Clinical trials: Natural History
Graft Versus Host Disease
Chronic Graft-versus-Host Disease in the Oral Cavity of Patients Following Allogeneic Hematopoietic Stem Cell Transplant and Including Healthy Controls
Background: This study follows people who have had, or will soon have, a transplant using stem cells from another person. This is known as an allogeneic hematopoietic stem cell transplant (HSCT).
- Trial name
- NCT03602599
- Where
- United States
- Volunteers
- 190 people
- Sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
Psoriasis
Genetic Analysis of Psoriasis and Psoriatic Arthritis
This study will examine the genetic basis of psoriasis and psoriatic arthritis. It is known that genes play an important role in determining who gets psoriasis or psoriatic arthritis.
- Trial name
- NCT00341809
- Where
- United States
- Volunteers
- 2,653 people
- Sponsor
- National Cancer Institute (NCI)
Sanfilippo Syndrome Type A
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including…
- Trial name
- NCT01047306
- Testing
- assessment
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Shire
Chronic Kidney Disease
Vascular Changes Following Forearm Loop Arteriovenous Graft Placement
This study will determine whether upper arm vessels increase in size following forearm loop arteriovenous graft placement and the timing of these changes.
- Trial name
- NCT00836563
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of Nebraska
Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa), Obstructive Sleep Apnea
Evaluating the Genetics and Immunology of Periodic Fever, Aphthous Stomatitis, Pharyngitis, and Cervical Adenitis (PFAPA) Syndrome and Other Tonsil Disorders
Background: Periodic fever, aphthous stomatitis, pharyngitis, and cervical adenitis (PFAPA) is the most common periodic fever syndrome of childhood.
- Conditions
- Periodic Fever, Aphthous Stomatitis, Pharyngitis, And Cervical Adenitis (Pfapa), Obstructive Sleep Apnea, Tonsillitis, Tonsil Disorder
- Trial name
- NCT05656365
- Where
- 3 sites in United States
- Volunteers
- 1,500 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Acute Ischemic Stroke, Hemorrhagic Stroke
Mapping the Natural History of Parenychymal and Cerebral Perfusion Changes in Acute Ischemic and Hemorrhagic Strokes
Study using ultrasound of the brain as a diagnostic tool to differentiate between ischemic stroke and hemorrhagic stroke. Correlation of brain perfusion and size of stroke in relation to systemic hemodynamic targets will be…
- Trial name
- NCT05865795
- Where
- United States
- Volunteers
- 83 people
- Sponsor
- Wake Forest University Health Sciences
Dermatologic Diseases, Systemic Diseases With Cutaneous Manifestations
Evaluation and Treatment of Patients With Dermatologic Diseases
This is a training, natural history of disease, and screening protocol for the evaluation, treatment and follow-up of patients with dermatologic diseases and systemic diseases with cutaneous manifestations.
- Trial name
- NCT00001506
- Where
- United States
- Volunteers
- 453 people
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Leukodystrophies
Natural History of the Leukodystrophies
The purpose of this study is to: 1. define novel homogeneous groups of patients with LDs and 2. work toward finding the cause of these disorders.
- Trial name
- NCT02843555
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor Research Institute
Sensorineural Hearing Loss, Cytomegalovirus Infection
Clinical and Genetic Analysis of Enlarged Vestibular Aqueducts
This study will try to identify and understand the genetic factors that lead to an inner ear malformation called "enlarged vestibular aqueducts", that can be associated with hearing loss.
- Trial name
- NCT00023036
- Where
- United States
- Volunteers
- 324 people
- Sponsor
- National Institute on Deafness and Other Communication Disorders (NIDCD)
Hepatitis C, Anemia
A Study to Evaluate the Erythropoietic Response in Hepatitis C Virus (HCV) Patients Receiving Combination Ribavirin (RBV)/Interferon (IFN) Therapy or RBV/PEG IFN (NATURAL HISTORY-HCV)
The purpose of this study was to describe the time course and extent of hemoglobin (Hb) changes and the erythropoietic response to PEG-IFN/RBV-induced anemia In HCV-infected subjects.
- Trial name
- NCT00328549
- Testing
- Pegylated Interferon and Ribavirin
- Volunteers
- 105 people
- Sponsor
- Ortho Biotech Products, L.P.
Inflammation
Evaluation and Long-Term Follow-up of Patients With Inflammatory Disorders
This study will investigate inflammatory diseases and disorders. Inflammation is the body s immune response to many things, including infections and other acute or chronic irritants.
- Trial name
- NCT00001351
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Geographic Atrophy
Evolution and Risk Factors Associated With Geographic Atrophy Progression
Age-related macular degeneration is one of the leading causes of blindness worldwide. The factors that induce the progression of geographic atrophy, the advanced form of dry age-related macular degeneration, remain poorly…
- Trial name
- GAIN (NCT01694095)
- Where
- Spain
- Volunteers
- 96 people
- Sponsor
- Institut de la Macula y la Retina
Cancer, Childhood Malignancy
Radiation Therapy and the Natural History of Childhood Cancers
This study will: 1) provide standard non-experimental radiation therapy to children who have a form of cancer or similar disease process that is of scientific interest, importance, or educational value; 2) determine the effects…
- Trial name
- NCT00064883
- Where
- United States
- Volunteers
- 241 people
- Sponsor
- National Cancer Institute (NCI)
Fabry Disease
Natural History in Fabry Disease With IVS4+919G>A Mutations
Fabry disease (FD) is a genetic condition where mutations in the GLA gene cause low activity of an important enzyme (alpha-Galactosidase A). This leads to harmful substances building up in the body.
- Trial name
- NCT07506083
- Where
- Hong Kong
- Volunteers
- 31 people
- Sponsor
- Chinese University of Hong Kong
Williams Syndrome, Supravalvular Aortic Stenosis
Williams Syndrome (WS) and Supravalvar Aortic Stenosis (SVAS) DNA and Tissue Bank
Williams syndrome is a rare genetic disorder occurring in 1:8000-12,000 individuals. It is caused by the deletion of 25-27 coding genes, including elastin (ELN) on the 7th human chromosome.
- Conditions
- Williams Syndrome, Supravalvular Aortic Stenosis, Cardiovascular Disease
- Trial name
- NCT02706639
- Where
- 3 sites in United States
- Volunteers
- 305 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
X-Linked Retinitis Pigmentosa
Natural History of the Progression of X-Linked Retinitis Pigmentosa
The objective of the study is to gain a better understanding of disease progression over time in participants with X-linked retinitis pigmentosa (XLRP).
- Trial name
- XOLARIS (NCT04926129)
- Testing
- Other: Assessments
- Where
- 22 sites in United States, Canada and 5 more
- Volunteers
- 201 people
- Sponsor
- NightstaRx Ltd, a Biogen Company
X-linked Myotubular Myopathy
Myotubular Myopathy Event Study
X-Linked myotubular myopathy (XLMTM), a form of centronuclear myopathy (CNM) is the result of a mutation in the MTM1 (myotubularin) gene which leads to altered myotubularin. Myotubularin is essential for optimum muscle function.
- Trial name
- MTMES (NCT01840657)
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Cure CMD
Healthy Volunteer, Magnetic Resonance Imaging
Study of New Magnetic Resonance Imaging Methods of the Brain
The purpose of this investigation is to develop improved magnetic resonance imaging (MRI) techniques and hardware for studying brain function.
- Conditions
- Healthy Volunteer, Magnetic Resonance Imaging, Healthy, fMRI
- Trial name
- NCT00004577
- Where
- United States
- Volunteers
- 1,100 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
PGM3 Deficiency, Eosinophilic and/or Atopic Dermatitis
Natural History of Severe Allergic Inflammation and Reactions
Background: Allergic inflammation is central to allergy-related diseases and disorders, such as asthma, food allergies, and atopic dermatitis.
- Conditions
- PGM3 Deficiency, Eosinophilic and/or Atopic Dermatitis, OSMR Deficiency, Primary Localized Cutaneous Amyloidosis
- Trial name
- NCT01164241
- Where
- United States
- Volunteers
- 945 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathy IV
A Natural History Study of Charcot-Marie-Tooth 4J (CMT4J)
Charcot-Marie-Tooth 4J (CMT4J) is a rare inherited peripheral neuropathy often characterized by rapidly progressive, asymmetrical upper and lower extremity weakness, muscle atrophy leading to loss of ambulation, respiratory…
- Trial name
- CMT4J (NCT03810508)
- Where
- 2 sites in United States
- Volunteers
- 21 people
- Sponsor
- Neurogene Inc.
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