113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details


臨床試験数 : 646 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 170

  
1 trial found
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enrollment
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agemin
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PhaseCountries
1NCT05524883
(ClinicalTrials.gov)
August 12, 202230/8/2022Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingA Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 SkippingDuchenne Muscular Dystrophy (DMD)Drug: DYNE-251;Drug: PlaceboDyne TherapeuticsNULLRecruiting4 Years16 YearsMale46Phase 1/Phase 2United States;Belgium;Spain