Clinical trials: Dwarfism, open to volunteers
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
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
Safety, Tolerability
A Clinical Study of GenSci134 in Healthy Adults and Adult Growth Hormone Deficiency(AGHD)
To check how safe and well-tolerated a single subcutaneous injection of GenSc134 is in healthy male volunteers， a multiple-doses subcutaneous injection of GenSc134 is in healthy volunteers，a single subcutaneous injection of…
- Conditions
- Safety, Tolerability, Adult Growth Hormone Deficiency
- Trial name
- NCT07016802
- Testing
- GenSci134, Recombinant Human Growth Hormone Injection (Norditropin®)
- Where
- China
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Changchun GeneScience Pharmaceutical Co., Ltd.
Oral Manifestations, Short Stature
Oral Manifestations in Egyptian Children With Short Stature
Children with short stature have been reported to show a higher prevalence of enamel hypoplasia, delayed tooth eruption, and malocclusion compared to children of normal growth status
- Trial name
- NCT06372366
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Cairo 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
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
Congenital Hypothyroidism
Infants With Primary Congenital Hypothyroidism and Development
A review of the literature has focused primarily on the cognitive and motor development of children with congenital hypothyroidism, but no studies on sensory processing skills have been found.
- Trial name
- NCT07280104
- Testing
- neurological status, sensory processing functions, Motor development
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Nigde Omer Halisdemir University
Taybi Linder Syndrome, Microcephalic Osteodysplastic Primordial Dwarfism Types I and III
Study of the Pathophysiology of RNU4ATAC and RTTN Associated Syndromes
In the human genome, about 750 genes contain one intron excised by the minor spliceosome. These genes are named U12 genes, and these introns, minor or U12 introns.
- Conditions
- Taybi Linder Syndrome, Microcephalic Osteodysplastic Primordial Dwarfism Types I and III, Roifman Syndrome, Lowry Wood Syndrome
- Trial name
- ATAC (NCT06111950)
- Testing
- Blood samples, Skin biopsies, Fetal samples
- Where
- 6 sites in France
- Volunteers
- 45 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Chromosome Aberrations, Growth Hormone Deficiency
Chromosome 18 Clinical Research Center
Our vision, that of the researchers at the University of Texas Health Science Center at San Antonio, is that every person with a chromosome 18 abnormality will have an autonomous and healthy life.
- Conditions
- Chromosome Aberrations, Growth Hormone Deficiency, Hypomyelination
- Trial name
- Chromosome18 (NCT00227253)
- Testing
- Determination of growth hormone status, Measurement of growth, thyroid and sex hormone levels, Behavior and neuropsychometric evaluations
- Where
- 2 sites in United States
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center at San Antonio
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)
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
Healthy, Growth Hormone Deficiency
Effects of GH and Lirglutide on AgRP
This is a randomized, placebo-controlled, cross-over study with 4 arms. Healthy and GH deficient adults ages 18-45 years will be studied. Arms will consist of 21-day treatment periods and be separated by 8-week washout periods.
- Trial name
- NCT05681299
- Testing
- growth hormone and lirglutide, growth hormone, liraglutide
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Columbia University
Statin Adverse Reaction
Exercise Intensity and Muscle Recovery in Statin Users
This study aims to investigate how different types and intensities of exercise affect muscle recovery in individuals taking statin medications compared to healthy controls.
- Trial name
- STN-EX (NCT07563452)
- Testing
- Dose-Escalation Exercise Protocol
- Where
- Austria
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Raffaele Mazzolari