All results
36 results for randomized double blind placebo controlled study assess efficacy safety and
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Clinical Trials
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann…
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Clinical Trials
A study of EDG-5506 in Becker muscular dystrophy (known as CANYON) and pivotal cohort (known as GRAND CANYON). The EDG-5506-201 CANYON study was expanded to…
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Clinical Trials
The goal of Parts A and B of this Phase 1, first-in-human, randomized study is to assess the safety, tolerability, and pharmacokinetics (PK) of single (SAD)…
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Clinical Trials
This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating…
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Clinical Trials
Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Escalation Study of the Safety, Tolerability, and Efficacy of Intravenous AP…
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Clinical Trials
Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Escalation Study of the Safety, Tolerability, and Efficacy of Intravenous AP…
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Clinical Trials
This is a Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, pharmacokinetics (PK),…
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Clinical Trials
A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of tecovirimat for the treatment of people with laboratory-confirmed or…
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Clinical Trials
The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is…
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Clinical Trials
This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with…