Clinical trials: Dwarfism, open to volunteers
Osteogenesis Imperfecta, Osteoporosis
REMS25: Study on the Use of REMS Technology in Diseases Commonly Associated With Reduced Bone Mineral Density (BMD)
This study evaluates bone mineral density (BMD) in pediatric patients aged 5-18 years with conditions negatively affecting bone health, using REMS (Radiofrequency Echographic Multi Spectrometry), a non-invasive and radiation-free…
- Conditions
- Osteogenesis Imperfecta, Osteoporosis, Hypogonadisms, Neoplasia
- Trial name
- NCT07412782
- Testing
- REMS
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Meyer Children's Hospital IRCCS
Idiopathic Short Stature
The Efficacy and Safety of Inpegsomatropin Injection in Children With Idiopathic Short Stature
This is a multicenter, randomized, open-label, active-controlled Phase III clinical trial to evaluate the efficacy and safety of Inpegsomatropin injection，once a week，compared with recombinant human growth hormone (rhGH) in…
- Trial name
- NCT06927310
- Testing
- Inpegsomatropin Injection, Recombinant Human Growth Hormone Injection
- Where
- 38 sites in China
- Volunteers
- 300 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Idiopathic Short Stature
A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children…
- Trial name
- NCT06382155
- Testing
- Vosoritide Injection, Human Growth Hormone
- Where
- 47 sites in United States, Australia and 4 more
- Volunteers
- 100 people
- Sponsor
- BioMarin Pharmaceutical
Short Stature
The Dosage Exploration Study of PEG-rhGH for Treating Short Stature in Prepubertal and Pubertal Children
This study is dedicated to addressing the lack of research on the most effective dosage of long-acting growth hormone for children with short stature.
- Trial name
- NCT06768411
- Testing
- PEG-rhGH, GnRHa
- Volunteers
- 240 people
- Sponsor
- Second Affiliated Hospital of Wenzhou Medical University
Growth Hormone Deficiency
The AgRP and GH/IGF-1 Axis in Children
Recent data support the existence of a GH-Agouti-related peptide (AgRP) axis. The neuropeptide AgRP promotes food intake and has important effects on energy homeostasis.
- Trial name
- NCT05645211
- Volunteers
- 156 people, healthy volunteers welcome
- Sponsor
- Columbia University
Thyroid Volume, Thyroid Ultrasound
Ultrasound Measurement of Thyroid Volume in Term Newborns
First, to date, no data are available on the thyroid volume of full-term newborns in Italy, making it essential to conduct studies to establish reference values for normality.
- Conditions
- Thyroid Volume, Thyroid Ultrasound, Congenital Hypothyroidism, Healthy Newborns
- Trial name
- NEOTIR (NCT07579988)
- Testing
- Thyroid ultrasound
- Where
- Italy
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- IRCCS Ospedale San Raffaele
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
Hypochondroplasia
Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with…
- Trial name
- NCT07393373
- Testing
- Infigratinib
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 135 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Congenital Hypothyroidism
Comparison of Levothyroxine Formulations in the Treatment of Congenital Hypothyroidism
Observational, retrospective, prospective, pharmacological, single-centre, non-profit study. The aim is to evaluate, in newborns diagnosed with Congenital Hypothyroidism, the medium- and long-term efficacy and safety of the new…
- Trial name
- NCT06724224
- Where
- Italy
- Volunteers
- 120 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Leukodystrophy, White Matter Disease
The Myelin Disorders Biorepository Project
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects.
- Conditions
- Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome
- Trial name
- MDBP (NCT03047369)
- Where
- 23 sites in United States
- Volunteers
- 12,000 people
- Sponsor
- Children's Hospital of Philadelphia
Growth Hormone Deficiency, Adult Growth Hormone Deficiency
BELux Children OutcoME During A(@)Dulthood With GHD
Our objective is to evaluate the outcome in adulthood of Belgian and Luxembourgish patients treated with rhGH during childhood for CO-GHD.
- Trial name
- BELCOME@GHD (NCT06694506)
- Testing
- Biological Sample
- Where
- Belgium
- Volunteers
- 200 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Congenital Hypothyroidism
Role of Next Generation Sequencing in the Etiological Diagnosis of Permanent Congenital Hypothyroidism With in Situ Thyroid
Retro-prospective, exploratory, single-centre observational study conducted at the Endrocrine-Metabolic Diseases Centre of the Pediatrics Unit, IRCCS Azienda Ospedaliero-Universitaria di Bologna, Italy.
- Trial name
- NCT06728735
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Growth Hormone Deficiency, Idiopathic Short Stature
Long-term Safety and Effectiveness of Growtropin®-II Treatment in Children With Short Stature
This study evaluates long-term safety and effectiveness of Growtropin®-II treatment in children with short stature.
- Conditions
- Growth Hormone Deficiency, Idiopathic Short Stature, Turner Syndrome, Small for Gestational Age
- Trial name
- NCT04798690
- Where
- South Korea
- Volunteers
- 5,000 people
- Sponsor
- Dong-A ST Co., Ltd.
Adult Growth Hormone Deficiency
A Study to Follow Patients With Adult Growth Hormone Deficiency (AGHD) Treated With Sogroya® for Long Term Safety Information
In this study, the general long-term safety and effectiveness of Sogroya (somapacitan) in adults with growth hormone deficiency (AGHD) being treated per normal clinical practice is looked into.
- Trial name
- NCT05718570
- Testing
- Somapacitan
- Where
- 55 sites in United States, France and 4 more
- Volunteers
- 400 people
- Sponsor
- Novo Nordisk A/S
Congenital Hypothyroidism, Metabolic Syndrome
Metabolic Risk Assessment in Prepubertal Children With Congenital Hypothyroidism
We propose a multicenter prospective study to define the prevalence and severity score of metabolic syndrome in a prepubertal pediatric cohort with congenital hypothyroidism, compared to a healthy and normal-weight pediatric…
- Trial name
- IpoMet (NCT07126353)
- Testing
- prospective observational study (clinical, hormonal and auxological data)
- Where
- Italy
- Volunteers
- 170 people, healthy volunteers welcome
- Sponsor
- Buzzi Children's Hospital
Growth Hormone Deficiency in Children
Special Use-results Surveillance on Long Term Use of Sogroya® in Children With Short Stature Due to Growth Hormone Deficiency Where Epiphysial Discs Are Not Closed
The purpose of the study is to investigate the safety and effectiveness of Sogroya® in children with short stature due to growth hormone deficiency where epiphysial discs are not closed under real-world clinical practice in…
- Trial name
- NCT06109935
- Testing
- Somapacitan
- Where
- 86 sites in Japan
- Volunteers
- 200 people
- Sponsor
- Novo Nordisk A/S
Growth Hormone Treatment, Growth Disorders
The Efficacy and Safety of PEGylated GH for the Treatment of Short Stature in Chinese Children-GLOBE Reg
In order to further observe the long-term safety and effectiveness of real-world polyethylene glycol-recombinant human growth hormone(PEG-rhGH) treatment of GHD, idiopathic short stature, and SGA in children, explore and analyze…
- Trial name
- NCT06037473
- Testing
- Polyethylene glycol recombinant human growth hormone injection
- Where
- China
- Volunteers
- 2,600 people
- Sponsor
- Beijing Children's Hospital
Achondroplasia, Hypochondroplasia
Prospective Longitudinal Multicentric Study to Measure Limb Movement in Patients With FGFR3-related Skeletal Dysplasia
The study aims to identify which Syde®-derived digital outcomes are reliable in FGFR3-related Skeletal Dysplasia. This requires to set-up a natural history study to measure limb movements in patients with ACH or HCH.
- Trial name
- SKY-MOT3 (NCT07388966)
- Where
- 4 sites in Belgium, France and 1 more
- Volunteers
- 140 people
- Sponsor
- SYSNAV
Achondroplasia
A Study in Children With Achondroplasia
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural…
- Trial name
- NCT07301463
- Testing
- No Interventions, complete a natural history observation of ACH for at least 6 months and up to 2 years
- Where
- 8 sites in China
- Volunteers
- 260 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
MOPDII, Meier-Gorlin Syndrome
Primordial Dwarfism Registry
The goal of this registry is to collect information on individuals with forms of microcephalic primordial dwarfism as well as related conditions.
- Conditions
- MOPDII, Meier-Gorlin Syndrome, Saul-Wilson Syndrome, Microcephalic Primordial Dwarfism
- Trial name
- NCT04569149
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Nemours Children's Clinic