AZD0120 for Light Chain Amyloidosis
(ALACRITY Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests a new treatment called AZD0120 for individuals with light chain (AL) amyloidosis. The main goal is to determine if AZD0120 is safe and effective for those with this condition, characterized by abnormal protein buildup in organs. The trial seeks participants diagnosed with AL amyloidosis, who have experienced organ involvement, and require additional therapy after trying at least one other treatment. Participants should not have other types of amyloidosis or severe symptoms that limit daily activities. As a Phase 1, Phase 2 trial, this research aims to understand how AZD0120 works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this new treatment.
Will I have to stop taking my current medications?
The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
Is there any evidence suggesting that AZD0120 is likely to be safe for humans?
Research shows that AZD0120 is being tested for safety and tolerance in people with light chain amyloidosis. As this is an early-stage study, detailed safety information from human trials is not yet available. However, AZD0120's progression to a Phase 1/2 study indicates promise in earlier tests, possibly in labs or with animals.
Early-stage trials like this primarily assess how well participants tolerate the treatment. Researchers closely monitor participants for any side effects. Those considering joining this trial can expect the research team to closely monitor their health to ensure safety.12345Why do researchers think this study treatment might be promising for amyloidosis?
AZD0120 is unique because it offers a fresh approach to treating Light Chain Amyloidosis by targeting the underlying disease process in a novel way. Unlike current treatments, which primarily focus on reducing the production of the amyloid light chains, AZD0120 acts directly to interfere with the deposition process of these harmful proteins. Researchers are excited about this treatment because it could slow or even stop the progression of organ damage, offering hope for patients who have limited options with existing therapies.
What evidence suggests that AZD0120 might be an effective treatment for light chain amyloidosis?
Research has shown that AZD0120, the investigational treatment in this trial, holds promise for treating light chain amyloidosis. One study found that AZD0120 improved the condition in 96% of patients. Impressively, 85% of these patients experienced a complete response, with their symptoms disappearing entirely. Additionally, many patients showed no detectable signs of the disease after treatment. These results suggest that AZD0120 could be a highly effective option for managing light chain amyloidosis.23567
Are You a Good Fit for This Trial?
This trial is for people who have relapsed or are not responding to treatment for AL amyloidosis, a condition where abnormal proteins build up in organs. Participants must have had at least one prior therapy, be relatively active and healthy (ECOG 0-1), and show measurable signs of the disease. Women able to bear children must test negative for pregnancy and agree to use birth control.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a weight-based dose of AZD0120, a CAR T cell therapy targeting CD19 and BCMA
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension (optional)
Participants may opt into continuation of treatment long-term
What Are the Treatments Tested in This Trial?
Interventions
- AZD0120
Find a Clinic Near You
Who Is Running the Clinical Trial?
Alexion Pharmaceuticals, Inc.
Lead Sponsor
Dr. Alberto R. Martinez
Alexion Pharmaceuticals, Inc.
Chief Medical Officer since 2010
MD from University of Sao Paulo
Marc Dunoyer
Alexion Pharmaceuticals, Inc.
Chief Executive Officer since 2021
PhD in Molecular Biology