Treatments for Transthyretin Amyloidosis
(MaesTTRo Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial aims to understand how transthyretin (ATTR) amyloidosis affects people over time and how treatments, such as eplontersen (a new potential drug), perform in real-world settings. It examines different types of ATTR amyloidosis, including heart-related issues (ATTR-CM), nerve problems (ATTRv-PN), and mixed symptoms. The trial is best suited for individuals diagnosed with ATTR amyloidosis who are not currently participating in an interventional trial. Participants will contribute by sharing information about their health through electronic reports. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to the potential availability of a new treatment.
Do I need to stop my current medications for this trial?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What is the safety track record for eplontersen?
Research has shown that eplontersen is generally well-tolerated by patients. For those with hereditary polyneuropathy, safety data indicate that the side effects of eplontersen are similar to those of a placebo (a substance with no active drug), meaning the drug does not cause more issues than inactive treatments. Additionally, the FDA has approved eplontersen for treating polyneuropathy in hereditary transthyretin amyloidosis, supporting its safety for similar conditions.
For patients with transthyretin cardiomyopathy, studies have shown that eplontersen remains safe over time. No new safety concerns have emerged with long-term use, which is encouraging for those considering this treatment.
Overall, eplontersen appears to be a safe option for managing ATTR amyloidosis, whether in cases of cardiomyopathy, hereditary polyneuropathy, or a mixed form of the condition.12345Why are researchers excited about this trial?
Researchers are excited about this trial because it aims to provide a comprehensive understanding of transthyretin (ATTR) amyloidosis, specifically targeting different manifestations like ATTR cardiomyopathy (ATTR-CM), hereditary polyneuropathy (ATTRv-PN), and mixed ATTR amyloidosis phenotypes. Unlike treatments that focus solely on managing symptoms, this study seeks to gather real-world data from patients, which could lead to more personalized treatment approaches in the future. By observing patients across these different ATTR forms, researchers hope to identify patterns and insights that could improve diagnosis, management, and treatment strategies, potentially leading to breakthroughs that current standard treatments, such as tafamidis or patisiran, haven't achieved yet.
What evidence suggests that this trial's treatments could be effective for transthyretin amyloidosis?
Research has shown that eplontersen effectively treats hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). One study found that it significantly lowered transthyretin protein levels in the blood and improved nerve function, key indicators of disease progression. Eplontersen is the only approved treatment in the EU for ATTRv-PN and can be easily self-administered once a month. It works by turning off genes that produce harmful transthyretin proteins, targeting both mutated and normal forms. These encouraging results suggest that eplontersen could be a valuable option for managing transthyretin amyloidosis. Participants in this trial will be enrolled in different arms based on their specific condition, such as ATTR cardiomyopathy (ATTR-CM), hereditary polyneuropathy (ATTRv-PN), or a mixed ATTR amyloidosis phenotype.678910
Are You a Good Fit for This Trial?
Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Enrollment
Participants are enrolled in the study to collect real-world data on ATTR amyloidosis
Follow-up
Participants are monitored for treatment patterns and outcomes, with data collection every 6 months
What Are the Treatments Tested in This Trial?
Interventions
- Eplontersen
How Is the Trial Designed?
3
Treatment groups
Experimental Treatment
Patients with ATTRv-PN at enrollment
Patients with a mixed ATTR amyloidosis phenotype
Patients with ATTR-CM at enrollment
Find a Clinic Near You
Who Is Running the Clinical Trial?
AstraZeneca
Lead Sponsor
Sir Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Dr. Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
Pascal Soriot
AstraZeneca
Chief Executive Officer since 2012
Veterinary Medicine from École nationale vétérinaire d'Alfort, MBA from HEC Paris
Cristian Massacesi
AstraZeneca
Chief Medical Officer since 2021
MD from Marche Polytechnic University, Medical Oncology training at Royal Marsden Hospital, Kaplan Comprehensive Cancer Center, and European Institute of Oncology
ICON plc
Industry Sponsor
Dr. Steve Cutler
ICON plc
Chief Executive Officer since 2017
PhD from the University of Sydney, MBA from the University of Birmingham
Dr. Greg Licholai
ICON plc
Chief Medical Officer since 2023
Degrees from Harvard Business School, Yale School of Medicine, Columbia University, and Boston College
Citations
Non-interventional Study of Patients With Transthyretin ...
In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene ...
Eplontersen for Hereditary Transthyretin Amyloidosis With ...
Primary efficacy end points at week 65/66 were changes from baseline in serum transthyretin concentration, modified Neuropathy Impairment Score ...
3.
ir.ionis.com
ir.ionis.com/news-releases/news-release-details/wainzua-eplontersen-approved-eu-treatment-hereditaryWAINZUA (eplontersen) approved in the EU for ...
WAINZUA is the only approved medicine in the EU for the treatment of ATTRv-PN that can be self-administered monthly via an auto-injector.
Diagnosis and Management of Mixed Phenotype ...
This paper provides a review surrounding the diagnosis and management of mixed phenotype ATTRv amyloidosis, addressed through 3 clinical questions.
Non-interventional Study of Patients with Transthyretin ...
The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease.
6.
ir.ionis.com
ir.ionis.com/news-releases/news-release-details/eplontersen-granted-us-fda-fast-track-designation-patientsEplontersen granted U.S. FDA Fast Track designation for ...
The global CARDIO-TTRansform Phase 3 study of eplontersen in adults with ATTR-CM is fully enrolled with more than 1,400 patients – making it the largest study ...
NCT05667493 | An Extension Study to Assess Long-Term ...
The purpose of this study is to evaluate the safety and tolerability of extended dosing with eplontersen in participants with ATTR-CM.
8.
astrazeneca.com
astrazeneca.com/content/astraz/media-centre/press-releases/2023/eplontersen-demonstrated-sustained-benefit-in-phase-iii-trial.htmlEplontersen demonstrated sustained benefit in Phase III ...
TTR reductions were consistent with those reported at week 35. Eplontersen continued to demonstrate a safety and tolerability profile consistent ...
9.
bmccardiovascdisord.biomedcentral.com
bmccardiovascdisord.biomedcentral.com/articles/10.1186/s12872-025-04653-4a systematic review and meta-analysis of randomized trials
This meta-analysis highlights the potential of TTR-targeting therapies as an effective option for managing ATTR-CM, with significant improvements in survival.
Transthyretin amyloid cardiomyopathy: a paradigm for ...
Extended follow-up from the LTE study demonstrated a 41.2% relative risk reduction in mortality in patients initially treated with tafamidis 80 ...
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