/dL with abnormal Kappa/Lambda ratio, or SPEP m-spike >0.5 g/dL
- ✓Histopathologic diagnosis of amyloidosis by Congo red staining with green bi-refringence AND confirmation of AL deposits by immunohistochemistry/immunofluorescence, mass spectrometry, or electron microscopy
- ✓Documented cardiac involvement: clinical heart failure signs/symptoms plus either endomyocardial biopsy showing AL cardiac amyloidosis, echocardiogram with mean LV wall thickness >12 mm, or cardiac MRI with gadolinium confirming amyloidosis
- ✕Any form of amyloidosis other than AL amyloidosis
- ✕Prior therapy for AL amyloidosis or multiple myeloma (except up to 2 weeks of CyBorD-based therapy after screening labs but before randomization)
- ✕POEMS syndrome or multiple myeloma (>10% clonal bone marrow plasma cells or biopsy-proven plasmacytoma) with CRAB features or malignancy biomarkers (≥60% clonal plasma cells or >1 focal MRI lesion ≥5 mm)
- ✕Supine systolic blood pressure <90 mmHg or symptomatic orthostatic hypotension (>30 mmHg decrease on standing) despite medical management
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 3 clinical trial evaluating CAEL-101, Placebo, and 1 other intervention for AL Amyloidosis. Active but no longer recruiting, targeting 281 participants across 110 sites in 19 countries.
Signals
Detailed 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.
Study Details
Timeline
Arms & 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.
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.
Interventions
The investigational product, CAEL-101, is formulated as a sterile liquid solution of protein plus excipients for dilution in a single-use, stoppered, glass vial. Each 10 mL vial contains 300 mg of CAEL-101 at a concentration of 30 mg/mL. CAEL-101 will be diluted with commercially available 0.9% Normal Saline.
Commercially available 0.9% Normal Saline will be used as the placebo.
According to institutional standard of care.