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Active trials

5 of 77 total trials

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1) (Galileo)

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple d...

Enrolling
Myotonic Dystrophy Type 1 (DM1)
Drug: Placebo
Drug: VX-670

This study will assess the efficacy and safety of vamikibart in participants with uveitic macular edema.

Active, not recruiting
Uveitic Macular Edema
Other: Sham
Drug: Vamikibart

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Explora...

Enrolling
Muscular Dystrophy, Landouzy Dejerine
Fascioscapulohumeral Muscular Dystrophy
Drug: Placebo
Drug: AOC 1020

A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, and Pharmacokinetics of AOC 1020 Administered Intraven...

Invitation-only
Muscular Dystrophy, Landouzy Dejerine
Fascioscapulohumeral Muscular Dystrophy
Drug: AOC 1020

The primary objective of this study is to demonstrate the efficacy of Iomab-B, in conjunction with a Reduced Intensity Conditioning (RIC) regimen and...

Active, not recruiting
AML
Bone Marrow Transplant
Drug: Iomab-B
Drug: Conventional Care

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