Clinical trials: Natural History
Metachromatic Leukodystrophy
The Natural History of Metachromatic Leukodystrophy
There have not been longitudinal studies which track patients' neurologically or developmentally in a systematic manner. By simultaneously tracking patients' neurodevelopment along with neuroimaging and neurophysiologic studies…
- Trial name
- NH-US (NCT00639132)
- Sponsor
- University of Pittsburgh
Cystinosis
Use of Cysteamine in the Treatment of Cystinosis
Cystinosis is an inherited disease resulting in poor growth and kidney failure. There is no known cure for cystinosis, although kidney transplantation may help the renal failure and prolong survival.
- Trial name
- NCT00359684
- Testing
- Cysteamine
- Where
- United States
- Volunteers
- 330 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Usher Syndromes
Natural History Study of Usher Syndrome ( Light4Deaf )
Clinical centres in the LIGHT4DEAF consortium have developed and will continue to improve a reliable, early molecular diagnosis and protocols for full clinical characterisation of Usher syndrome, which will be valuable for the…
- Trial name
- Light4Deaf (NCT04665726)
- Where
- 4 sites in France
- Volunteers
- 400 people
- Sponsor
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
Parkinson Disease, Idiopathic Parkinson Disease
The Natural History of Parkinson's Disease-associated Spinal Disorders
This is a prospective single-institution longitudinal study to access quality of life and serial imaging and biodynamic testing to assess spinal disorders associated with Parkinson's disease patients.
- Trial name
- NCT04101513
- Where
- United States
- Sponsor
- Ohio State University
Chronic Active Epstein-Barr Virus
Genetic Studies of Chronic Active Epstein-Barr Disease
Epstein-Barr virus (EBV) is a member of the human herpes virus family that infects more than 95 percent of the U.S. population. Most infections occur in childhood and cause no symptoms; in adolescents and adults, EBV often causes…
- Trial name
- NCT00032513
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immunodeficiency
Sample Collection for Systems Evaluation of Patients With Unknown or Incompletely Characterized Immune Defects
Background: The immune system defends the body against disease. It has many different parts spread out in the body, including in the blood and skin.
- Trial name
- NCT04408950
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sensorineural Hearing Loss, Bilateral, AUNB1
Natural History in Children up to 16 Years With Mild to Profound Hearing Loss Due to Mutations in GJB2 / OTOF Genes
The purpose of this study is to follow the natural history of non-syndromic hearing loss caused by mutations in two genes (GJB2 or OTOF) in children up to 16 years of age.
- Conditions
- Sensorineural Hearing Loss, Bilateral, AUNB1, DFNB1A, Congenital Deafness
- Trial name
- Otoconex (NCT05402813)
- Testing
- Pure Tone Audiometry Assessment, Quality of Life Questionnaires
- Where
- France
- Volunteers
- 180 people
- Sponsor
- Sensorion
Healthy Volunteer
Studies of Blood Flow to the Brain During Thought
The purpose of this study is to use brain imaging technology to measure changes in blood flow to areas in the brain as individuals perform intellectual tasks.
- Trial name
- NCT00001360
- Where
- United States
- Volunteers
- 4,100 people, healthy volunteers welcome
- Sponsor
- National Institute of Mental Health (NIMH)
Mood Disorders, Anxiety Disorders
Evaluation of Patients With Mood and Anxiety Disorders and Healthy Volunteers
The purpose of this protocol is to allow for the careful screening of patients and healthy volunteers for participation in research protocols in the Experimental Therapeutics and Pathophysiology Lab (ETPB) at the National…
- Conditions
- Mood Disorders, Anxiety Disorders, Healthy Volunteers, Bipolar Disorder
- Trial name
- NCT00024635
- Where
- United States
- Volunteers
- 16,000 people, healthy volunteers welcome
- Sponsor
- National Institute of Mental Health (NIMH)
Pulmonary Hypertension, Sickle Cell Anemia
Secondary Pulmonary Hypertension in Adults With Sickle Cell Anemia
The purpose of this study is to determine how often people with sickle cell anemia develop pulmonary hypertension a serious disease in which blood pressure in the artery to the lungs is elevated.
- Conditions
- Pulmonary Hypertension, Sickle Cell Anemia, Sickle Cell Disease
- Trial name
- NCT00011648
- Where
- 2 sites in United States
- Volunteers
- 986 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Inflammatory Bowel Disease, Ulcerative Colitis
Immune Regulation in Ulcerative Colitis or Crohn s Disease
This study will investigate in patients with Crohn s disease and ulcerative colitis how the body s immune system controls inflammation in the gastrointestinal tract (stomach and intestines)-specifically, how lymphocytes (a type…
- Conditions
- Inflammatory Bowel Disease, Ulcerative Colitis, Chrohn's Disease
- Trial name
- NCT00001184
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Sickle Cell Disease (SCD)
Early Diagnosis of Sickle Acute Chest Syndrome Using a Combination of Plasma Bimarkers and Chest Imaging
Background: Painful vasoocclusive crisis (VOC) occurs in people with sickle cell disease (SCD). People with VOC have many visits to the hospital.
- Trial name
- NCT03478917
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Healthy Volunteer, Traumatic Brain Injury
Estimating Brain Biomechanics Using MRI
Objective: In this study we will develop and apply imaging techniques to perform the first three-dimensional (3-D) measurements of brain biomechanics during mild head movement in healthy human subjects.
- Conditions
- Healthy Volunteer, Traumatic Brain Injury, Brain Mapping, Craniocerebral Trauma
- Trial name
- NCT01633268
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Rheumatoid Arthritis
Study of Arthritis and Related Conditions
This research protocol will recruit patients with arthritis and related conditions for the purpose of screening patients for treatment protocols, and evaluating the natural history of arthritis and related conditions.
- Trial name
- NCT00006333
- Where
- United States
- Volunteers
- 697 people
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
A Retrospective, Natural History Study in Children With CLN2
This is a multi center, retrospective, chart review study to document the evolution of ocular disease progression in pediatric patients with CLN2.
- Trial name
- NCT04480476
- Sponsor
- REGENXBIO Inc.
Healthy Volunteers
Study of Skin Cells That Stop Replicating (Senescent) During Wound Healing
Background: Cellular senescence is the aging of cells. It is a complex process that may be connected with aging and age-related diseases.
- Trial name
- NCT02755584
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- National Institute on Aging (NIA)
Tuberous Sclerosis Complex
Tuberous Sclerosis Complex Natural History Study: Renal Manifestations
Eighty percent of patients with tuberous sclerosis complex (TSC) have renal angiomyolipomata. These lesions grow and can lead to significant morbidity by hemorrhage or renal failure.
- Trial name
- NCT00598455
- Where
- United States
- Volunteers
- 450 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Hereditary Inclusion Body Myopathy, GNE Myopathy
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and…
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM)
- Trial name
- NCT01784679
- Where
- 5 sites in United States, Bulgaria and 3 more
- Volunteers
- 319 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Stargardt Disease
A Natural History of the Progression of Stargardt Disease: Retrospective and Prospective Studies
Stargardt disease is currently an incurable and untreatable macular dystrophy that causes severe visual loss in children and young adults, thereby causing enormous morbidity with economic, psychological, emotional, and social…
- Trial name
- ProgSTAR (NCT01977846)
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 259 people
- Sponsor
- Foundation Fighting Blindness
Pregnancy Outcome
NIH Intramural Research Program s Pregnancy Registry Protocol for Subjects and Their Partners
Background: In 2018, the NIH intramural research program had almost 23,000 people taking part in active clinical research studies. Half of those people were female. More than a third were of child-bearing age.
- Trial name
- NCT04977466
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
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