Monoclonal Antibodies

CAEL-101 for AL Amyloidosis

Cleveland Clinic Amyloidosis Center, Cleveland, OH
CAEL-101 +2 morePhase 3RecruitingResearch Sponsored by Alexion

Study Summary

This trial is testing a drug to see if it can improve overall survival for people with stage IIIa AL amyloidosis, a disease where abnormal proteins build up in organs.

Eligible Conditions
  • AL Amyloidosis

Eligibility Criteria

Inclusion Criteria

You will be eligible if you check “Yes” for the criteria below
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You must have at least one of the following:
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You have a heart condition that may affect your ability to participate in the study.
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You have signs and symptoms of heart failure along with a confirmed diagnosis of AL amyloidosis, and there is no other reason for your heart failure.
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The planned initial treatment for your plasma cell disorder involves a combination of medications called cyclophosphamide, bortezomib, and dexamethasone, which is the standard of care.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~50 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 50 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of patients with treatment emergent adverse events as assessed by CTCAE v5.0
Time from the date of randomization to date of death or end of study
Secondary outcome measures
Cardiac Improvement by Global Longitudinal Strain (GLS%)
Change in distance walked (in meters) during a six-minute walk test
Quality of Life (QOL) by the Kansas City Cardiomyopathy Questionnaire-Overall Score (KCCQ-OS)
+1 more
Other outcome measures
24-hour Urine Protein Measure
Assessment of limitation during physical activity
Changes in Amyloid Load of the Heart, Liver and Spleen
+13 more

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.
Approved for 60 Other Conditions
This treatment demonstrated efficacy for 60 other conditions.

Trial Design

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. The minimum planned treatment time for each patient will be at least 50 weeks or until the patient's death. It is planned that all patients will continue their double-blind treatment until the last patient completes at least 50 weeks of treatment. As this is an event driven study, the study will continue, and all patients will continue to receive study treatment until at least 79 deaths have been observed.
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). The minimum planned treatment time for each patient will be at least 50 weeks or until the patient's death. It is planned that all patients will continue their double-blind treatment until the last patient completes at least 50 weeks of treatment. As this is an event driven study, the study will continue, and all patients will continue to receive study treatment until at least 79 deaths have been observed.

Find a site

Who is running the clinical trial?

AlexionLead Sponsor
241 Previous Clinical Trials
39,124 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
AstraZenecaIndustry Sponsor
4,118 Previous Clinical Trials
336,391,938 Total Patients Enrolled
1 Trials studying AL Amyloidosis
124 Patients Enrolled for AL Amyloidosis
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,512 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
Caelum BiosciencesLead Sponsor
2 Previous Clinical Trials
149 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
Caelum Biosciences, Inc.Lead Sponsor
2 Previous Clinical Trials
149 Total Patients Enrolled
2 Trials studying AL Amyloidosis
149 Patients Enrolled for AL Amyloidosis
Masood Sadaat, MD, MSc, ACRP-CPStudy Director
Alexion, AstraZeneca Rare Disease
Cristina C Quarta, MD, PhDStudy Director
Alexion, AstraZeneca Rare Disease
1 Previous Clinical Trials
124 Total Patients Enrolled
1 Trials studying AL Amyloidosis
124 Patients Enrolled for AL Amyloidosis

Media Library

CAEL-101 (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04512235 — Phase 3
AL Amyloidosis Research Study Groups: CAEL-101 combined with SoC plasma cell dyscrasia, Placebo combined with SoC plasma cell dyscrasia
AL Amyloidosis Clinical Trial 2023: CAEL-101 Highlights & Side Effects. Trial Name: NCT04512235 — Phase 3
CAEL-101 (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04512235 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there other similar testing procedures for CAEL-101?

"Right now, there are over 1300 trials researching CAEL-101 with almost 300 of those in Phase 3. Most of the testing for CAEL-101 is happening in Philadelphia, but there are 43000+ locations running studies related to CAEL-101."

Answered by AI

Does this trial have a lot of locations in North America?

"67 medical centres across the United States are participating in this clinical trial, including renowned institutions such as Indiana University School of Medicine Amyloid Research and Treatment, Medical College of Wisconsin, and Memorial Sloan Kettering Cancer Center."

Answered by AI

What are the conditions that CAEL-101 is designed to address?

"Oftentimes, CAEL-101 is used to ameliorate synovitis. Additionally, it has shown efficacy in treating ophthalmia, cancerous growths in the lungs, and branch retinal vein occlusion."

Answered by AI

Has CAEL-101 received permission from the FDA to be used?

"CAEL-101 is safe according to our team's estimation. This is a Phase 3 trial, which means that efficacy has been supported by some data and safety has multiple rounds of supportive data."

Answered by AI

Who else is applying?

What state do they live in?
Florida
How old are they?
18 - 65
What site did they apply to?
Hospital of the University of Pennsylvania, Perelman Center for Advanced Medicine
Clinical Trial Site
What portion of applicants met pre-screening criteria?
Met criteria
~87 spots leftby Mar 2025