Clinical trials: Bone Diseases, Developmental
Scoliosis; Adolescence, Bone Diseases
Thumb Ossification Composite Index (TOCI) to Predict Skeletal Maturity and Curve Progression in AIS
Accurate skeletal maturity assessment is important for prediction of curve progression and clinical management of adolescent idiopathic scoliosis (AIS) including bracing decision and counseling for prognosis.
- Conditions
- Scoliosis; Adolescence, Bone Diseases, Bone Growth Abnormal
- Trial name
- TOCI (NCT03904914)
- Testing
- TOCI
- Where
- Hong Kong
- Volunteers
- 1,500 people, healthy volunteers welcome
- Sponsor
- Chinese University of Hong Kong
Achondroplasia
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
This is a Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia.
- Trial name
- ACH (NCT02724228)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
An Extension Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia
- Trial name
- NCT03424018
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 119 people
- Sponsor
- BioMarin Pharmaceutical
Hypochondroplasia
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
- Trial name
- CANOPY-HCH-3 (NCT06455059)
- Testing
- Vosoritide
- Where
- 23 sites in United States, Australia and 7 more
- Volunteers
- 80 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
Study of Infigratinib in Children With Achondroplasia
This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase…
- Trial name
- NCT04265651
- Testing
- Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 84 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Bone Growth Abnormal, Inflammation
Effects of Increased Greek Yogurt Consumption in Youth and Young Adult Athletes
Physical activity and dairy consumption during growth and development are each known to improve peak bone mass in young adults. Peak bone mass, the maximum amount of bone a person will have in their lifetime, is typically reached…
- Trial name
- NCT05922462
- Testing
- Greek yogurt, Whey protein
- Where
- Canada
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Brock University
Achondroplasia
A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia
Study 111-206 is a Phase 2 randomized, double-blind, placebo-controlled clinical trial of BMN 111 in infants and young children with a diagnosis of achondroplasia.
- Trial name
- NCT03583697
- Testing
- BMN 111
- Where
- 16 sites in United States, Australia and 2 more
- Volunteers
- 75 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia
The intent and design of this Phase 3 study is to assess BMN 111 as a therapeutic option for the treatment of children with Achondroplasia.
- Trial name
- NCT03197766
- Testing
- BMN 111
- Where
- 24 sites in United States, Australia and 5 more
- Volunteers
- 121 people
- Sponsor
- BioMarin Pharmaceutical
Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
This protocol provides expanded access to bone marrow transplants for children who lack a histocompatible (tissue matched) stem cell or bone marrow donor when an alternative donor (unrelated donor or half-matched related donor)…
- Conditions
- Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT01200017
- Testing
- CD34+ enriched, T Cell Depleted donor stem cell product
- Where
- United States
- Sponsor
- Christopher Dvorak
Achondroplasia
A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia
This is a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. The primary objective is to assess the safety and tolerability of daily BMN 111 administered to children with…
- Trial name
- ACH (NCT02055157)
- Testing
- BMN 111
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 35 people
- Sponsor
- BioMarin Pharmaceutical
Bone Diseases, Developmental
Developing a COMputerised Bone Age Tool
The Greulich and Pyle technique (G\&P) is that most commonly used to assess bone age in children, particularly in the context of suspected skeletal dysplasia.
- Trial name
- COMBAT1 (NCT02617901)
- Testing
- Left hand DXA
- Where
- United Kingdom
- Volunteers
- 20 people
- Sponsor
- Sheffield Children's NHS Foundation Trust
Autism Spectrum Disorder, Bone Diseases, Developmental
Bone Mass Accrual in Children With Autism Spectrum Disorder
This is a observational study to investigate the degree to which bone mineral density is impaired in boys with autism compared with typically developing controls.
- Trial name
- NCT03162445
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Growth Hormone Deficiency
VRS-317 in Adult Subjects With Growth Hormone Deficiency
The purpose of this research study is to determine the safety and tolerability of up to five doses of VRS-317 in Adult Growth Hormone Deficient patients.
- Trial name
- NCT01359488
- Testing
- VRS-317
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Versartis Inc.
Developmental Bone Disease, Dwarfism
Study of Skeletal Disorders and Short Stature
This study will determine the genes responsible for skeletal dysplasias (disorders of the skeleton) and short stature and define the range and type of medical problems they cause over time.
- Conditions
- Developmental Bone Disease, Dwarfism, Skeletal Dysplasias
- Trial name
- NCT00001754
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- National Human Genome Research Institute (NHGRI)