Clinical trials: Natural History
Neurofibroma, Plexiform, Neurofibromatosis Type I
R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform Neurofibromas
This study will examine whether the experimental drug R115777 (Tipifarnib) can shrink or slow the growth of plexiform neurofibromas in children and young adults with neurofibromatosis type 1 (NF1) and determine what side effects…
- Trial name
- NCT00021541
- Testing
- tipifarnib
- Where
- 12 sites in United States, Germany
- Volunteers
- 62 people
- Sponsor
- National Cancer Institute (NCI)
Lysosomal Acid Lipase Deficiency, Wolman Disease
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
This is a Natural History study to characterize key aspects of the clinical course of lysosomal acid lipase (LAL) deficiency/Wolman phenotype in patients.
- Trial name
- NCT01358370
- Where
- 14 sites in United States, Canada and 3 more
- Volunteers
- 40 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Progressive Multifocal Leukoencephalopathy
Natural History Study of Progressive Multifocal Leukoencephalopathy (PML)
Background: Progressive multifocal leukoencephalopathy (PML) is a severe viral infection of the brain. It is caused by JC virus.
- Trial name
- NCT01730131
- Where
- United States
- Volunteers
- 700 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Eye Diseases, Healthy Volunteers
Adaptive Optics Retinal Imaging
Background: By the time diseases of the retina are detected, serious damage has often already been done. Researchers want to find better ways of viewing the retina. One way called adaptive optics may help detect problems earlier.
- Trial name
- NCT02317328
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Eye Institute (NEI)
Acute Coronary Syndrome (ACS)
PROSPECT II & PROSPECT ABSORB - an Integrated Natural History Study and Randomized Trial.
The present study has two components, an overall prospective observational study using multimodality imaging (PROSPECT II) that will examine the natural history of patients with unstable atherosclerotic coronary artery disease…
- Trial name
- P2 (NCT02171065)
- Testing
- ABSORB BVS
- Where
- Sweden
- Volunteers
- 902 people
- Sponsor
- Uppsala University
Hepatitis B
Effectiveness of the Hepatitis B Vaccine Post-Hematopoietic Stem Cell Transplant
Background: Stem cell transplants (SCTs) are important in treating many diseases. There are two main types of transplants. Autologous stem cells come from the person getting the cells.
- Trial name
- NCT03511794
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Sanfilippo Syndrome A
Natural History Observational Study of MPS IIIa in SMC
To characterize the clinical course of mucopolysaccharidosis type IIIA (MPS IIIA), and identify potential endpoints for future treatment trials.
- Trial name
- NCT04918641
- Testing
- assessment
- Volunteers
- 25 people
- Sponsor
- Samsung Medical Center
Hypertrophic Cardiomyopathy
Natural History and Results of Dual Chamber (DDD) Pacemaker Therapy of Children With Obstructive Hypertrophic Cardiomyop...
Several studies have shown that specialized pacemaking devices (DDD pacing) can improve the symptoms associated with hypertrophic cardiomyopathy (HCM) in adults.
- Trial name
- NCT00001396
- Testing
- Paragon Pacemaker
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
B Cell, Acute Lymphoblastic Leukemia
Pilot Prospective Study for PET-CT Imaging in Participants With Relapsed/Refractory Acute Leukemias
Background Acute lymphoblastic leukemia (ALL) accounts for about 25 percent of childhood cancers and for about 20 percent of adult leukemias.
- Conditions
- B Cell, Acute Lymphoblastic Leukemia, Relapsed/Refractory
- Trial name
- NCT05969002
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Spinal Muscular Atrophy
Natural History of Types 2 and 3 SMA in Taiwan
The primary objective of this study is to investigate the natural history of spinal muscular atrophy (SMA) types 2 and 3 patients in Taiwan. This study will provide further insights into the clinical course SMA.
- Trial name
- NCT03300869
- Where
- Taiwan
- Volunteers
- 300 people
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant
Natural History of Autosomal Dominant Optic Atrophy (ADOA), Caused by OPA1 Mutation
The purpose of this study is to characterize the disease progression of confirmed OPA1 mutation-associated autosomal dominant optic atrophy (ADOA) by evaluating the changes in ocular structural and functional outcomes.
- Conditions
- Autosomal Dominant Optic Atrophy, Optic Atrophy, Autosomal Dominant, Optic Atrophies, Hereditary, Kjer Optic Atrophy
- Trial name
- NCT06140329
- Where
- 7 sites in United States, Australia and 4 more
- Volunteers
- 1 people
- Sponsor
- PYC Therapeutics
PANS, PANDAS
PEDI Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcus Study
The objective of the Pediatric Epidemiological Data and Incidence (PEDI) PANDAS study is to demonstrate the feasibility of enrollment and retention of subjects in a study to determine the incidence and natural history of children…
- Trial name
- PANDAS (NCT06811935)
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- University of Wisconsin, Madison
LAMA2-related Muscular Dystrophy, SELENON-related Myopathy
A 5-year Natural History Study in LAMA2-related Muscular Dystrophy and SELENON-related Myopathy.
SELENON-related myopathy (SELENON-RM) and LAMA2-related muscular dystrophy (LAMA2-MD) are congenital neuromuscular disorders presenting with slowly, progressive axial muscle weakness, spinal rigidity, scoliosis and respiratory…
- Trial name
- NCT06132750
- Testing
- No intervention
- Where
- Netherlands
- Volunteers
- 40 people
- Sponsor
- Radboud University Medical Center
Chronic Graft Versus Host Disease
Improving Outcomes Assessment in Chronic GVHD
The purpose of this study is to see if recent guidelines proposed by the National Institutes of Health for the diagnosis, staging, and response assessment of people with chronic GVHD can improve our understanding of this…
- Trial name
- NCT00637689
- Where
- 9 sites in United States
- Volunteers
- 601 people
- Sponsor
- Fred Hutchinson Cancer Center
Hereditary Angioedema Type I and II
Global Registry to Gather Data on Natural History of Patients With Hereditary Angioedema Type I and II
The objective of this international hereditary angioedema (HAE) register is to collect homogeneous clinical and laboratory data on patients with HAE type I and II gathering better information on the natural course of the disease…
- Trial name
- HGR (NCT03828279)
- Testing
- functional and antigenic C1 inhibitor
- Where
- Italy
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- HAE Global Registry Foundation
Retinitis Pigmentosa
Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures
Background: Retinitis pigmentosa (RP) is a group of blinding eye diseases. It is caused mostly by mutations in photoreceptor-expressed genes. RP affects about 2 million people globally.
- Trial name
- NCT03845218
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- National Eye Institute (NEI)
Severe Combined Immunodeficiency (SCID), Leaky SCID
Natural History Study of SCID Disorders
This study is a prospective evaluation of children with Severe Combined Immune Deficiency (SCID) who are treated under a variety of protocols used by participating institutions.
- Conditions
- Severe Combined Immunodeficiency (SCID), Leaky SCID, Omenn Syndrome, Reticular Dysgenesis
- Trial name
- NCT01186913
- Where
- 44 sites in United States, Canada
- Volunteers
- 690 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Diffuse Intrinsic Pontine Glioma, PNET
Exploratory Study of Effects of Radiation Therapy in Pediatric Patients With Central Nervous System Tumors
This study will analyze the effects of radiation given to children who have tumors of the central nervous system (CNS). Researchers want to learn more about changes in the quality of life that patients may experience as a result…
- Conditions
- Diffuse Intrinsic Pontine Glioma, PNET, Ependymoma, Germinoma
- Trial name
- NCT01445288
- Where
- United States
- Volunteers
- 76 people
- Sponsor
- National Cancer Institute (NCI)
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases
Natural History Study of SLC25A46 Mutation-related Mitochondriopathy
The purpose of the study is to systematically characterize the clinical course of the progressive neuropathy and optic atrophy observe in pediatric and adult patients with biallelic mutations in the solute carrier family 25…
- Conditions
- Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
- Trial name
- NCT04594590
- Testing
- Mutation analysis
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- State University of New York at Buffalo
Amyloidosis Cardiac
Multimodality Cardiac Imaging for Disease Progression in ATTR-CM
The goal of this clinical trial is to investigate whether new imaging techniques can help us to better understand the cardiac amyloidosis.
- Trial name
- FAPI-ATTR (NCT07112066)
- Testing
- FAPI tracer
- Where
- Switzerland
- Volunteers
- 50 people
- Sponsor
- Dominik Benz