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Found 39 clinical trials
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Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.

This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries. Information collected will include in anthropometric characteristics, related symptoms, tests, & treatments Children's information will be collected in the registry for a maximum of 5 years.

  • 27 views
  • 20 Jun, 2021
  • 52 locations
None
A Multi-center Longitudinal Observational Study of Children With Achondroplasia

This is a long-term, multi-center, longitudinal, observational study in children with achondroplasia (ACH). The aim is to study height velocity and comorbidities in children with ACH. This is a natural history study and no study medication will be administered.

  • 36 views
  • 18 Feb, 2021
  • 29 locations
None
Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)

This is a long-term, multi-center, observational study in children 2.5 to 10 years with achondroplasia (ACH). The objective is to evaluate growth, ACH-related medical complications and treatments of study participants. No study medication will be administered.

  • 11 views
  • 07 Oct, 2021
  • 19 locations
None
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.

  • 0 views
  • 25 Mar, 2021
  • 1 location
None
Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum Cockayne Syndrome or Trichothiodystrophy

Four rare genetic diseases, xeroderma pigmentosum (XP), Cockayne syndrome (CS), the XP/CS complex and trichothiodystrophy (TTD) have defective DNA excision repair although only XP has increased cancer susceptibility. We plan to perform careful clinical examination of selected patients with XP, XP/CS, CS, or TTD and follow their clinical course. We …

cancer
cancer prevention
Accepts healthy volunteers
  • 59 views
  • 31 Oct, 2021
  • 1 location
None
Long-term Safety and Effectiveness of Growth Hormone With GHD TS CRF SGA ISS and PWS in Children

Syndrome),CRF (Chronic Renal Failure), SGA (Small for Gestational Age), and ISS (Idiopathic Short Stature).

renal failure
somatotropin
turner syndrome
hormone deficiency
deficiency
  • 306 views
  • 28 Feb, 2021
  • 1 location
None
The Myelin Disorders Biorepository Project

The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to support ongoing and future research projects. The MDBP is one of the world's largest leukodystrophy biorepositories, having enrolled nearly 2,000 affected individuals since it was launched over a decade …

  • 30 views
  • 12 Sep, 2021
  • 10 locations
None
BBD Longitudinal Study of Osteogenesis Imperfecta

may have bowed arms and legs, very short stature and be unable to walk. People with the mildest form of OI may only break bones occasionally and have normal height and lifespan. People with OI also

bone disease
dental exam
scoliosis
col1a2
short stature
  • 113 views
  • 24 Jan, 2021
  • 10 locations
None
Natural History Study of Patients With Excess Androgen

describe problems associated with it. Too much androgen in childhood, if untreated, results in rapid growth and early puberty with early cessation of growth and short stature in adulthood. Too much

fasting
other hormones
testicular
21-ohd
deficiency
  • 0 views
  • 08 Dec, 2021
  • 2 locations
None
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases. This program allows patients and researchers to connect as easily as possible to help advance treatments and cures …

rare diseases
other disease
  • 4 views
  • 08 Sep, 2021
  • 2 locations