Murdoch Children's Research Institute
Murdoch Children's Research Institute
Parkville, Victoria, Australia21 results · Page 1 of 1
Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Determining the Effectiveness of Working Out Dads to Reduce Mental Health Difficulties in Fathers of Young Children | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Neoplasms by Site, Neoplasms, Multiple Primary, endocrine system disorder, hereditary disease, hereditary neoplastic syndrome, malignant endocrine neoplasm, multiple endocrine neoplasia, multiple endocrine neoplasia type 1, neoplasm | Active, not recruiting | Not applicable | 2026-10-08 | NCT04813042 | |
| Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 / Phase 3 | 2026-09-24 | NCT07441876 | |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment | Bone Diseases, Developmental, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Craniofacial Abnormalities, Dwarfism, Musculoskeletal Abnormalities, Noonan syndrome, Skin and Connective Tissue Diseases, bone disorder, cardiovascular disorder, congenital heart disease, connective tissue disorder, endocrine system disorder, heart disorder, hereditary disease, musculoskeletal system disorder | Active, not recruiting | Phase 2 | 2026-09-23 | NCT06668805 | |
| Generation Victoria Cohort 2020s: A Statewide Longitudinal Cohort Study of Victorian Children and Their Parents | Body Weight, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, Infections, Inflammation, Injuries, Noncommunicable Diseases, Nutritional and Metabolic Diseases, Overweight, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Signs and Symptoms, immune system disorder, infectious disease, injury, nutritional disorder, obesity disorder, overnutrition | Recruiting | Not stated | 2026-09-17 | NCT05394363 | |
| A Study to Learn About the Safety of an Expanded Pneumococcal Vaccine in Healthy Infants | Bacterial Infections and Mycoses, Infections, bacterial infectious disease, gram-positive bacterial infections, pneumococcal infection, streptococcal infection | Recruiting | Phase 3 | 2026-09-15 | NCT07573462 | |
| 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) | CLOVES syndrome, Cardiovascular Abnormalities, Congenital Abnormalities, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hemic and Lymphatic Diseases, Lymphatic Abnormalities, angiomatosis, angioosteohypertrophic syndrome, cardiovascular disorder, lymphatic system disorder, megalencephaly-capillary malformation-polymicrogyria syndrome, vascular disorder, vascular malformation | Recruiting | Phase 2 | 2026-08-25 | NCT06789913 | |
| A Study to Evaluate the Safety and Efficacy of Zilganersen (ION373) in Patients With Alexander Disease (AxD) | Alexander disease, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Hereditary Central Nervous System Demyelinating Diseases, Leukoencephalopathies, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, demyelinating disease, hereditary disease, inborn errors of metabolism, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease | Active, not recruiting | Phase 3 | 2026-08-21 | NCT04849741 | |
| Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Duchenne muscular dystrophy, Muscular Disorders, Atrophic, X-linked disease, hereditary disease, muscle tissue disorder, muscular dystrophy, musculoskeletal system disorder, nervous system disorder, neuromuscular disease | Active, not recruiting | Phase 1 / Phase 2 | 2026-08-11 | NCT05524883 | |
| Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, achondroplasia, bone disorder, connective tissue disorder, cutaneous mucinosis, endocrine system disorder, hereditary disease, hereditary endocrine growth disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 4, musculoskeletal system disorder, osteochondrodysplasia | Recruiting | Phase 2 | 2026-07-13 | NCT07169279 | |
| TAG TEAM - Trans Adolescent Group Therapy | anxiety, anxiety disorder, psychiatric disorder | Recruiting | Not applicable | 2026-06-05 | NCT07151079 | |
| A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature | Idiopathic Short Stature | Recruiting | Phase 2 | 2026-05-22 | NCT06382155 | |
| A Long-Term Study of JNT-517 in Participants With Phenylketonuria | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Nutritional and Metabolic Diseases, brain disorder, central nervous system disorder, hereditary disease, inborn disorder of amino acid metabolism, inborn errors of metabolism, metabolic disease, nervous system disorder, phenylketonuria | Recruiting | Phase 3 | 2026-05-01 | NCT06628128 | |
| International Multicentric Observational Study to Characterize Subpopulations of Patients With Autism Spectrum Disorder | autism spectrum disorder, neurodevelopmental disorder, pervasive developmental disorder, psychiatric disorder | Recruiting | Not stated | 2026-04-27 | NCT05590715 | |
| A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT04554940 | |
| An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT03989947 | |
| An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 3 | 2026-03-13 | NCT03424018 | |
| A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH) | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, achondroplasia, bone disorder, endocrine system disorder, hereditary disease, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 2 | 2026-03-13 | NCT02724228 | |
| A Long Term, Observational Follow-Up Study of Children and Young People Who Underwent an 18-Month Course of Oral Immunotherapy Treatment for Peanut, Egg or Milk Allergy (5-15 Years Post-Treatment) | Egg Hypersensitivity, Hypersensitivity, Immediate, Milk Hypersensitivity, Nut and Peanut Hypersensitivity, Peanut Hypersensitivity, food allergy, immune system disorder | Recruiting | Not stated | 2026-02-02 | NCT07359183 | |
| Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia | Bone Diseases, Developmental, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Dwarfism, bone disorder, endocrine system disorder, hereditary disease, hypochondroplasia, musculoskeletal system disorder, osteochondrodysplasia | Active, not recruiting | Phase 3 | 2026-01-20 | NCT06455059 | |
| A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Friedreich ataxia, Nutritional and Metabolic Diseases, Spinocerebellar Degenerations, brain disorder, central nervous system disorder, cerebellar disorder, hereditary disease, inborn mitochondrial metabolism disorder, inherited neurodegenerative disorder, metabolic disease, nervous system disorder, neurodegenerative disease, spinal cord disorder | Active, not recruiting | Phase 3 | 2025-10-30 | NCT05515536 | |
| The PEBBLES Study - Testing a Strategy for Preventing Eczema and Food Allergy in High Risk Infants | Hypersensitivity, Immediate, Respiratory Hypersensitivity, Skin Diseases, Eczematous, Skin and Connective Tissue Diseases, asthma, atopic eczema, bronchial disorder, dermatitis, food allergy, immune system disorder, lung disorder, obstructive lung disease, respiratory system disorder, skin disorder | Active, not recruiting | Phase 3 | 2023-05-10 | NCT03667651 |