About the CARES Studies for AL (Light Chain) Amyloidosis
The CARES Studies are evaluating the effectiveness and safety of an investigational medication in adults who have AL amyloidosis.
Who May Qualify
Eligible participants must:
- Be at least 18 years of age
- Have a diagnosis of Mayo stage IIIa or IIIb AL amyloidosis
- Not be pregnant or breastfeeding
- Not have any other form of amyloidosis other than AL amyloidosis
- Not have received prior therapy for AL amyloidosis or multiple myeloma
There are additional eligibility requirements that the study doctor can explain to you.
About the Investigational Medication
The investigational medication is called CAEL-101. “Investigational” means that it has not been approved by country-specific regulatory health authorities, such as the Food and Drug Administration (FDA), to be used for AL amyloidosis, and its use is being allowed for research purposes only. Participants will be assigned at random to receive either CAEL-101 and plasma cell dyscrasia (PCD) therapy or a placebo and PCD therapy. (A placebo looks just like the investigational medication but contains no active ingredients.) 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 intravenous (IV) infusion.
Study Participation
The total duration of participation in the study treatment period for the CARES Studies will be at least 52 weeks and consists of:
Screening
This period lasts up to 28 days. During this time, the study staff reviews a potential participant’s medical history and performs tests to see if they qualify for one of the studies.
Study Treatment
Participants are selected at random to receive either the investigational medication and PCD therapy or a placebo and PCD therapy. The study doctor performs and monitors procedures and assessments during study site visits to check on participants’ health. Participants will receive their assigned study treatment every seven days for the first four infusions, then every 14 days following for at least one year.
Follow-Up
The study doctor will continue to monitor participants’ health every 12 weeks after the last visit of the study treatment period.
Throughout the study, the study doctor will perform a number of tests and procedures, including but not limited to:
Medical history review
Physical exam
Vital sign measurement
Electrocardiogram (ECG)
Pregnancy test (if applicable)
Blood and urine sample collections