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Found 295 clinical trials
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants (LVXSCID-ND)

SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system. The purpose of this study is to determine if

  • 26 views
  • 25 Mar, 2022
  • 3 locations
Epigenetic Regulation of Osteogenesis Imperfecta Severity : miROI Study (miROI)

Osteogenesis Imperfecta (OI) is a heterogeneous group of rare connective tissue hereditary diseases responsible for fragility and bone deformity. OI is caused by an autosomal dominant mutation

col1a1
autosomal dominant
col1a2
genetic disorder
bone disease
  • 4 views
  • 24 Mar, 2022
  • 1 location
Evaluation of the Benefits of Adaptive Physical Activity in Children and Adolescents With Osteogenesis Imperfecta (MOVE-OI)

Osteogenesis imperfecta (OI) is a rare genetic disorder of increased bone fragility and low bone mass. It is conceivable that children and adolescents with OI are less active than healthy peers

bone mineral density
genetic disorder
bone disease
  • 0 views
  • 20 Feb, 2022
Epigenetic Regulation in Fibrous Dysplasia of Bone: mirDYS Study.

Fibrous dysplasia of bone is a rare congenital but non-hereditary disease caused by a post-zygotic activation mutation of the GNAS gene. Patients with fibrous dysplasia may present pain and bone

  • 0 views
  • 01 Feb, 2021
  • 1 location
MExiletine Versus Lamotrigine in Non-Dystrophic Myotonias

genetic condition for which there is no cure. It affects patients for the duration of their life and impacts work, leisure and can lead to significant morbidity. The study is a cross-over design

  • 0 views
  • 03 Sep, 2021
  • 1 location
Oral Ifetroban in Subjects With Duchenne Muscular Dystrophy (DMD)

inevitably premature death of affected young men in their late twenties. DMD is the most common fatal genetic disorder diagnosed in childhood. It affects approximately 1 in every 3,500 live male births

ejection fraction
beta blockers
aldosterone antagonists
aldosterone
angiotensin
  • 7 views
  • 26 Apr, 2022
  • 10 locations
A Trial of Dabrafenib, Trametinib and Hydroxychloroquine for Patients With Recurrent LGG or HGG With a BRAF Aberration

mutations including BRAF V600 mutations or BRAF fusion/duplication, with or without neurofibromatosis type 1. Neurofibromatosis type 1 is an inherited genetic condition that causes tumors to grow on nerve

  • 4 views
  • 29 May, 2022
  • 9 locations
Interventions for Reading Disabilities in NF1

Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of

attention deficit hyperactivity disorder
lovastatin 20 mg
hearing impairment
stimulant
tenderness
  • 169 views
  • 29 Jun, 2022
  • 1 location
Information About Alzheimer's Disease for Latinos in New York City (IDEAL)

This study will assess the psychosocial and behavioral impacts of receiving Alzheimer's disease genetic risk assessment incorporating APOE genotypes among Latinos in northern Manhattan. The

cognitive assessment
alzheimer's disease
  • 0 views
  • 24 Mar, 2022
  • 1 location
A Trial of Metformin in Individuals With Fragile X Syndrome (Met)

This study is a controlled trial of metformin in individuals with fragile X syndrome between the ages of 6 and 35 years. Participants will be randomized in a double-blind design to either drug or placebo and will attend three visits to the study site in a 4-month period for a …

obesity
metformin
problem behavior
  • 65 views
  • 11 Feb, 2022
  • 1 location