Clinical trials: Natural History
COVID-19
Plasma Collection From Convalescent and/or Immunized Donors for the Treatment of COVID-19
Background: The Coronavirus disease 2019 (COVID-19) pandemic is a major public health issue. Researchers want to collect plasma from people who have recovered from COVID-19, and use this plasma to treat people who are sick with…
- Trial name
- NCT04360278
- Where
- United States
- Volunteers
- 258 people
- Sponsor
- National Institutes of Health Clinical Center (CC)
Endocrine Disease, Infertility
Evaluation of Women With Endocrine and Reproductive-Related Conditions
This study was designed to allow inpatient and outpatient evaluation of women with a variety of reproductive and endocrine-related disorders for purposes of research and physician education.
- Conditions
- Endocrine Disease, Infertility, Leiomyoma, Endometriosis
- Trial name
- NCT00001850
- Where
- United States
- Volunteers
- 833 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Eosinophilic Disease, Immune Deficiency
Screening Protocol for Genetic Diseases of Allergic Inflammation
Background: Mast cells are responsible for most symptoms of allergic reactions. In some allergic diseases, it is unusually easy to cause mast cells to release their contents and cause allergic reactions.
- Conditions
- Eosinophilic Disease, Immune Deficiency, HaTS, Urticaria Anaphylaxis
- Trial name
- NCT00852943
- Where
- United States
- Volunteers
- 834 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Neurogenetic Disorders
Neurogenetics Patient Registry
The objective of this project is to develop a Neurogenetics patient database and bio repository - which includes clinical information regarding history, physical examination, laboratory testing including genetic testing (NextGen…
- Trial name
- NCT02995538
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- University of Pittsburgh
Danon Disease
Natural History of Danon Disease
This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD).
- Trial name
- NCT05548855
- Testing
- Heart Transplant, Cardiac Assistive Device
- Where
- 15 sites in Czechia, France and 3 more
- Volunteers
- 59 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Spinocerebellar Ataxia Type 3, Machado-Joseph Disease
Astrocytic Markers and the Pre-ataxic Period of SCA3/MJD - BIGPRO Study Astrocytes
The study will consist of a prospective observation of subjects in a natural history design. The investigators will monitor changes of clinical scales, quality of life, messenger ribonucleic acid (mRNA) of candidate genes (CCL11…
- Conditions
- Spinocerebellar Ataxia Type 3, Machado-Joseph Disease, SCA3, MJD
- Trial name
- NCT04419974
- Testing
- Molecular Diagnosis, Clinical Scales - Baseline, Blood Draw - Baseline
- Where
- Brazil
- Volunteers
- 95 people, healthy volunteers welcome
- Sponsor
- Hospital de Clinicas de Porto Alegre
Methylmalonic Acidemia, Propionic Acidemia
Natural History Study of Patients With Methylmalonic Acidemia and Propionic Acidemia
The JUMP (Journey to Understand MMA and PA) Study is being conducted by HemoShear Therapeutics and AllStripes, a rare disease online research platform.
- Trial name
- JUMP (NCT05438485)
- Testing
- Registry
- Where
- 3 sites in United States, Canada and 1 more
- Volunteers
- 11 people
- Sponsor
- HemoShear Therapeutics
Developmental Psychology
The Psychobiology of Childhood Temperament
The purpose of this study is to use brain imaging technology to examine brain changes that occur in children when they are exposed to various kinds of emotional tasks and to determine if these changes are related to the child's…
- Trial name
- NCT00060775
- Where
- United States
- Volunteers
- 550 people
- Sponsor
- National Institute of Mental Health (NIMH)
Tuberous Sclerosis
Study of Skin Tumors in Tuberous Sclerosis
Tuberous sclerosis is a rare, hereditary disease in which patients develop multiple tumors. Although not cancerous, the tumors can affect various organs, including the heart, lungs, kidneys, skin, and central nervous system, with…
- Trial name
- NCT00001975
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Chemotherapy-induced Peripheral Neuropathy
Chemotherapy Induced Peripheral Neuropathy Natural History Study (EPIPHANY)
This is a prospective natural history study of CIPN in approximately 200 participants receiving taxanes (paclitaxel, docetaxel) for breast cancer, bortezomib for multiple myeloma, oxaliplatin-based regimens for colorectal cancer…
- Trial name
- EPIPHANY (NCT03997981)
- Where
- 10 sites in United States
- Volunteers
- 200 people
- Sponsor
- Disarm Therapeutics
Heart, Respiratory, Disease, Functional
Natural History of Cardiac and Respiration Function in Patients With Muscular Dystrophies on Home Mechanical Ventilation
Muscular dystrophies are inherited disorders that affect skeletal muscle. Cardiac and respiratory function may be affected in this group of diseases.
- Trial name
- NCT02501083
- Testing
- no intervention
- Where
- France
- Volunteers
- 350 people
- Sponsor
- Centre d'Investigation Clinique et Technologique 805
Myotubular Myopathy
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe.
- Trial name
- MTM (NCT02057705)
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 48 people
- Sponsor
- Valerion Therapeutics, LLC
Dermatomyositis, Adult Polymyositis
Environmental Risk Factors for Myositis in Military Personnel
Background: Myositis is a rare disease in which the body s immune cells attack the muscle tissue. It can cause muscle weakness, swelling, and pain. It can develop in people with no history of muscle problems.
- Conditions
- Dermatomyositis, Adult Polymyositis, Inclusion Body Myositis, Myositis
- Trial name
- NCT01734369
- Where
- 7 sites in United States
- Volunteers
- 37 people
- Sponsor
- National Institute of Environmental Health Sciences (NIEHS)
Pseudoxanthoma Elasticum, Angioid Streaks
Progression Assessment of PXE-associated Alterations
This monocentric clinical observational study at the University Eye Hospital Bonn investigates the natural history and structural consequences of a diseased Bruch's membrane (BrM) in Pseudoxanthoma elasticum (PXE).
- Conditions
- Pseudoxanthoma Elasticum, Angioid Streaks, Peau d'Orange, Grönblad-Stranberg Disease (Pseudoxanthoma Elasticum)
- Trial name
- PAPA (NCT07048106)
- Where
- Germany
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- University Hospital, Bonn
HIV
Analysis of HIV-1 Replication During Antiretroviral Therapy
This study will determine if the level of virus in HIV-infected patients taking antiretroviral medications for prolonged periods decreases or persists at a stable level.
- Trial name
- NCT00767312
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Facioscapulohumeral Muscular Dystrophy Type 2
An 18-month Prospective Natural History Study to Gain Insight Into FSHD2 Pathophysiology and Disease Progression
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common inherited myopathies in adults. It is associated with genetic and epigenetic deregulation of the D4Z4 locus on the sub-telomeric region of chromosome 4q35…
- Trial name
- INSIGHT FSHD2 (NCT06079567)
- Testing
- Validation of new COMs for FSHD2 patients
- Where
- 9 sites in Belgium, France and 3 more
- Volunteers
- 50 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor
ATHN 7: Hemophilia Natural History Study
This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment.
- Conditions
- Hemophilia A With Inhibitor, Haemophilia A Without Inhibitor, Hemophilia B With Inhibitor, Haemophilia B Without Inhibitor
- Trial name
- ATHN 7 (NCT03619863)
- Where
- 29 sites in United States
- Volunteers
- 395 people
- Sponsor
- American Thrombosis and Hemostasis Network
Aneurysm, Intracranial, Natural History
The Natural Course of Unruptured Intracranial Aneurysms in a Chinese Cohort
This is a registry study of the natural course of unruptured intracranial aneurysms (UIA). In addition, the investigators will analyze the risk factors for the rupture of the UIA and their joint effect.
- Trial name
- NCT03117803
- Where
- China
- Volunteers
- 5,000 people
- Sponsor
- Tang-Du Hospital
Eye Diseases
Myopic Optic Neuropathy in Chinese High Myopia Population
This study intends to establish a registry cohort to enroll patients with high myopia to study the natural course of myopic optic neuropathy in Chinese adult population.
- Trial name
- NCT04302220
- Where
- China
- Volunteers
- 813 people, healthy volunteers welcome
- Sponsor
- Sun Yat-sen University
Thoracic Organ Transplantation
Genome Transplant Dynamics
Study Description: Heart and lung transplants can save lives, but long-term success is often limited by organ rejection that is hard to detect early.
- Trial name
- NCT02423070
- Where
- 7 sites in United States
- Volunteers
- 991 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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