Clinical trials

The purpose of this study is to determine the PK, safety and tolerability of multiple doses of intravenous (i.v.) secukinumab in pediatric participants with JPsA

Investigator

Melissa E Elder

Ages

2 Years - 17 Years

Sexes

All

The primary purpose of the study is to evaluate the safety and tolerability of multiple intravenous (IV) doses of DYNE-101 administered to participants with Myotonic Dystrophy Type 1 (DM1). The study consists of 4 periods: A Screening Period (up to…

Investigator

Sub Subramony

Ages

18 Years - 65 Years

Sexes

All

A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC for the Treatment of Myotonic Dystrophy Type 1

Investigator

Sub Subramony

Ages

16 Years - N/A

Sexes

All

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigator

Sub Subramony

Ages

16 Years - 70 Years

Sexes

All

A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1

Investigator

Sub Subramony

Ages

16 Years - 65 Years

Sexes

All

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5).

Investigator

Barry J Byrne

Ages

6 Years - 13 Years

Sexes

All

This is a Phase I/II open label study of intravenous (IV) infusion of ONC-841 as a single agent or in combination in patients with advanced/metastatic solid tumors. The study will evaluate seven dose levels of ONC-841. The Phase 1 part is dose…

Investigator

Thomas J George

Ages

18 Years - N/A

Sexes

All

A Phase 2 Open-label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of AOC 1020 Administered Intravenously to Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)

Investigator

Sub Subramony

Ages

16 Years - 72 Years

Sexes

All