Murdoch Childrens Research Institute

Parkville, Victoria VIC 3052

9 studies enrolling now · 38 studies all time

What they study most

Achondroplasia, Alexander Disease, Atopic Dermatitis, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD), CLOVES Syndrome, Diabetes Mellitus, Type 2, Duchenne Muscular Dystrophy, Duchenne Muscular Dystrophy (DMD), Epilepsy, Fibrodysplasia Ossificans Progressiva (FOP), Friedreich Ataxia

Studies at this site

• Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia — Recruiting now
• Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) — Recruiting now
• A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study) — Recruiting now
• To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva — Recruiting now
• A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature — Recruiting now
• A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment — Recruiting now
• A Long-Term Study of JNT-517 in Participants With Phenylketonuria — Recruiting now
• International Multicentric Observational Study to Characterize Subpopulations of Patients With Autism Spectrum Disorder — Recruiting now
• Otoferlin Gene-mediated Hearing Loss Natural History Study — Recruiting now
• Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study — Recruiting now
• A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia — Completed
• A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) — Running, not enrolling
• An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia — Running, not enrolling
• A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) — Running, not enrolling
• An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia — Running, not enrolling
• A Research Study to Compare a New Medicine Oral Semaglutide to a Dummy Medicine in Children and Teenagers With Type 2 Diabetes — Completed
• A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia — Completed
• Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia — Running, not enrolling
• Extension Study of Pimavanserin in Irritability Associated With Autism Spectrum Disorder — Stopped early
• AB-2004 in Treatment of Irritability Associated With Autism Spectrum Disorder (ASD) — Completed
• Allergic Disease Onset Prevention Study — Completed
• A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia — Running, not enrolling
• Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder — Completed
• Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping — Running, not enrolling
• Long-Term Extension Study of Vosoritide to Treat Children With Hypochondroplasia — Enrolling by invitation
• Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia — Withdrawn before enrolling
• Adalimumab in JIA-associated Uveitis Stopping Trial — Completed
• Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia. — Stopped early
• FA Clinical Outcome Measures — Running, not enrolling
• Phase 3 Trial of Pamrevlumab or Placebo in Combination With Systemic Corticosteroids in Participants With Ambulatory DMD — Stopped early
• A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia — Completed
• Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD) — Stopped early
• A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia — Stopped early
• Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia — Stopped early
• Gene-STEPS: Shortening Time of Evaluation in Paediatric Epilepsy Services — Status unconfirmed
• A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia — Completed
• A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients With Achondroplasia — Completed
• A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia — Completed