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
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.