Clinical trials: Bone Diseases, Endocrine
Congenital Hypothyroidism, Hearing Loss
The Prevalence of Hearing Loss Among Children With Congenital Hypothyroidism
Congenital hypothyroidism (CH) occurs in 1:4000 live births. Neurological disturbances like speech delay, motor delay and lower IQ were reported in children with CH.
- Trial name
- NCT01223638
- Testing
- Audiometry
- Where
- Israel
- Volunteers
- healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
End Stage Renal Disease, Hyperparathyroidism, Secondary
Mineral and Bone Disorders Outcomes in Stage 5D of Chronic Kidney Disease
The MBD-5D is a prospective observational study with a case-cohort and a cohort design. Eligible patients are receiving hemodialysis and have secondary hyperparathyroidism.
- Trial name
- MBD-5D (NCT00995163)
- Where
- Japan
- Volunteers
- 8,020 people
- Sponsor
- Kyowa Kirin Co., Ltd.
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
Achondroplasia
A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia
Study 111-209 is a Phase 2 randomized, open-label clinical trial of BMN 111 in infants and young children with a diagnosis of Achondroplasia at a heightened risk of requiring cervicomedullary decompression surgery
- Trial name
- NCT04554940
- Testing
- vosoritide
- Where
- 3 sites in Australia, United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Growth Hormone Deficiency
Comparison of a Needle-free Injection Method With a Needle-syringe Injection Method
The purpose of this study is to obtain psychological response and user preference information on the use of the T jet® device versus the traditional subcutaneous injection administration of Tev Tropin®.
- Trial name
- T-jet® (NCT00990340)
- Testing
- T-jet® containing TevTropin®, TevTropin® needle-syringe injection method
- Volunteers
- 52 people
- Sponsor
- Teva Branded Pharmaceutical Products R&D, Inc.
Chronic Myeloproliferative Disorders, Leukemia
A Web-Based Stem Cell Transplant Support System or Standard Care in Young Patients Undergoing Stem Cell Transplant and Their Families
RATIONALE: A Web site for stem cell transplant health information and support may be effective in helping parents improve their health-related knowledge, skills, and quality of life, which may also improve their children's…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00782145
- Testing
- educational intervention, informational intervention, internet-based intervention
- Where
- 6 sites in United States
- Volunteers
- 198 people
- Sponsor
- Tufts Medical Center
Solid Tumors, CNS Tumors
Study Of Entrectinib (Rxdx-101) in Children and Adolescents With Locally Advanced Or Metastatic Solid Or Primary CNS Tumors And/Or Who Have No Satisfactory Treatment Options
This is an open-label, Phase 1/2 multicenter dose escalation study in pediatric patients with relapsed or refractory extracranial solid tumors (Phase 1), with additional expansion cohorts (Phase 2) in patients with primary brain…
- Trial name
- STARTRK-NG (NCT02650401)
- Testing
- Entrectinib
- Where
- 26 sites in United States, Canada and 7 more
- Volunteers
- 69 people
- Sponsor
- Hoffmann-La Roche
Panhypopituitarism, Gigantism/Acromegaly
An Investigation of Pituitary Tumors and Related Hypothalmic Disorders
There is a variety of tumors affecting the pituitary gland in childhood; some of these tumors (eg craniopharyngioma) are included among the most common central nervous system tumors in childhood.
- Conditions
- Panhypopituitarism, Gigantism/Acromegaly, Prolactinoma, Cushing Disease
- Trial name
- NCT00001595
- Testing
- Tissue specimen collection, MRI
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Marfan Syndrome, Turner Syndrome
National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions
The National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (GenTAC) was initiated in 2006 by the National Heart, Lung, and Blood Institute (NHLBI) and the National Institute of…
- Conditions
- Marfan Syndrome, Turner Syndrome, Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome
- Trial name
- GenTAC (NCT01322165)
- Where
- 7 sites in United States
- Volunteers
- 3,706 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Turner Syndrome, Short Stature
D3-GHR Polymorphism and Turner Syndrome
The protein polymorphism of the growth hormone receptor characterized by the genomic deletion of exon 3 has been linked to the magnitude of the first-year-growth response to growth hormone (GH) in girls with Turner syndrome.
- Trial name
- NCT00443144
- Testing
- recombinant human growth hormone
- Where
- Germany
- Sponsor
- University Hospital Tuebingen
Pituitary Disease
Safety and Efficacy of Pegvisomant in Children With Growth Hormone Excess
Background: For children with gigantism, too much growth hormone (GH) in the body causes abnormal growth and many other problems. Current treatments often don t work; no medical treatment is approved by FDA.
- Trial name
- NCT03882034
- Testing
- Pegvisomant
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Short Stature
Vosoritide for Selected Genetic Causes of Short Stature
Short stature can be caused by a number of genetic etiologies, many of which directly affect the growth plate. The FGFR3/CNP pathway is central to growth of the chondrocyte.
- Trial name
- NCT04219007
- Testing
- Vosoritide
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Andrew Dauber
Cancer
Captopril in Treating Patients Undergoing Bone Marrow or Stem Cell Transplantation
RATIONALE: Captopril may protect the lungs from the side effects of bone marrow or stem cell transplantation. PURPOSE: Randomized phase III trial to determine the effectiveness of captopril to lessen the side effects in patients…
- Trial name
- NCT00004230
- Testing
- captopril, cyclophosphamide, autologous bone marrow transplantation
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Northwestern University
Growth Hormone Deficiency, Endocrine System Diseases
Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency
This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH)…
- Conditions
- Growth Hormone Deficiency, Endocrine System Diseases, Hormones, Pituitary Diseases
- Trial name
- NCT04326374
- Testing
- TransCon hGH, daily hGH
- Where
- China
- Volunteers
- 150 people
- Sponsor
- Visen Pharmaceuticals (Shanghai) Co., Ltd.
Growth Hormone Disorder, Pituitary Dwarfism
Somatropin (Norditropin) in Insulin-like Growth Factor (IGF) Deficient Children
This trial is conducted in the United States of America (USA). This is a 12 month study to determine if Norditropin is safe and effective in children with IGF deficiency.
- Trial name
- NCT00102817
- Testing
- somatropin
- Where
- 27 sites in United States
- Volunteers
- 149 people
- Sponsor
- Novo Nordisk A/S
Breast Cancer, Prostate Cancer
Tempus Priority Study: A Pan-tumor Observational Study
Observational study that will be collecting clinical and molecular health information from cancer patients who have received comprehensive genomic profiling and meet the specific eligibility criteria outlined for each cohort with…
- Conditions
- Breast Cancer, Prostate Cancer, Bladder Cancer, Lung Cancer
- Trial name
- NCT05179824
- Testing
- Observation
- Where
- 2 sites in United States
- Volunteers
- 1,000 people
- Sponsor
- Tempus AI
Cancer
Laboratory Assay in Determining Cancer Resistance in Patients With Metastatic Cancer and in Healthy Participants
RATIONALE: Studying samples of blood and tissue in the laboratory from patients with cancer and from healthy participants may help doctors learn more about cancer.
- Trial name
- NCT00899496
- Testing
- immunological diagnostic method, physiologic testing
- Where
- United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Prader-Willi Syndrome, Growth Hormone Deficiency
Is There a Sensibility Increased in the Growth Hormone at Child With Prader-Willi Syndrome?
The purpose of this study is to estimate the sensibility at the growth hormone in vivo at the children presenting a Prader-Willi syndrome (SPW) in comparison with children presenting a deficit in growth hormone (GHD).
- Trial name
- NCT01298180
- Testing
- Growth hormone (Genotonorm® or Omnitrope®), DEXA, blood tests, H.G.P.O, osseous age., biopsy
- Where
- 26 sites in France
- Volunteers
- 111 people
- Sponsor
- University Hospital, Toulouse
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