Clinical trials: Short Stature
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
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
Loss of Chromosome 18q, Growth Hormone Deficiency
Growth Hormone and Chromosome 18q- and Abnormal Growth
We, the investigators at the University of Texas Health Science Center at San Antonio, want to learn if height and IQ (intelligence quotient) scores are improved by growth hormone (GH) treatment in children with chromosome 18…
- Trial name
- NCT00134420
- Testing
- Nutropin AQ, Arginine and Clonidine Stimulation Testing, Growth Factors Laboratory Testing
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
Growth Hormone Deficiency
Phase 2 Study of LUM-201 in Children With Growth Hormone Deficiency (OraGrowtH210 Trial)
This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD) and investigate a predictive enrichment marker (PEM) strategy to select subjects…
- Trial name
- OraGrowtH210 (NCT04614337)
- Testing
- LUM-201, rhGH Norditropin® pen (34 µg/kg)
- Where
- 43 sites in United States, Australia and 4 more
- Volunteers
- 104 people
- Sponsor
- Lumos Pharma
Congenital Hypothyroidism
Quality of Life and Long-term Outcome of Adequately Treated Congenital Hypothyroidism
The primary objective of this observational study is the evaluation of the quality of life and long-term outccome of young-adult patients with congenital hypothyroidism diagnosed by neonatal screening receiving replacement…
- Trial name
- CHQoL (NCT06864039)
- Testing
- SF36 test, WAIS IV test, SCL-90 psychopathological screening questionnaire
- Where
- Italy
- Volunteers
- 150 people
- Sponsor
- Federico II University
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
Idiopathic Short Stature
A 4 Year Combination Therapy of Growth Hormone and (GnRH) Agonist in Children With a Short Predicted Height
Estrogens are responsible for the disappearance of growth cartilage in the long bones at the end of the pubertal growth spurt both in boys and in girls.
- Trial name
- ZomaTrip (NCT00840944)
- Testing
- somatropin, triptorelin
- Where
- 6 sites in Belgium
- Volunteers
- 44 people
- Sponsor
- Belgian Study Group for Pediatric Endocrinology
Congenital Hypothyroidism
Congenital Hypothyroidism in Children With Eutopic Gland or Thyroid Hemiagenesis: Predictive Factors for Transient vs Permanent Hypothyroidism.
In France, the incidence of congenital hypothyroidism has increased significantly since the newborn screening program was introduced in 1978. The largest increase is seen in children with eutopic thyroid gland.
- Trial name
- NCT04712760
- Where
- France
- Volunteers
- 60 people
- Sponsor
- Central Hospital, Nancy, France
Growth Hormone Deficiency
Exploring Interactions Between Growth Hormone and the Microbiome
The goal of this study is to characterize the microbiome and metabolomics of children with Growth Hormone (GH) deficiency, prior GH treatment initiation and compare it to healthy children
- Trial name
- NCT03327207
- Testing
- Non Interventional
- Where
- Israel
- Volunteers
- 200 people
- Sponsor
- Rambam Health Care Campus
Growth Hormone Deficiency, Panhypopituitarism
Growth Hormone and Endothelial Function in Children
Objective: This study is designed to determine whether growth hormone treatment in children 8 to 18 years of age alters function of the lining of the arteries.
- Conditions
- Growth Hormone Deficiency, Panhypopituitarism, Short Stature
- Trial name
- NCT00373386
- Testing
- growth hormone
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Ohio State University
Short Stature, Short; Stature, Psychosocial
Short Stature and Psychological Well-being
The first aim of the present study is to evaluate the psychological impact of the condition of short stature (family) in a sample of Italian children, comparing them with subjects of normal stature, measuring their levels of…
- Trial name
- PSICOSHORT (NCT06295341)
- Testing
- Recombinant human growth hormone (only for children with growth hormone deficiency)
- Where
- Italy
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Istituto Auxologico Italiano
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
Short Stature
Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
This is an open-label, single-arm prospective pilot study to study the effects of a single dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with Aggrecan deficiency.
- Trial name
- NCT03288103
- Testing
- Norditropin
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Growth Hormone Deficiency
Use of miRNAs in Growth Hormone Deficiency (GHD)
This study aims at improving knowledge about the diagnosis of growth hormone deficiency (GHD) and treatment with growth hormone (GH), with the goal of providing information on the presence of new biomarkers, such as miRNAs, for…
- Trial name
- GH-miRNA2 (NCT06455956)
- Where
- Italy
- Volunteers
- 400 people
- Sponsor
- University of Parma
Short Stature, Growth Hormone Deficiency
The IGFBP-3 Stimulation Test: A New Tool for the Diagnosis of Growth Hormone Deficiency in Children.
This project is designed to answer the question: Is there an acute IGFBP-3 response in normal children? Our specific hypothesis states that under the influence of growth hormone secretagogues, intact IGFBP-3 molecule will undergo…
- Trial name
- NCT00235599
- Testing
- IGFBP-3 Stimulation Test
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
Achondroplasia
A Study to Learn About Recifercept in Patients With Achondroplasia
The purpose of this study is to learn about the study medicine (called recifercept) in people with achondroplasia. Achondroplasia is a very rare disease and patients of achondroplasia have short arms and legs.
- Trial name
- NCT05659719
- Testing
- Recifercept
- Where
- United States
- Volunteers
- 248 people
- Sponsor
- Pfizer
Idiopathic Short Stature
A Study (Phase 1b/2) of GenSci134 in Children With Idiopathic Short Stature (ISS)
This study comprises two phases: Phase Ib and Phase II. Phase Ib is a multicenter, randomized, open-label, active-controlled, single-dose, dose-escalation study to evaluate the safety, tolerability, PK/PD profile, and…
- Trial name
- NCT07450053
- Testing
- GenSci134 Injection, Recombinant Human Growth Hormone Injection(Norditropin® FlexPro®)
- Where
- China
- Volunteers
- 128 people
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Achondroplasia
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
The purpose of this clinical trial is to evaluate efficacy and safety of once weekly subcutaneous (SC) doses of navepegritide 100 μg/kg compared to placebo (inactive drug) in adolescents aged 12 to 18 years with Achondroplasia.
- Trial name
- teACH (NCT06732895)
- Testing
- Navepegritide
- Where
- 5 sites in Canada, Denmark and 3 more
- Volunteers
- 25 people
- Sponsor
- Ascendis Pharma A/S
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
Hypogonadism, Precocious Puberty
Study of Gonadotropin-Releasing Hormone Pulse Frequency in Sexual Maturation and in the Menstrual Cycle
OBJECTIVES: I. Evaluate the sleep-entrained patterns of gonadotropin-releasing hormone (GnRH) and sex steroid secretion in normal and hypogonadal children. II.
- Trial name
- NCT00004335
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Center for Research Resources (NCRR)