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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-519674-40-00 | Other Identifier | EU CT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ALXN2350 | Experimental | Participants will receive one of three dose levels of ALXN2350 depending on the cohort. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ALXN2350 | Drug | ALXN2350 is a gene therapy product consisting of an AAV9 capsid containing BAG3 transgene. It is administered as a single intravenous (IV) infusion. |
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| Measure | Description | Time Frame |
|---|---|---|
| Part A: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | Baseline up to Week 78 |
| Measure | Description | Time Frame |
|---|---|---|
| Part B: Number of Participants With TEAEs and SAEs | Baseline up to Week 78 | |
| Parts A and B: Number of Participants With Cardiac Events at Weeks 52 and 78 | Weeks 52 and 78 | |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Alexion Pharmaceuticals, Inc. (Sponsor) | Contact | 1-855-752-2356 | clinicaltrials@alexion.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Recruiting | Birmingham | Alabama | 35233 | United States |
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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| Parts A and B: Time to the First Event of Death, Heart Transplant, Mechanical Circulating Support, or Aborted Sudden Cardiac Death |
| Baseline up to Week 78 |
| Research Site | Recruiting | Boston | Massachusetts | 02115 | United States |
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| Research Site | Recruiting | Cincinnati | Ohio | 45219 | United States |
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| Research Site | Recruiting | Portland | Oregon | 97239-3098 | United States |
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| Research Site | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
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| Research Site | Recruiting | Houston | Texas | 77030 | United States |
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| Research Site | Recruiting | Massa | 54100 | Italy |
| Research Site | Recruiting | Barcelona | 08035 | Spain |
| Research Site | Recruiting | Majadahonda | 28222 | Spain |
| Research Site | Recruiting | London | SW3 6NP | United Kingdom |