All results
33 results for randomized double blind placebo controlled study assess efficacy safety and
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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
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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
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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
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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
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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
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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
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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
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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
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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
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PVT-2201-301
This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with…
- Ages
- 18 Years - 75 Years
- Sexes
- All