A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Фаза 1/2 – комбинирано ранно изпитване
Заболявания:
Myotonic Dystrophy Type 1 (DM1)
Спонсор: Vertex Pharmaceuticals Incorporated
Налично на:
БГ
Обобщение
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.
Кой може да участва
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.
БГ
EN