The Children's Hospital at Westmead | Cancer Centre for Children

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

The overall aims of this study are to demonstrate that treatment with PVX108 immunotherapy has an acceptable safety profile and is effective for redu...

This phase II trial studies the side effects and how well nirogacestat works in treating patients less than 18 years of age with desmoid tumors that...

The study's purpose is to see if the drug, abemaciclib, is safe and effective when given with other drugs to kill cancer cells. The study is open to...

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambul...

The reason for this study is to see if the study drug called baricitinib works and is safe in children and teenage participants with atopic dermatiti...

The reason for this study is to see if the study drug baricitinib is safe and effective in the treatment of JIA in participants ages 1 to 17. This st...

The main purpose of this study is to measure the effect, safety and how well the body absorbs lebrikizumab in pediatric participants 6 months to \<18...

Study participants will be screened during the platform study and randomly assigned to receive mirikizumab or another intervention. The purpose of th...

This is an open-label, multi-center Phase 1/2 study of oral LOXO-292 in pediatric participants with an activating rearranged during transfection (RET...

Phase 1 will evaluate the safety and tolerability at different dose levels of repotrectinib in pediatric and young adult subjects with advanced or me...

The goal of the study is to assess how tirzepatide impacts bodyweight and cardiovascular risk factors when used in conjunction with healthy nutrition...

Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by the presence of bone in soft tissue where bone nor...

The main purpose of this study is to assess the long-term safety and efficacy of lebrikizumab in participants 6 Months to \<18 years of age with mode...

FIREFLY-1 is an ongoing, Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, a...

A 2-part study to evaluate the safety, pharmacokinetics and efficacy of EDP-938 in children with RSV infection.

This is a Phase IIIb, multicenter, open-label, single-arm study of prophylactic emicizumab in previously untreated and minimally treated patients at...

The purpose of this study is to learn more about the safety and efficacy of tirzepatide compared to placebo in children or teenagers with type 2 diab...

Researchers are looking for a better way to treat children who have chronic kidney disease (CKD), which is long-term kidney disease, and proteinuria,...

This is a Phase 1b/2, randomized, double-blind, multi-center study to evaluate the safety, tolerability, and preliminary clinical efficacy of STMC-10...