All results

35 results for randomized double blind placebo controlled study assess efficacy safety and

• CTD-TCNPC-301

  A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann…

  Ages

  3 Years - N/A

  Sexes

  All

• Edgewise-201 (CANYON)

  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…

  Ages

  12 Years - 50 Years

  Sexes

  Male

• A Multiple Dose Study to Evaluate Safety, Tolerability, PK, and Efficacy of SER-155 in Adults Underg

  An Open-Label and Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of SER-155…

  Ages

  18 Years - N/A

  Sexes

  All

• JNT-517

  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)…

  Ages

  18 Years - 65 Years

  Sexes

  All

• Soleno C602

  This is a multi-center, multi-period study with an open-label period followed by a double-blind, placebo-controlled, randomized withdrawal period evaluating…

  Ages

  4 Years - N/A

  Sexes

  All

• Armata

  Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Escalation Study of the Safety, Tolerability, and Efficacy of Intravenous AP…

  Ages

  18 Years - N/A

  Sexes

  All

• diSArm

  Phase 1b/2a, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Escalation Study of the Safety, Tolerability, and Efficacy of Intravenous AP…

  Ages

  18 Years - N/A

  Sexes

  All

• CC-90001 in Subjects With Idiopathic Pulmonary Fibrosis

  This is a Phase 2, multicenter, multinational, randomized, double-blind, placebo-controlled study evaluating the efficacy, safety, pharmacokinetics (PK),…

  Ages

  40 Years - N/A

  Sexes

  All

• STOMP Gainesville

  A5418 is a randomized, placebo-controlled, double-blind study to establish the efficacy of tecovirimat for the treatment of people with laboratory-confirmed or…

  Ages

  N/A - N/A

  Sexes

  All

• 9001-303 (ENVISION)

  The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is…

  Ages

  N/A - N/A

  Sexes

  Male