Clinical trials: Musculoskeletal Growth and Development
- Condition Musculoskeletal Growth and Development
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Growth Hormone Insensitivity Syndrome
Long-Term Treatment With rhIGF-1 in GHIS
The objective of this study was to evaluate the long-term safety and effectiveness of mecasermin (the study drug) in children with growth failure due to severe Primary insulin-like growth factor-1 deficiency (Primary IGFD).
- Trial name
- NCT00571727
- Testing
- mecasermin
- Where
- United States
- Volunteers
- 92 people
- Sponsor
- Ipsen
Growth Hormone Deficiency
A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
- Trial name
- SkyPASS (NCT05775523)
- Testing
- No intervention
- Where
- 27 sites in United States
- Volunteers
- 500 people
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
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
Growth Hormone Deficiency, Growth Retardation
Treatment With Recombinant Human Growth Hormone Genotonorm (Registered) In Children With Short Stature Secondary
To assess the effect of long-term treatment by Genotonorm on linear growth
- Trial name
- NCT00174278
- Testing
- Somatropin
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Pfizer
Growth Hormone Deficiency
Phase 2 Study of LUM-201 in Children Who Have Previously Completed the LUM-201-01 Trial (OraGrowtH213)
This is a multi-national trial. The goals of the trial are to study the growth response to LUM-201 administration in children with idiopathic growth hormone deficiency (GHD) previously treated with daily rhGH for 12 months in the…
- Trial name
- OraGrowtH213 (NCT05250063)
- Testing
- LUM-201
- Where
- 10 sites in United States, Poland
- Volunteers
- 15 people
- Sponsor
- Lumos Pharma
Idiopathic Short Stature, Small for Gestational Age
A Trial of YPEG-rhGH in Children With Short Stature
To explore the dose-response relationship between pharmacokinetics and pharmacodynamics of Y- Shaped Pegylated growth hormone injection (YPEG-GH) in children with short stature (idiopathic short stature (ISS), small for…
- Conditions
- Idiopathic Short Stature, Small for Gestational Age, Turner Syndrome
- Trial name
- NCT05838885
- Testing
- YPEG-rhGH, rhGH
- Where
- 19 sites in China
- Volunteers
- 78 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
SGA, Turner Syndrome
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
The purpose of this study is to find out if somapacitan is safe and how well somapacitan works in children either born small for gestational age or with Turner syndrome, Noonan syndrome or idiopathic short stature.
- Conditions
- SGA, Turner Syndrome, Noonan Syndrome, ISS
- Trial name
- REAL 9 (NCT05723835)
- Testing
- Somapacitan
- Where
- 18 sites in United States, Malaysia and 4 more
- Volunteers
- 47 people
- Sponsor
- Novo Nordisk A/S
Pediatric Growth Hormone Deficiency
A Study to Compare the Experiences of Taking Daily Growth Hormone Injections to Weekly Ngenla in Children With Low Levels of Growth Hormone
The purpose of this study is to learn about: how children stick to taking their injections their experience with the study medicines (Ngenla and daily growth hormone) prescribed to children with low levels of growth hormone.
- Trial name
- NCT06113952
- Testing
- Sharps bin to collect used needles/injections
- Sponsor
- Pfizer
Turner Syndrome, Short Stature Homeobox Gene Mutation
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
This basket trial will enroll prepubertal children and adolescents with clinically diagnosed and genetically confirmed (if applicable) TS, SHOX-D, SGA, or ISS between ages of ≥2 and <18 years with open growth plates.
- Conditions
- Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
- Trial name
- NCT07221851
- Testing
- Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
- Where
- 51 sites in United States, France and 5 more
- Volunteers
- 186 people
- Sponsor
- Ascendis Pharma A/S
Adult Growth Hormone Deficiency
Post-Marketing Surveillance (Special Use-results Surveillance on Long-term Use) With Sogroya®
Participants are invited to take part in this study because they have AGHD (only severe case). The purpose of this study is to assess long term safety and effectiveness of Sogroya® in patients with AGHD (only severe case) under…
- Trial name
- NCT05230550
- Testing
- Somapacitan
- Where
- 47 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Achondroplasia
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)
This is a long-term, multi-center, observational study in children 2.5 to <17 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications, assessments of health-related quality of life…
- Trial name
- NCT04035811
- Where
- 32 sites in United States, Argentina and 9 more
- Volunteers
- 271 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Osteoarthritis, Cancer Pain
Protocol to Monitor the Neurological Development of Infants With Exposure in Utero From Birth to 15 Months in Tanezumab Clinical Studies
A4091065 is a multicenter, prospective, cohort study with enhance physical an neurodevelopmental surveillance to characterize the outcomes related to the development of infants up to the age of 15 months who were potentially…
- Conditions
- Osteoarthritis, Cancer Pain, Recurrent Low Back Pain
- Trial name
- NCT03031938
- Testing
- Investigational medical product (IMP) administered in parent study
- Where
- 6 sites in United States
- Volunteers
- 4 people
- Sponsor
- Pfizer
Growth Hormone Deficiency
Practicability and Acceptability of Stylomax® in Children
Primary objective: to evaluate the practicability and acceptability of STYLOMAX®, a new injection device for MAXOMAT®, in children, for 1 year.
- Trial name
- NCT00476385
- Testing
- somatropine
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Sanofi
Dwarfism, Growth Hormone Deficiency
A Multicentre, Randomised, Open-label, Controlled Study to Evaluate the Effects of Saizen® on Cardiac Function in Growth Hormone Deficient(GHD) Subjects During the Transition Phase From Childhood to Adulthood
This was a 48-week, open-label, prospective, multicentric, randomised, comparative with parallel control, Phase 4 study to evaluate the effects of Saizen on cardiac function in GHD subjects during the transition phase from…
- Trial name
- NCT01157793
- Testing
- r-hGH
- Volunteers
- 34 people
- Sponsor
- Merck KGaA, Darmstadt, Germany
SGA, Turner Syndrome, Noonan Syndrome, ISS
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
The study compares two medicines for treatment of children born small and who stay small, or with Turner Syndrome, Noonan Syndrome, or idiopathic short stature.
- Trial name
- REAL 8 (NCT05330325)
- Testing
- Somapacitan, Norditropin®
- Where
- 199 sites in United States, Austria and 31 more
- Volunteers
- 412 people
- Sponsor
- Novo Nordisk A/S
Pediatric Growth Hormone Deficiency
Versartis Trial in Children to Assess Long-Acting Growth Hormone
This is a Phase 1b/2a study of VRS-317 (long-acting growth hormone) in pediatric patients with growth hormone deficiency. During Phase 1b, pediatric patients each will receive a single subcutaneous injection of VRS-317.
- Trial name
- VERTICAL (NCT01718041)
- Testing
- Experimental: VRS-317
- Where
- 23 sites in United States
- Volunteers
- 64 people
- Sponsor
- Versartis Inc.
Achondroplasia
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants < 3 years old with ACH.
- Trial name
- NCT07169279
- Testing
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 77 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Growth Hormone Deficiency, Neoplasms
A Study to Follow Paediatric Participants With Growth Hormone Deficiency Treated With Somapacitan for Long Term Safety Information and Clinical Parameters
The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions.
- Conditions
- Growth Hormone Deficiency, Neoplasms, Diabetes Mellitus Type 2
- Trial name
- NCT06709040
- Testing
- Somapacitan
- Where
- United Kingdom
- Volunteers
- 500 people
- Sponsor
- Novo Nordisk A/S