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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-506028-10-00 | Other Identifier | EUCT Number |
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The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple doses in participants with DM1.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Part A: Single Ascending Dose | Experimental | Participants will be randomized to receive a single dose of different dose levels of VX-670. |
|
| Part A: Placebo | Placebo Comparator | Participants will be randomized to receive single dose of placebo matched to VX-670. |
|
| Part B: Single and Multiple Ascending Dose | Experimental | Participants will be randomized to receive single and multiple doses of different dose levels of VX-670. The dose levels will be determined based on the data from Part A. |
|
| Part B: Placebo | Placebo Comparator | Participants will be randomized to receive single or multiple doses of placebo matched to VX-670. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| VX-670 | Drug | Solution for intravenous administration. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) | Part A: From Baseline up to Day 42; Part B: From Baseline and up to Day 168 |
| Measure | Description | Time Frame |
|---|---|---|
| Part A: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma | From Day 1 up to Day 42 | |
| Part A: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma | From Day 1 up to Day 42 |
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Key Inclusion Criteria:
- Documented clinical diagnosis of DM1 with age of onset greater than (>) 1 year of age and documented positive genetic test for DM1 in the subject with cytosine thymine guanine (CTG) repeat of at least 100
Key Exclusion Criteria:
- History of any illness or any clinical condition as pre-specified in the protocol
Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Contact | 617-341-6777 | medicalinfo@vrtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Neuromuscular Research | Recruiting | San Carlos | California | 94070 | United States | |
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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| Placebo | Drug | Solution for intravenous administration. |
|
| Part B: Maximum Observed Concentration (Cmax) of VX-670 and its Active Component in Plasma After Each Dose | From Day 1 up to Day 168 |
| Part B: Area Under the Concentration Versus Time Curve (AUC) of VX-670 and its Active Component in Plasma After Each Dose | From Day 1 up to Day 168 |
| Part B: Concentration of VX-670 and its Active Component in Muscle | Baseline, Day 15 and Day 120 |
| Part B: Change in Splicing Index in Muscle Biopsy | Baseline, Day 15 and Day 120 |
| University of Florida Clinical Research Center |
| Recruiting |
| Gainesville |
| Florida |
| 32608 |
| United States |
| University of Kansas Medical Center | Recruiting | Fairway | Kansas | 66205 | United States |
| Boston Children's Hospital | Recruiting | Boston | Massachusetts | 02115 | United States |
| Washington University School of Medicine / St. Louis Children's Hospital | Recruiting | St Louis | Missouri | 63110 | United States |
| Wake Forest Baptist Health | Recruiting | Winston-Salem | North Carolina | 27104 | United States |
| University of Pennsylvania | Recruiting | Philadelphia | Pennsylvania | 19104 | United States |
| Virginia Commonwealth University (Sanger Hall) | Recruiting | Richmond | Virginia | 23298 | United States |
| Wesley Research Institute | Recruiting | Auchenflower | Australia |
| Neuroscience Clinical Trials Unit, Alfred Brain | Recruiting | Melbourne | Australia |
| Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg | Recruiting | Leuven | Belgium |
| Hopital de Chicoutimi | Recruiting | Chicoutimi | Canada |
| Altasciences Montreal | Recruiting | Montreal | Canada |
| McGIll University | Recruiting | Montreal | Canada |
| University of Ottawa | Recruiting | Ottawa | Canada |
| CHU Research Centre of Quebec | Recruiting | Québec | Canada |
| Neuromuscular Reference Center Institute of Myology | Recruiting | Paris | France |
| Ludwig Maximilians Universitaet Muenchen | Recruiting | München | Germany |
| Centro Clinico NeMO | Recruiting | Milan | Italy |
| Maastricht University Medical Center | Recruiting | Maastricht | Netherlands |
| Hospital Universitario y Politécnico La Fe | Recruiting | Valencia | Spain |
| Clinical Research Facility, Queen Elizabeth University Hospital | Recruiting | Glasgow | United Kingdom |
| Leonard Wolfson Experimental Neurology Centre CRF | Recruiting | London | United Kingdom |
| St. George's University Hospital | Recruiting | London | United Kingdom |
| Salford Royal Hospital | Recruiting | Salford | United Kingdom |
| Royal Hallamshire Hospital | Recruiting | Sheffield | United Kingdom |
| ID | Term |
|---|---|
| D009223 | Myotonic Dystrophy |
| D009222 | Myotonia |
| D020967 | Myotonic Disorders |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D009136 | Muscular Dystrophies |
| ID | Term |
|---|---|
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D020879 | Neuromuscular Manifestations |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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