Clinical trials: Dwarfism, open to volunteers
Congenital Hypothyroidism
Evaluation of an Intensified Systematic Screening for Congenital Hypothyroidism in Premature Newborns
Currently in France, screening for congenital hypothyroidism (CH) in premature infants is done by a single TSH assay on filter paper. However, European recommendations advise repeating the assay within the first month of life.
- Trial name
- PREMATHYRO (NCT07425028)
- Testing
- Blood test
- Where
- France
- Volunteers
- 1,600 people
- Sponsor
- University Hospital, Lille
Short Stature Children Born Small for Gestational Age (SGA)
A Phase II Study of PEG-rhGH Injection for Short Children Born Small for Gestational Age: Efficacy, Safety, and Pharmacokinetics
This study will take place at multiple sites and is divided into three phases:an initial 4-week getting-to-know-you phase,a 26-week main phase where participants receive medication,and a 4-week follow-up phase.Participants will…
- Trial name
- NCT07260500
- Testing
- PEG-rhGH Injection, hGH Injection
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Small for Gestational Age
A Phase III Clinical Study of Jinpei Growth Hormone Injection in the Treatment of Short Stature Children With Small for Gestational Age (SGA)
This is a multicenter, randomized, open-label, active-controlled Phase III clinical study to evaluate the efficacy, safety and pharmacokinetic (PK) characteristics of once-weekly PEG-hGH injection compared with once-daily hGH…
- Trial name
- NCT07841678
- Testing
- PEG-hGH Injection, hGH Injection
- Where
- China
- Volunteers
- 105 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Hypochondroplasia
Prospective Clinical Assessment Study in Children With Hypochondroplasia
This is a long-term, multicenter, non-interventional study of children ages 2.5 to <17 years with hypochondroplasia (HCH).
- Trial name
- HCH (NCT06410976)
- Where
- 25 sites in United States, Australia and 8 more
- Volunteers
- 150 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
Non-invasive Functional Assessment and Pathogenesis of Morquio A
Morquio A disease is a devastating systemic skeletal disease in which detailed progression and pathogenesis remain unknown. The proposed project aims to establish a non-invasive objective assessment that can be applicable to all…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- NIFAMA (NCT05284006)
- Testing
- Imaging, gait analysis, growth, joint test, hearing test, questionnaire, etc.
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Nemours Children's Clinic
Adult Growth Hormone Deficiency
Safety, Pharmacokinetic (PK) Profile and Efficacy of Inpegsomatropin Injection in Adults With Growth Hormone Deficiency
This is a multiple-dose, dose-escalation, randomized, double-blind, placebo-controlled phase Ⅱa clinical trial. A total of 36 adult participants with growth hormone deficiency will be enrolled sequentially in ascending dose…
- Trial name
- NCT07551804
- Testing
- Inpegsomatropin Injection
- Where
- 6 sites in China
- Volunteers
- 36 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Idiopathic Short Stature
A Study to Assess Growth in Children With Idiopathic Short Stature
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
- Trial name
- NCT06309979
- Where
- 38 sites in United States
- Volunteers
- 300 people
- Sponsor
- BioMarin Pharmaceutical
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
Turner Syndrome
The Efficacy and Safety of Inpegsomatropin Injection in Children With Turner Syndrome (TS) and Short Stature
This is a multicenter, randomized, open-label, positive-controlled phase III confirmatory clinical study. A total of 84 children with short stature due to Turner Syndrome (TS) are planned to be enrolled.
- Trial name
- NCT07614152
- Testing
- Inpegsomatropin-Injection, Givopegsomatropin Solution Injection
- Where
- 51 sites in China
- Volunteers
- 84 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Growth Hormone Deficiency, Traumatic Brain Injury
Charactertizing Growth Hormone Deficiency in Traumatic Brain Injury
The goal of this observational study is to characterize the impact of growth hormone deficiency (GHD) on quality of life, symptom burden, and sleep in adults with traumatic brain injury (TBI), and to evaluate whether these…
- Trial name
- NCT07795736
- Testing
- Not applicable (observational study)
- Where
- 2 sites in Canada
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- University of Calgary
Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism
Growing up With the Young Endocrine Support System (YESS!)
Transition from paediatric to adult endocrinology is a challenge for adolescents, families and doctors. Up to 25% of young adults with chronic endocrine disorders are lost to follow-up ('drop-out') once the young adult moves out…
- Conditions
- Congenital Adrenal Hyperplasia, Hypogonadotropic Hypogonadism, Growth Hormone Deficiency, Combined Pituitary Hormone Deficiency
- Trial name
- YESS (NCT04252001)
- Testing
- YESS! game, Snake '97, Transition-toolkit
- Where
- 7 sites in Belgium, Netherlands and 2 more
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- dr. Laura C. G. de Graaff-Herder
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 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
Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children
GB06 Phase III Trial for Pediatric Growth Hormone Deficiency
This study aims to evaluate the efficacy and safety of GB06 (a biosimilar of Norditropin®FlexProTM from Novo Nordisk) for the treatment of growth disorders caused by growth hormone deficiency (GHD) in children.
- Conditions
- Growth Hormone Deficiency (GHD), Growth Hormone Deficiency in Children, Growth Hormone Deficiency, Pediatric
- Trial name
- NCT07553351
- Testing
- GB06, Norditropin®FlexProTM
- Where
- China
- Volunteers
- 192 people
- Sponsor
- Kexing Biopharm Co., Ltd.
Childhood Short Stature
Long-term Efficacy and Safety Evaluation of Growth Hormone in Children in China(CGLS)
Short stature is a relatively common pediatric condition, referring to individuals whose height is more than 2 (-2 SD) standard deviations below the average height of a similar age, gender, and ethnicity population in similar…
- Trial name
- NCT06110910
- Testing
- Polvethylene glycol recombinant human growth hormone injection (PEG-rhGH) or recombinanthuman growth hormone injection (rhGH)
- Where
- China
- Volunteers
- 10,000 people
- Sponsor
- Xiaoping Luo
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Idiopathic Short Stature
Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature Patients
This trial aims to evaluate the prevalence of idiopathic short stature among children whose growth is above -2,5SD (AFPA- CRESS/Inserm -CompuGroup Medical 2018 curve) or above -2SD of the parental target size (taking child gender…
- Trial name
- PAG PETI (NCT05858606)
- Testing
- Evaluation of the prevalence of truly (authentified) idiopathic short stature after multidisciplinary clinico-radiological evaluation, analysis in a multidisciplinary consultation meeting via a secure platform (ShareConfrère) for evaluation by multidisciplinary team, Whole genome analysis for authentified idiopathic short stature
- Where
- France
- Volunteers
- 200 people
- Sponsor
- University Hospital, Montpellier
Achondroplasia
VIrtual STudy in Achondroplasia for the US (VISTA)
This is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™.
- Trial name
- VISTA (NCT06168201)
- Where
- 8 sites in United States
- Volunteers
- 170 people
- Sponsor
- BioMarin Pharmaceutical
Congenital Hypothyroidism, Neurodevelopmental Disorders
Understanding the Subnormal Rescue ofNeurological Development in Congenital Hypothyroidism (2022E5SZEM)
Congenital hypothyroidism (CH) is a condition present at birth in which the thyroid gland does not produce enough thyroid hormones. These hormones are essential for normal brain development.
- Trial name
- 2022E5SZEM (NCT07893522)
- Where
- 3 sites in Italy
- Volunteers
- 178 people
- Sponsor
- Istituto Giannina Gaslini
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of TransCon CNP Compared With Placebo in Infants (0 to <2 Years of Age) With Achondroplasia
This trial is a Phase 2, multicenter, double-blind, randomized (ratio 2:1 TransCon CNP vs. placebo), placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 μg CNP/kg of Navepegritide…
- Trial name
- NCT06079398
- Testing
- Navepegritide
- Where
- 18 sites in United States, Australia and 13 more
- Volunteers
- 72 people
- Sponsor
- Ascendis Pharma A/S