Clinical trials: Dwarfism
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
Growth Hormone Deficiency (GHD)
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive…
- Trial name
- NCT06948214
- Testing
- LUM-201
- Where
- 55 sites in United States, Argentina and 6 more
- Volunteers
- 150 people
- Sponsor
- Lumos Pharma
Growth Hormone Deficiency Without Epiphyseal Closure
Ngenla Subcutaneous Injection Special Investigation
The purpose of this study is to learn about the long-term safety and effects of Ngenla. Ngenla is approved for treatment of GHD (Growth hormone deficiency) without epiphyseal closure under daily medical practice.
- Trial name
- NCT05602766
- Testing
- NGENLA (Somatrogon)
- Where
- Japan
- Volunteers
- 1 people
- 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
Osteogenesis Imperfecta, Short Stature
Natural History of the Collagen-Related Disorder Osteogenesis Imperfecta and Genotype Phenotype Correlation
Background: Osteogenesis Imperfecta (OI) is a connective tissue disorder. OI affects many aspects of a person s health and growth. It can cause frequent fractures, short stature, and bowing of the long bones.
- Trial name
- NCT03575221
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
Dwarfism, Pituitary
A Study to Learn About Somatrogon in Patients With Pediatric Growth Hormone Deficiency (p GHD) in India.
The purpose of this study is to learn about the safety of Somatrogon for the treatment of pediatric growth hormone deficiency (p GHD) in India.
- Trial name
- NCT06587035
- Testing
- Somatrogon
- Where
- 4 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Noonan Syndrome
A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
The purpose of this study in children with Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment.
- Trial name
- NCT06668805
- Testing
- Vosoritide Injection
- Where
- 38 sites in United States, Australia and 5 more
- Volunteers
- 30 people
- Sponsor
- BioMarin Pharmaceutical
Growth Hormone Deficiency
Evaluation of the Propensity of Patients Under rhGH to Envision a Modification of Their Treatment Regimen Toward LAGH
Daily subcutaneous injections of rhGH can be burdensome for patients, leading to poor adherence and reduced growth outcomes. This has spurred the development of long-acting GH (LAGH) analogues that allow for weekly, biweekly, or…
- Trial name
- TOWARD-LAGH (NCT06542809)
- Testing
- Questionnaire
- Where
- Belgium
- Volunteers
- 500 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
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
Turner Syndrome, Isolated Growth Hormone Deficiency
Neuropsychological Assessment of Children and Adolescents With Turner Syndrome
Turner syndrome (TS) is a rare chromosomal disorder characterized by partial or complete loss of one of the X chromosomes that affects about one in every 2000 female babies born.
- Conditions
- Turner Syndrome, Isolated Growth Hormone Deficiency, Cognitive Functions, Social Cognition
- Trial name
- ENEAST (NCT03812913)
- Testing
- psychological evaluation
- Where
- 3 sites in France
- Volunteers
- 70 people
- Sponsor
- University Hospital, Angers
Achondroplasia
A Clinical Trial to Investigate Long-term Safety, Tolerability, and Efficacy of Weekly Subcutaneous Doses With TransCon CNP in Children and Adolescents With Achondroplasia
TransCon CNP administered once-weekly in children and adolescents with achondroplasia who have completed a prior TransCon CNP clinical trial.
- Trial name
- AttaCH (NCT05929807)
- Testing
- TransCon CNP
- Where
- 19 sites in United States, Australia and 10 more
- Volunteers
- 140 people
- Sponsor
- Ascendis Pharma Growth Disorders A/S
Achondroplasia
A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Multiple Subcutaneous Doses of TransCon CNP Administered Once Weekly in Children With Achondroplasia
Purpose of the study: The main purpose of this study is to determine the safety and evaluate the effect of a once weekly dose of TransCon CNP in prepubertal children with achondroplasia in China.
- Trial name
- NCT05246033
- Testing
- TransCon CNP
- Where
- 5 sites in China
- Volunteers
- 24 people
- Sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.
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.
Skeletal Disorders
Study of Skeletal Disorders
Background: There are 461 conditions that affect the bones (skeletal disorders). Many of these are not well understood and do not have any specific treatments. Researchers want to collect more data about these conditions.
- Trial name
- NCT05031507
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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
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
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 (GHD)
Safety, PK/PD (Pharmacokinetics/Pharmacodynamics) and Efficacy of ACP-001 Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
A six month study of ACP-001, a long-acting growth hormone product, versus standard human growth hormone therapy. ACP-001 will be given once-a-week, standard human growth hormone (hGH) will be given on a daily basis.
- Trial name
- NCT01947907
- Testing
- ACP-001, Human Growth Hormone
- Where
- 36 sites in Belarus, Bulgaria and 12 more
- Volunteers
- 53 people
- Sponsor
- Ascendis Pharma A/S
Growth Hormone Deficiency (GHD)
Phase 3 Long Term Safety Extension Study of LUM-201 in Children With Growth Hormone Deficiency
This is a Multi-national Trial. The Goal of the Trial is to Offer Subjects Who Complete 12 Months in the LUM-201-10 Phase 3 Trial up to an Additional 36 Months of Treatment of LUM-201 While Evaluating Safety and Tolerability of…
- Trial name
- NCT07129759
- Testing
- LUM-201
- Volunteers
- 150 people
- Sponsor
- Lumos Pharma