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)
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
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Vaping Teens, Healthy Volunteers
Hippocampal Network Changes Following Mindfulness Training in Tobacco Vaping Adolescents in an Open-label, Pilot Study
The purpose of this translational bench-to-bedside study is to examine the neurobiological effects of an evidence-based technology-delivered mindfulness training (MT) program on vaping-related rsFC alterations in hippocampal…
- Trial name
- NCT06503159
- Testing
- Mindfulness-based Stress Reduction (MBSR) Program, functional magnetic resonance imaging (fMRI)
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- National Institute on Drug Abuse (NIDA)
Duchenne Muscular Dystrophy
A Natural History Study In Chinese Male Patients With Duchenne Muscular Dystrophy
This is a multicenter, prospective, single cohort study designed to describe the natural history of DMD in Chinese male patients.
- Trial name
- NCT03760029
- Testing
- Visit frequency
- Where
- 8 sites in China
- Volunteers
- 312 people
- Sponsor
- Pfizer
Erythropoietic Protoporphyria, EPP
Erythropoietic Protoporphyrias: Studies of the Natural History, Genotype-Phenotype Correlations, and Psychosocial Impact
The initial objective of this protocol is to assemble a well-documented group of patients with confirmed diagnoses of the erythropoietic protoporphyrias, including autosomal recessive Erythropoietic Protoporphyria (EPP) and…
- Conditions
- Erythropoietic Protoporphyria, EPP, X-Linked Protoporphyria, XLP
- Trial name
- NCT01688895
- Where
- 6 sites in United States
- Volunteers
- 150 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Exotropia
Observation Versus Occlusion Therapy for Intermittent Exotropia
The present study is being conducted to assess the natural history of intermittent exotropia and to establish the effectiveness of occlusion in its treatment.
- Trial name
- IXT2 (NCT01032330)
- Testing
- occlusion treatment
- Where
- 2 sites in United States
- Volunteers
- 611 people
- Sponsor
- Jaeb Center for Health Research
Abscesses, Furunculosis
The Natural History of Community-Associated MRSA Infections and Decolonization Strategies
The purpose of this study is to determine the natural history of community-associated Staphylococcus aureus infections in both adult and pediatric patients by monitoring the rate of recurrent infections in those colonized with S.
- Conditions
- Abscesses, Furunculosis, Staphylococcus Aureus, Staphylococcal Skin Infections
- Trial name
- StLStaRS (NCT00513799)
- Testing
- Mupirocin ointment, Chlorhexidine showers, Bleach baths (dilute)
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- Washington University School of Medicine
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
Immunodeficiencies, Immune Dysregulations
COVID-19 Vaccination and Outcomes in Individuals With and Without Immune Deficiencies and Dysregulations
Background: The immune system defends the body against disease and infection. Immune deficiencies are health conditions that decrease the strength of this response.
- Trial name
- PERSIST (NCT04852276)
- Where
- United States
- Volunteers
- 308 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Urticaria
Natural History of Chronic Urticaria
The purpose of this study is to examine the natural history of chronic urticaria in children and to identify the predictors for chronic urticaria remission.
- Trial name
- NHCU (NCT01284426)
- Where
- Thailand
- Volunteers
- 94 people
- Sponsor
- Mahidol University
Upper Respiratory Infection, Common Cold
The Natural History of Viral Upper Respiratory Infections in Children Aged 6 to Less Than 14 Years
This study examines the cold processes of children aged 6 to less than 14. Children will be seen by the study staff 6 days in a row during the course of their naturally-acquired colds.
- Trial name
- NCT00405509
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- West Penn Allegheny Health System
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Achondroplasia
Observational Study Investigating Clinical & Anthropometric Characteristics of Children With Achondroplasia.
This is a registry study in children with achondroplasia, age 0-10 years, to be conducted at multiple clinical centers in several countries.
- Trial name
- NCT03794609
- Where
- 57 sites in United States, Australia and 12 more
- Volunteers
- 315 people
- Sponsor
- Pfizer
Leber's Hereditary Optic Neuropathy (LHON)
Study to Assess the Efficacy and Safety of Raxone in LHON Patients
LEROS is an open-label interventional Phase IV study, designed to further assess the efficacy and safety of Raxone® in the long-term treatment of LHON patients.
- Trial name
- LEROS (NCT02774005)
- Testing
- Idebenone
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 199 people
- Sponsor
- Santhera Pharmaceuticals
Von Hippel-Lindau Disease
Natural History and Management of Pancreatic Lesions in Von Hippel-Lindau Disease
Von Hippel-Lindau disease (VHL) is an inherited cancer syndrome. Patients are at risk for developing pancreatic cysts and tumors. These tumors are more aggressive in some people than in others.
- Trial name
- NCT00062166
- Where
- United States
- Volunteers
- 340 people
- Sponsor
- National Cancer Institute (NCI)
Respiration Disorders, Body Composition, Beneficial
The Austrian LEAD (Lung hEart sociAl boDy) Study
The LEAD study is the first longitudinal cohort investigating respiratory, cardiac, and metabolic health in a reference population of Austria.
- Conditions
- Respiration Disorders, Body Composition, Beneficial, Metabolic Health
- Trial name
- LEAD (NCT01727518)
- Testing
- As the LEAD study is an observational cohort study, there is no intervention to report.
- Where
- Austria
- Volunteers
- 14,966 people, healthy volunteers welcome
- Sponsor
- Ludwig Boltzmann Institute for Lung Health
Lymphomatoid Granulomatosis, Granulomatosis, Lymphomatoid
Treatment and Natural History Study of Lymphomatoid Granulomatosis
This study will evaluate the response and long-term effects of alpha-interferon in patients with lymphomatoid granulomatosis (LYG).
- Conditions
- Lymphomatoid Granulomatosis, Granulomatosis, Lymphomatoid, Non-Hodgkins Lymphoma, Lymphoproliferative Disorder
- Trial name
- NCT00001379
- Testing
- Interferon, Rituxan and EPOCH, Tumor biopsy
- Where
- United States
- Volunteers
- 94 people
- Sponsor
- National Cancer Institute (NCI)
Hypophosphatasia (HPP)
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.
- Trial name
- NCT02104219
- Where
- 19 sites in United States, Australia and 6 more
- Volunteers
- 32 people
- Sponsor
- Alexion Pharmaceuticals, Inc.