BAG3 Mutation Associated Dilated CardiomyopathyPhase 1/2DCMRestoreNCT07218887
ALXN2350 in Adult Participants With BAG3-Associated Dilated Cardiomyopathy
A Phase 1/2 study testing ALXN2350 in people with BAG3 Mutation Associated Dilated Cardiomyopathy, run by Alexion Pharmaceuticals, Inc. at 11 sites in 4 countries.
Trial summary
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- BAG3 Mutation Associated Dilated Cardiomyopathy
- What is being tested?
- ALXN2350
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 11 sitesin 4 countries
- Study dates
- 24 Oct 2025to 27 Jan 2032
- Phase
- Phase 1/2
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Pathogenic or likely pathogenic mutation in BAG3
- Medical history of diagnosis of DCM
- Stable combination of HF SoC medications
- Adequate acoustic windows for echocardiography
- LVEF > 15% and ≤ 45% at Screening by echocardiography
- Diagnosis of chronic HF for at least 3 months
- NYHA Class I/II/III at Screening
Who cannot join
- Presence of antibodies to AAV9
- Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
- Decompensated HF
- Cardiac ventricular arrhythmia that requires treatment
- History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
- NYHA Class IV
- History of substance abuse that could contribute to cardiac dysfunction
- Note: Other inclusion and exclusion criteria may apply.
Trial locations and contacts
11 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
11 sites in 4 countries
United States7 sites, 7 recruiting
- Recruiting
Research Site
Birmingham, Alabama, 35233
Nehal Vekariya, 205-934-7173, nvekariya@uabmc.edu
- Recruiting
Research Site
Boston, Massachusetts, 02115
Alyx Vogle, 617-732-6320, avogle@bwh.harvard.edu
- Recruiting
Research Site
Cincinnati, Ohio, 45219
Sara Black, 513-585-1777, sara.black@thechristhospital.com
- Recruiting
Research Site
Houston, Texas, 77030
Jennifer Garrett, 346-238-4516
- Recruiting
Research Site
Palo Alto, California, 94304
Shannon Schultz, 650-498-9617, sks01@stanford.edu
- Recruiting
Research Site
Philadelphia, Pennsylvania, 19104
Joseph Puhl, 267-399-3049, joseph.puhl@pennmedicine.upenn.edu
- Recruiting
Research Site
Portland, Oregon, 97239-3098
Hailey Volk, 503-418-2246, volkh@ohsu.edu
Spain2 sites, 2 recruiting
- Recruiting
Research Site
Barcelona, 08035
Germán Escudero, +34 934896329 ext. 6267, german.escudero@vhir.org
- Recruiting
Research Site
Majadahonda, 28222
María de la Iglesia, +34 91 191 67 49, cardiopatiasfamiliares.hpth@salud.madrid.org
Italy1 site, 1 recruiting
- Recruiting
Research Site
Massa, 54100
Claudio Passino, +39 0503152191, passino@ftgm.it
United Kingdom1 site, 1 recruiting
- Recruiting
Research Site
London, SW3 6NP
Nikula Vicario, +44 0330 128 4043, Nikula.vicario@nhs.net
About this study
Official title: A Phase 1/2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
- Being tested
- ALXN2350
- Keywords
- ALXN2350, Gene Therapy, BAG3 Mutation Associated Dilated Cardiomyopathy
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALXN2350Drug
ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| ALXN2350 | Experimental | Participants will receive one of three dose levels of ALXN2350 depending on the cohort. Receives: Drug: ALXN2350 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Baseline up to Week 78
Secondary outcomes (4)
Part B: Number of Participants With TEAEs and SAEs
Measured: Baseline up to Week 78
Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78
Measured: Weeks 52 and 78
Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death
Measured: Baseline up to Week 78
CFB in cardiac serum biomarker NT-pro-BNP
Measured: Weeks 52 and 78
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2025
- Study start24 Oct 2025
- Primary completion27 Jan 2032 (estimated)
- Study completion27 Jan 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for BAG3 Mutation Associated Dilated CardiomyopathyStudies 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07218887
- Sponsor's protocol ID
- D7030C00001
- Other IDs
- 2024-519674-40-00
- Acronym
- DCMRestore
- Status last verified
- Aug 2026
- First submitted
- 17 Oct 2025
- Last updated
- 28 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- 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 28 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-Label, Multicenter, Dose Finding and Dose Expansion Study to Investigate the Safety, Tolerability, and Efficacy of ALXN2350 Gene Therapy in Adult Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
- ClinicalTrials.gov ID
- NCT07218887
- Acronym / sponsor ID
- DCMRestore / D7030C00001
- Keywords
- ALXN2350, Gene Therapy, BAG3 Mutation Associated Dilated Cardiomyopathy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Oct 2025 (actual)
- Primary completion
- 27 Jan 2032 (estimated)
- Study completion
- 27 Jan 2032 (estimated)
- First posted
- 20 Oct 2025 (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.
This Phase 1/2 study is an open-label, dose finding and dose expansion study investigating the safety, tolerability, and efficacy of a single IV infusion of ALXN2350 in adult participants with BAG3 associated DCM.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ALXN2350 | Experimental | Drug: ALXN2350Participants will receive one of three dose levels of ALXN2350 depending on the cohort. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Pathogenic or likely pathogenic mutation in BAG3
- Medical history of diagnosis of DCM
- Stable combination of HF SoC medications
- Adequate acoustic windows for echocardiography
- LVEF > 15% and ≤ 45% at Screening by echocardiography
Key exclusion criteria (8 listed)
- Presence of antibodies to AAV9
- Presence of a pathogenic or likely pathogenic variant in another gene where that other gene is authoritatively recognized as causal for DCM
- Decompensated HF
- Cardiac ventricular arrhythmia that requires treatment
- History of untreated clinically significant valve disease or a Screening confirmation of severe aortic stenosis, severe mitral stenosis, moderate or severe aortic insufficiency or severe mitral insufficiency
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ALXN2350ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion. | Drug | ALXN2350 |
Primary outcome
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Baseline up to Week 78
4 secondary outcomes
Part B: Number of Participants With TEAEs and SAEs
Time frame: Baseline up to Week 78
Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78
Time frame: Weeks 52 and 78
Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death
Time frame: Baseline up to Week 78
CFB in cardiac serum biomarker NT-pro-BNP
Time frame: Weeks 52 and 78
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 28 Aug 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.
Related trials for BAG3 Mutation Associated Dilated Cardiomyopathy
RecruitingPhase 1/2
An AAV Gene Therapy Trial of AFTX-201 in Adults With BAG3-Associated Dilated Cardiomyopathy (DCM)
Dilated Cardiomyopathy (DCM), BAG3 Mutation Associated Dilated Cardiomyopathy
RecruitingObservational study
Observational Study of Natural History of BAG3 Gene Mutation-Associated Dilated Cardiomyopathy in Chinese Adults
Dilated Cardiomyopathy
Active, not recruitingObservational study
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
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.
11 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ALXN2350 in people with BAG3 Mutation Associated Dilated Cardiomyopathy, run by Alexion Pharmaceuticals, Inc. at 11 sites in 4 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 7 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: looking for participants now, as of the sponsor's last update on 28 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Spain, Italy, United Kingdom. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Contact Alexion Pharmaceuticals, Inc. (Sponsor) (1-855-752-2356, clinicaltrials@alexion.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Baseline up to Week 78). 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 28 Aug 2026.