Refer a Patient
Have a Patient with AL Amyloidosis Who May Be Interested in the CARES Studies?
The CARES Studies are enrolling approximately 270 participants who have Mayo stage IIIa AL amyloidosis and 125 participants who have Mayo stage IIIb AL amyloidosis. The staging is based on the European modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement (Wechalekar, 2013; Dispenzieri, 2004).
Both of the CARES Studies are phase 3, double-blind studies. The total duration of participation in the study treatment period for each is at least 52 weeks or until the patient’s death. Both studies consist of a screening period, a study treatment period, and a follow-up period. The follow-up period will last until end of life or early withdrawal.
The investigational medication, CAEL-101, is a monoclonal antibody that is being given in combination with plasma cell dyscrasia (PCD) therapy and compared to PCD therapy on its own in treating patients with AL amyloidosis. Participants will be assigned at random to receive either CAEL-101 and PCD therapy or a placebo and PCD therapy. Neither the participant nor the study team will know which treatment option has been assigned, but in case of an emergency, they can quickly find out. The investigational medication will be administered as an IV infusion.
Primary Objective
The CARES Studies are determining if CAEL-101 and PCD therapy improves overall survival in Mayo stage IIIa and IIIb AL amyloidosis patients who have not received treatment, compared to PCD therapy alone.
Eligibility Criteria
Additional eligibility criteria apply.
Key Inclusion Criteria
- Be at least 18 years of age
- Have AL amyloidosis stage IIIa or IIIb based on the European modification of the 2004 Standard Mayo Clinic Staging in patients with advanced cardiac involvement (Wechalekar, 2013; Dispenzieri, 2004) at the time of screening
- Cardiac involvement as defined by:
- Documented clinical signs and symptoms supportive of a diagnosis of heart failure in the setting of a confirmed diagnosis of AL amyloidosis in the absence of an alternative explanation for heart failure
- AND
- At least 1 of the following:
- Endomyocardial biopsy demonstrating AL cardiac amyloidosis, or
- Echocardiogram demonstrating a mean left ventricle wall thickness (calculated as [IVSd+LPWd]/2) of > 12 mm at diastole in the absence of other causes (e.g., severe hypertension, aortic stenosis), which would adequately explain the degree of wall thickening, or
- Cardiac MRI with gadolinium contrast agent diagnostic of cardiac amyloidosis
Key Exclusion Criteria
- Have any other form of amyloidosis other than AL amyloidosis
- Received prior therapy for AL amyloidosis or multiple myeloma
- Have POEMS syndrome or multiple myeloma defined as clonal bone marrow plasma cells > 10% or biopsy-proven bony or extramedullary plasmacytoma AND any 1 or more of the following CRAB features:
- Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder, specifically:
- Hypercalcemia
- Renal insufficiency
- Anemia
- Bone lesions
- OR
- Any 1 of the following biomarkers or malignancy:
- 60% or greater clonal plasma cells on bone marrow examination
- More than 1 focal lesion on MRI that is at least 5 mm or greater in size
- Evidence of end organ damage that can be attributed to the underlying plasma cell proliferative disorder, specifically:
- Taking prednisone or its equivalent > 10 mg/day
- Taking doxycycline
- Receiving dialysis
- Have severe valvular stenosis (e.g., aortic or mitral stenosis with valve area < 1.0 cm2) or severe congenital heart disease
- Have history of sustained ventricular tachycardia or aborted ventricular fibrillation, or a history of atrioventricular nodal or sinoatrial nodal dysfunction, for which a pacemaker/implantable cardioverter-defibrillator (ICD) is indicated but not placed. (Patients who do have a pacemaker or ICD are allowed in the study.)