A study to assess the efficacy and safety of ALXN2220 in participants with transthyretin amyloid cardiomyopathy (ATTR-CM)
Summary description of the study
This study will investigate the investigational substance ALXN2220 (a so-called amyloid-reducing agent) in patients with transthyretin amyloidosis (ATTR, deposits of misfolded proteins) with cardiomyopathy (CM, disease of the heart muscle), referred to as ATTR-CM. The aim is to determine how effective and safe ALXN2220 is. Patients with ATTR-CM experience symptoms of heart muscle weakness (e.g., shortness of breath or difficulty exercising) and many die within a few years of the disease. Current treatment options for ATTR-CM include TTR stabilizers (medications that reduce deposits of misfolded proteins in the heart) and therapies to treat the symptoms of heart muscle weakness (e.g., diuretics, medications that promote urine production and its excretion). However, there is currently no treatment that can remove already deposited misfolded proteins that are present in the heart. Therefore, the only option for patients with advanced ATTR-CM may be a heart transplant. ALXN2220 works differently than the currently available treatment options. ALXN binds to the deposited misfolded proteins and activates the immune system, hoping that these will be removed from the heart and other organs, and that patients will experience an improvement in their heart muscle weakness and other symptoms. About 1000 women and men aged between 18 and 90 years will participate in this study worldwide, including about 10 from Switzerland.
(BASEC)
Intervention under investigation
All participants will be randomly assigned to a treatment in a 2:1 ratio.
The probability of receiving the investigational substance ALXN2220 is 2 out of 3 (approximately 66%). The probability of receiving the placebo (a placebo looks like the experimental drug but does not contain the active ingredient in the investigational substance) is 1 out of 3 (approximately 33%).
Before treatment with ALXN2220 or the placebo can begin, a screening will be conducted to verify that all inclusion criteria for participation in the study are met.
Participation in this study will last 2-4 years with visit appointments approximately every 4 weeks. Each visit appointment may vary in duration, from a short visit of about 4 hours to about 6 hours for longer visits.
During these visits, ALXN2220 (experimental group) or the placebo (control group) will be administered by infusion (controlled administration or the introduction of larger volumes of fluid into the body).
(BASEC)
Disease under investigation
Adults with transthyretin amyloidosis with cardiomyopathy (ATTR-CM)
(BASEC)
- Age ≥ 18 and ≤ 90 years - Men or women (not pregnant) - Confirmed diagnosis of ATTR-CM - Treatment with a diuretic (medications that promote urine production and its excretion) for at least 30 days prior to the screening - Life expectancy of ≥ 6 months according to the judgment of the investigator (BASEC)
Exclusion criteria
- History of bone marrow cancer or organ transplantation - Cardiomyopathy (disease of the heart muscle) not caused by ATTR-CM - Cardiovascular diseases - Hypertension - Arrhythmia - Liver or gallbladder disease - Malignant tumors or leukemia - Respiratory failure - Kidney failure - Disorders of the brain or spinal cord requiring the use of a wheelchair - Weight < 40 at screening (BASEC)
Trial sites
Basel, Bern, Luzern, St. Gallen, Zurich
(BASEC)
Sponsor
AstraZeneca AG, Switzerland
(BASEC)
Contact
Contact Person Switzerland
Dr. Eric Nawrotzky
+49 40809034207
eric.nawrotzky@clutterastrazeneca.comAstraZeneca GmbH
(BASEC)
Scientific Information
not available
Name of the authorising ethics committee (for multicentre studies, only the lead committee)
Ethics Committee Zurich
(BASEC)
Date of authorisation
19.04.2024
(BASEC)
ICTRP Trial ID
not available
Official title (approved by ethics committee)
DepleTTR-CM A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Amyloid Depleter ALXN2220 in Adult Participants with Transthyretin Amyloid Cardiomyopathy (ATTR-CM) (BASEC)
Academic title
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Public title
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Disease under investigation
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Intervention under investigation
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Type of trial
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Trial design
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Inclusion/Exclusion criteria
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Primary and secondary end points
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Registration date
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Incorporation of the first participant
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Secondary sponsors
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Additional contacts
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Secondary trial IDs
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Results-Individual Participant Data (IPD)
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Further information on the trial
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Results of the trial
Results summary
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Link to the results in the primary register
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