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The primary purpose of the study is to evaluate the safety and tolerability of single intravenous (IV) doses of PGN-EDODM1 administered to participants with Myotonic Dystrophy Type 1 (DM1).
The study consists of 2 periods: A Screening Period (up to 30 days) and a Treatment and Observation Period (16 weeks).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PGN-EDODM1 | Experimental | PGN-EDODM1 for infusion |
|
| Placebo | Placebo Comparator | 0.9% NaCl |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PGN-EDODM1 for infusion | Drug | Single dose of PGN-EDODM1 by intravenous (IV) infusion |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with Adverse Events, Serious Adverse Events, with abnormal Clinical Laboratory tests, abnormal ECGs, and abnormal Vital Signs | Baseline to Week 16 |
| Measure | Description | Time Frame |
|---|---|---|
| Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1 | Baseline up to Day 3 | |
| Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1 | Baseline up to Day 3 | |
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Inclusion Criteria:
Exclusion Criteria:
Note: Other inclusion and exclusion criteria may apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCI Center for Clinical Research | Irvine | California | 92697 | United States | ||
| Stanford University |
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| Placebo |
| Other |
Administered by IV infusion |
|
| Apparent Terminal Half-Life (t½) of PGN-EDODM1 |
| Baseline up to Day 3 |
| Area Under the Concentration-time Curve of PGN-EDODM1 | Baseline up to Day 3 |
| Palo Alto |
| California |
| 94304 |
| United States |
| Rare Disease Research | Atlanta | Georgia | 30329 | United States |
| University of Kansas Medical Center | Fairway | Kansas | 66205 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| University of Rochester Medical Center | Rochester | New York | 14642 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23298 | United States |
| University of Calgary | Calgary | Alberta | T3M 1M4 | Canada |
| Ottawa Hospital Research Institute (OHRI) | Ottawa | Ontario | Canada |
| CIUSSS du Saguenay-Lac-Saint-Jean | Chicoutimi | Quebec | Canada |
| University College London Hospital | London | UK | NW1 2PG | United Kingdom |
| Salford Royal Hospital | Salford | United Kingdom |
| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| D009136 | Muscular Dystrophies |
| D030342 | Genetic Diseases, Inborn |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D009140 | Musculoskeletal Diseases |
| D020967 | Myotonic Disorders |
| D020966 | Muscular Disorders, Atrophic |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009135 | Muscular Diseases |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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