Cardiomyopathy, DilatedNCT05981092
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
An observational study of Cardiomyopathy, Dilated and Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM), run by Alexion Pharmaceuticals, Inc. at 15 sites in 5 countries.
Trial summary
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough blood for the body. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
- How many people
- 35 participants
- Where
- 15 sitesin 5 countries
- Study dates
- 14 Oct 2022to 12 Aug 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: primary care clinic and referrals from primary care clinic to investigational sites
Who may be able to join
- Documented BAG3 mutation that causes or is likely to cause dilated cardiomyopathy
- Heart failure Stage B-D, New York Heart Association (NYHA) Class I-IV
- Left Ventricular Ejection Fraction ≤50% (i.e., Reduced Heart Function)
Who cannot join
- Acute decompensated heart failure within 1 month prior to enrollment.(such as hospitalization)
- Any of the following within 3 months prior to screening: myocardial infarction (MI), cardiac surgical procedures (other than for pacemaker/ICD/CRT-defibrillator [CRT-D] implantation), acute coronary syndrome, or hospitalization for cardiac arrhythmia.
- History of heart transplantation
- eGFR <30 mL/min/1.73 m2 (significantly impaired kidney function)
- Noncardiac condition that limits lifespan to <1 year.
- Presence of other form(s) of cardiomyopathy contributing to heart failure
- Previous administration with an investigational drug within 30 days (or as determined by the local requirement).
- No more than 3 first-degree members of the same family who are already participating in the study
Trial locations and contacts
15 sites in 5 countries.
15 sites in 5 countries
United States6 sites
Research Site
Aurora, Colorado, 80045
Research Site
Boston, Massachusetts, 02115
Research Site
Palo Alto, California, 94305
Research Site
Philadelphia, Pennsylvania, 19104
Research Site
Rochester, Minnesota, 55905
Research Site
The Bronx, New York, 10467
Spain4 sites
Research Site
A Coruña, A Coruna, 15006
Research Site
El Palmar, Murcia, 30120
Research Site
Madrid, Madrid, 28041
Research Site
Majadahonda, Madrid, 28222
Netherlands2 sites
Research Site
Amsterdam, Amsterdam, 1105 AZ
Research Site
Maastricht, Maastricht, 6229 HX
United Kingdom2 sites
Research Site
Harefield, London, UB9 6JH
Research Site
London, London, EC1A 7BE
About this study
Official title: A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough blood for the body.
The study is seeking up to about 35 participants who have:
Read more
- BAG3 mutation (change in the gene) that causes or is likely to cause dilated cardiomyopathy
- NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)
- Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart function)
All participants in this study will receive their usual treatment. The investigators will observe the natural progression of people who have BAG3 DCM. This will help the investigators better understand the disease and aid in future research.
Participants will take part in this study for three years. During this time, participants will visit the site at least 8 times (about every 3 months for the first year and annually during year 2 and three). Participants will undergo study procedures and give information about their health. These procedures will include a physical exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer questions about health and quality of life. The study team will also call participants about 1 time over the phone.
- Keywords
- BAG3, DCM, cardiomyopathy, dilated cardiomyopathy, heritable heart disease, heart disease
- Medical subject headings
- Cardiomyopathy, Dilated, Cardiomyopathies, Heart Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| BAG3 DCM | A single arm observational trial where all participants will undergo the same schedule of assessments. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine baseline of cardiac structure and function in BAG3 associated DCM.
Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
Measured: Baseline
Determine changes over time in cardiac structure and function in BAG3 associated DCM.
Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of 3 years follow up time.
Measured: 3 years
Secondary outcomes (8)
Change from Baseline in high sensitivity troponin I (hsTNI) levels
HsTNI will be collected at baseline through 3 years to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNI to severity of clinical disease progression.
Measured: Baseline to year 3
Change from Baseline in high sensitivity troponin T (hsTNT) levels
HsTNT will be collected at baseline through 3 years to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNT to severity of clinical disease progression.
Measured: Baseline to year 3
Change from Baseline in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels
NT-proBNP will be collected at baseline through 3 years to observe the natural history (baseline and variance) of the biomarker and the correlation of NT-proBNP to severity of clinical disease progression.
Measured: Baseline to year 3
Change from baseline in Measures of Oxygen Capacity
Measured via Cardio Pulmonary Exercise Testing (CPET).
Measured: Baseline to year 3
Change from Baseline in Scores of Kansas City Cardiomyopathy Questionnaire (KCCQ) domains
Measured: Baseline to year 3
Change from Baseline in Scores of Patient Global Impression of Severity (PGI-S)
Measured: Baseline to year 3
Change from Baseline in Scores of Patient Global Impression of Change (PGI-C)
Measured: Baseline to year 3
Define the rate of cardiac status changes over time.
The frequency and rate of incidence of death, heart transplant, MCS, heart failure, hospitalization, appropriate implantable cardioverter defibrillator shock, or aborted SCD.
Measured: 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Aug 2023
- Study start14 Oct 2022
- Primary completion12 Aug 2027 (estimated)
- Study completion12 Aug 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cardiomyopathy, DilatedStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05981092
- Sponsor's protocol ID
- R0765C00001
- Other IDs
- C4981001, ALXN2350-DCM-501
- Status last verified
- Jun 2026
- First submitted
- 14 Jun 2023
- Last updated
- 12 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
Study at a glance
From the registry record, last updated by the sponsor on 12 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY
- ClinicalTrials.gov ID
- NCT05981092
- Acronym / sponsor ID
- R0765C00001
- Keywords
- BAG3, DCM, cardiomyopathy, dilated cardiomyopathy, heritable heart disease, heart disease
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Oct 2022 (actual)
- Primary completion
- 12 Aug 2027 (estimated)
- Study completion
- 12 Aug 2027 (estimated)
- First posted
- 8 Aug 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough blood for the body.
The study is seeking up to about 35 participants who have:
- BAG3 mutation (change in the gene) that causes or is likely to cause dilated cardiomyopathy
- NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)
- Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart function)
All participants in this study will receive their usual treatment. The investigators will observe the natural progression of people who have BAG3 DCM. This will help the investigators better understand the disease and aid in future research.
Participants will take part in this study for three years. During this time, participants will visit the site at least 8 times (about every 3 months for the first year and annually during year 2 and three). Participants will undergo study procedures and give information about their health. These procedures will include a physical exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer questions about health and quality of life. The study team will also call participants about 1 time over the phone.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BAG3 DCM | – | –A single arm observational trial where all participants will undergo the same schedule of assessments. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- primary care clinic and referrals from primary care clinic to investigational sites
Key inclusion criteria (3 listed)
- Documented BAG3 mutation that causes or is likely to cause dilated cardiomyopathy
- Heart failure Stage B-D, New York Heart Association (NYHA) Class I-IV
- Left Ventricular Ejection Fraction ≤50% (i.e., Reduced Heart Function)
Key exclusion criteria (8 listed)
- Acute decompensated heart failure within 1 month prior to enrollment.(such as hospitalization)
- Any of the following within 3 months prior to screening: myocardial infarction (MI), cardiac surgical procedures (other than for pacemaker/ICD/CRT-defibrillator [CRT-D] implantation), acute coronary syndrome, or hospitalization for cardiac arrhythmia.
- History of heart transplantation
- eGFR <30 mL/min/1.73 m2 (significantly impaired kidney function)
- Noncardiac condition that limits lifespan to <1 year.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Determine baseline of cardiac structure and function in BAG3 associated DCM.
Time frame: Baseline
Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
Determine changes over time in cardiac structure and function in BAG3 associated DCM.
Time frame: 3 years
Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of 3 years follow up time.
8 secondary outcomes
Change from Baseline in high sensitivity troponin I (hsTNI) levels
Time frame: Baseline to year 3
Change from Baseline in high sensitivity troponin T (hsTNT) levels
Time frame: Baseline to year 3
Change from Baseline in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels
Time frame: Baseline to year 3
Change from baseline in Measures of Oxygen Capacity
Time frame: Baseline to year 3
Change from Baseline in Scores of Kansas City Cardiomyopathy Questionnaire (KCCQ) domains
Time frame: Baseline to year 3
Change from Baseline in Scores of Patient Global Impression of Severity (PGI-S)
Time frame: Baseline to year 3
Change from Baseline in Scores of Patient Global Impression of Change (PGI-C)
Time frame: Baseline to year 3
Define the rate of cardiac status changes over time.
Time frame: 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Jun 2026.
- Individual participant dataSharing planned: Yes. Alexion has a public commitment to allow requests for access to study data and will be supplying a protocol, CSR, and plain language summaries.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cardiomyopathy, Dilated and Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM), run by Alexion Pharmaceuticals, Inc. at 15 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 12 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, Spain, Netherlands, United Kingdom, Poland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determine baseline of cardiac structure and function in BAG3 associated DCM. (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Jun 2026.