#1 Clinical Trials Search in Europe

Alxn2220

Clinical trials are studying Alxn2220 in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), a heart condition caused by amyloid buildup. The trials are looking at safety, effectiveness, and how well re-treatment works. They include Phase 2 and Phase 3 studies in adult patients.

Table of contents

Trial overview

The available trial data show two interventional studies of Alxn2220 in adults with transthyretin amyloid cardiomyopathy (ATTR-CM), which is a heart disease linked to amyloid buildup.[1][2] Both studies are listed as authorised.[1][2]

One study is Phase 2 and includes 35 participants, while the other is Phase 3 and plans to enroll 1,000 participants.[1][2] Together, they focus on whether Alxn2220 can help adults with ATTR-CM and how safe it is to use in these study settings.[1][2]

Phase 2 study: re-treatment and disease markers

The Phase 2 trial is titled as a multicenter study of the effects of Alxn2220 on markers of disease severity and the safety of re-treatment in patients with ATTR-CM.[1] Multicenter means the study is being done at more than one site, which can help include a wider group of patients.[1]

This study aims to evaluate the pharmacodynamics of re-treatment, which means how the study treatment affects the body after it is given again.[1] The main outcomes are changes from baseline to Visit 16 in cMRI and scintigraphy, which are imaging tests used to look at the heart and amyloid-related findings.[1]

Phase 3 study: effectiveness and clinical events

The Phase 3 trial is designed to test whether Alxn2220 is effective, safe, and tolerable in adults with ATTR-CM.[2] It compares Alxn2220 with a placebo, which is an inactive product used as a comparison group.[2]

The brief summary says the study is assessing the difference between the Alxn2220 and placebo groups by looking at total occurrences of ACM and cardiovascular clinical events during the blinded treatment period.[2] In simple terms, the trial is checking whether people taking Alxn2220 have fewer serious heart-related events than people taking placebo.[2]

Who can participate

Based on the trial data, both studies are for adults with transthyretin amyloid cardiomyopathy (ATTR-CM).[1][2] No other eligibility details are provided in the source data, so the main known requirement is having this condition and being an adult.[1][2]

The Phase 2 study also focuses on patients who may receive re-treatment, so it is especially interested in people who have already had prior study treatment and are being studied again.[1]

Main outcomes being measured

The Phase 2 study measures change from baseline, which means the difference between the starting point and the later visit in the same patient.[1] Its main outcomes are cMRI and scintigraphy results at Visit 16.[1]

The Phase 3 study measures the total occurrence of all-cause mortality and cardiovascular clinical events during the blinded treatment period.[2] All-cause mortality means death from any cause, and cardiovascular events are serious problems related to the heart and blood vessels.[2]

Study design terms explained

Placebo is used in the Phase 3 study so researchers can compare Alxn2220 with a product that does not contain the active study treatment.[2] This helps show whether any differences in outcomes are likely due to the study treatment itself.[2]

Authorised means the study has been approved to move forward in the setting described in the source data.[1][2] Interventional means researchers are giving a treatment and then measuring the results.[1][2]

Trial IDPhaseCondition studiedStatusEnrollment
2025-520506-35-00Phase 2Adults with transthyretin amyloid cardiomyopathy (ATTR-CM)Authorised35
2023-506669-70-00Phase 3Adults with transthyretin amyloid cardiomyopathy (ATTR-CM)Authorised1000

FAQ

  • What is Alxn2220 being studied for? Alxn2220 is being studied in adults with transthyretin amyloid cardiomyopathy (ATTR-CM). The trials aim to see whether it can improve disease-related outcomes and whether it is safe and tolerable.
  • Who can take part in these trials? The studies are for adults with ATTR-CM. One trial also looks at patients who may receive re-treatment, so the target group is adults already living with this heart condition.
  • What phases are the Alxn2220 trials? The trial data include a Phase 2 study and a Phase 3 study. Phase 2 focuses on markers of disease severity and re-treatment safety, while Phase 3 looks at clinical events and overall effectiveness.
  • What outcomes are being measured? One study measures changes in cardiac MRI and scintigraphy, which are tests that show heart structure and amyloid activity. The other study measures total all-cause mortality and cardiovascular clinical events during the blinded treatment period.
  • Are these studies open to many participants? The Phase 2 study plans to enroll 35 people, and the Phase 3 study plans to enroll 1,000 people. Both studies are currently authorised.

Ongoing Clinical Trials on Alxn2220

  • Study on the Effects and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy

    Not recruiting

    1 1
    Investigated drugs:
    France Germany The Netherlands Spain

  • Study on the Effectiveness and Safety of ALXN2220 for Adults with Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

    Not recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark France Germany +8

Glossary

  • ATTR-CM: Short for transthyretin amyloid cardiomyopathy. This is a heart disease caused by amyloid protein buildup in the heart.
  • Transthyretin amyloid cardiomyopathy: A condition where abnormal protein deposits build up in the heart and can affect how well the heart works.
  • Phase 2: An earlier clinical trial phase that often looks at signs of benefit, safety, and the best way to continue treatment.
  • Phase 3: A later and larger trial phase that compares treatment with placebo or standard care and measures important health outcomes.
  • Interventional study: A study where researchers give a treatment and then measure what happens.
  • Placebo: An inactive product used for comparison, so researchers can see whether the study treatment works better.
  • Blinded treatment period: A study time when participants and sometimes researchers do not know who is receiving the study treatment, helping make results more reliable.
  • Cardiac MRI (cMRI): A heart scan that uses magnetic fields to create detailed pictures of the heart.
  • Scintigraphy: A scan test that uses a small amount of tracer to help show how much amyloid is in the body or heart.
  • Re-treatment: Giving the study treatment again after a previous treatment course.
  • Cardiovascular clinical events: Health problems related to the heart and blood vessels, such as serious heart-related outcomes measured in the trial.

References

  1. https://clinicaltrials.gov/study/2025-520506-35-00
  2. https://clinicaltrials.gov/study/2023-506669-70-00