A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Recruiting
Phase 1
Phase 2
Interventional Study
Myotonic Dystrophy Type 1 (DM1)
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Age
- 18 – 64
- Sex
- Any
- Trial phase
- Phase 1/2
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 52 (estimated)
- Sponsor
- Vertex Pharmaceuticals Incorporated · Industry
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About This Trial
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.
Trial Locations
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Eligibility Criteria
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 clinica…
Contacts