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Found 39 clinical trials
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Theophylline Treatment for Pseudohypoparathyroidism

Pseudohypoparathyroidism is a genetic disorder with limited treatment options. Patients have early-onset obesity, short stature and increased risk of type 2 diabetes. This phase 2 clinical trial

diabetes
  • 51 views
  • 14 Aug, 2021
  • 1 location
None
Theophylline Treatment for Pseudohypoparathyroidism - Children 2-12 Years Old

Pseudohypoparathyroidism is a genetic disorder with limited treatment options, characterized by early-onset obesity, short stature and resistance to multiple hormones. This phase 2 clinical

  • 0 views
  • 07 Oct, 2021
  • 1 location
None
Intervention in Chronic Pediatric Patients and Their Families.

This project consists of a psychological intervention in patients and their families with different chronic diseases in order to carry out a comparative study between medical pathologies to know which are the protective or risk variables for the adaptation to the disease.

chronic diseases
chronic disease
  • 0 views
  • 24 Jan, 2021
None
Clinical Trial in 22q13 Deletion Syndrome(Phelan-McDermid Syndrome)

short stature due to primary IGF-1 deficiency. It is being used off-label in the current study and is not FDA approved, nor has it yet been studied in humans for the treatment of SHANK3 deficiency.

  • 6 views
  • 23 Mar, 2021
  • 1 location
None
Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome

This study evaluate the efficacy of 3-hydroxy-3-methylglutaryl coenzyme A (HMG-COA) reductase inhibitors, also known as "statins" in the treatment of growth and skeletal abnormalities in children with Noonan syndrome. Half of patients will receive simvastatin while the other half will receive a placebo.

  • 29 views
  • 26 Jan, 2021
  • 20 locations
None
Safety of Fresolimumab in the Treatment of Osteogenesis Imperfecta

Osteogenesis Imperfecta (OI) is a rare disorder that causes bones to break easily. People with OI may have broken bones with little or no trauma, dentinogenesis imperfecta (DI), and, in adult years, hearing loss. OI can range from very severe to very mild. The current standard-of-care for severe types of …

col1a1
hearing loss
birth control method
rare disorder
bisphosphonate
  • 146 views
  • 13 Apr, 2021
  • 2 locations
None
PREPP: Preventing Postpartum Depression

The primary aim of this study is to determine if a behavioral intervention targeting maternal caregiving of young infants can increase infant sleep and reduce fuss/cry behavior, and thereby (1) reduce the incidence and/or severity of postpartum maternal depression and (2) improve the quality of the mother-infant interaction and subsequent …

psychotherapy
behavior therapy
prenatal
depressed mood
  • 39 views
  • 23 Mar, 2021
  • 1 location
None
Eltrombopag for People With Fanconi Anemia

Background Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia (low red blood cells) or low platelet counts or bleeding. Researchers want to see if a new …

platelet count
androgens
prbcs
cytopenia
fanca
  • 162 views
  • 25 Nov, 2021
  • 1 location
None
Autologous Gene Therapy for Artemis-Deficient SCID

This study aims to determine if a new method can be used to treat Artemis-deficient Severe Combined Immunodeficiency (ART-SCID), a severe form of primary immunodeficiency caused by mutations in the DCLRE1C gene. This method involves transferring a normal copy of the DCLRE1C gene into stem cells of an affected patient. …

primary immunodeficiency
igiv
stem cell transplantation
bone marrow procedure
undernutrition
  • 0 views
  • 06 Nov, 2021
  • 1 location
None
Study to Examine Insulin Resistance During Growth Hormone Treatment for Short Stature Due to Low Birthweight

Insulin resistance is common among children with low birthweight. Moreover, growth hormone treatment for ensuing short stature also causes insulin resistance. Our objective is to examine

balanced diet
  • 1 views
  • 07 Nov, 2020
  • 1 location