Clinical trials: Natural History
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor
Natural History Study of Patients With Neurofibromatosis Type I
Background: Neurofibromatosis Type 1 (NF1) is a genetic disorder in which patients are at increased risk of developing tumors (usually non-cancerous) of the central and peripheral nervous system.
- Conditions
- Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma
- Trial name
- NCT00924196
- Where
- United States
- Volunteers
- 259 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)
Background: NF1 is a genetic syndrome. Tumors appear early in life. Many people with NF1 develop PN. These tumors can become an aggressive cancer called MPNST.
- Trial name
- NCT04774289
- Where
- United States
- Volunteers
- 296 people
- Sponsor
- National Cancer Institute (NCI)
Undiagnosed Disease
The Role of Uncertainty in Coping: The Experience of Parents of Children With Undiagnosed Medical Conditions
Background: Parents of a child with an undiagnosed medical condition face a lot of uncertainty. They may not know how to take care of their child or how the illness will affect their family life.
- Trial name
- NCT01905865
- Where
- United States
- Volunteers
- 168 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
SCN1A, Dravet Syndrome
SCN1A Horizons A Natural History Study of SCN1A-related Epilepsies in the United Kingdom
The aims of this prospective natural history study are to define the seizure, neuro-developmental, and behavioural characteristics of SCN1A-related epilepsies/Dravet syndrome in children and adults longitudinally over a period of…
- Conditions
- SCN1A, Dravet Syndrome, Epilepsy
- Trial name
- NCT06504511
- Where
- United Kingdom
- Volunteers
- 400 people
- Sponsor
- NHS Greater Glasgow and Clyde
Danon Disease
Danon Disease Natural History Study
The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).
- Trial name
- NCT06214507
- Testing
- No intervention
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 60 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Crohn Disease
Genomic/Epigenomic Biomarkers of Deregulation of Immune System in Inflammatory Bowel Diseases
The purpose is to search for enterocyte-specific or non-specific molecular signature of post-operating recurrence of Crohn's disease in transcriptome (measurement of gene expression through number of mRNA copies transcribed for…
- Trial name
- PREDIPOSTOP (NCT02878395)
- Testing
- Ileocolic resection
- Volunteers
- 30 people
- Sponsor
- Central Hospital, Nancy, France
Sickle Cell Trait, Sickle Cell Disease
Blood Sampling for Research Related to Sickle Cell Disease
This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease.
- Conditions
- Sickle Cell Trait, Sickle Cell Disease, Sickle Cell Anemia
- Trial name
- NCT00542230
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Lung Disease, Pneumothorax
Study of the Disease Process of Lymphangioleiomyomatosis
Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor.
- Conditions
- Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
- Trial name
- NCT00001465
- Testing
- Toshibia Aquilion One CT
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Wilson Disease
Natural History of Wilson Disease
The purpose of the registry/repository is to provide a mechanism to store data and specimens to support the conduct of future research about Wilson disease (WD).
- Trial name
- NCT03334292
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 300 people
- Sponsor
- Yale University
Breast Neoplasms
BRCA Mutations in Latinas
Background: BRCA1 and BRCA2 gene mutations have been linked to a higher risk of developing breast cancer and other cancers, and may be associated with types of breast cancer that are more difficult to treat and more likely to…
- Trial name
- NCT01251900
- Where
- United States
- Volunteers
- 266 people
- Sponsor
- National Cancer Institute (NCI)
Eosinophilia, Hypereosinophilic Syndrome
A Longitudinal Study of Familial Hypereosinophilia (FE): Natural History and Markers of Disease Progression
Eosinophils are a type of white blood cell. Elevated eosinophil levels can damage the heart, nerves, and other organs, in the syndrome known as hypereosinophilic syndrome (HES).
- Trial name
- NCT00091871
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Methylmalonic Acidemia, Propionic Acidemia
"The MaP Study": Mapping the Patient Journey in MMA and PA
Longitudinal, exploratory, natural history study of patients with MMA due to mut deficiency and PA to characterize the changes in blood disease biomarkers over time and the frequency and severity of clinical events related to…
- Trial name
- NCT03484767
- Where
- 17 sites in United States, France and 2 more
- Volunteers
- 97 people
- Sponsor
- ModernaTX, Inc.
TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy
WiTNNess - TNNT1 Myopathy Natural History Study
WiTNNess is designed to accurately document the natural course and variation of muscle disease caused by pathogenic changes of the TNNT1 gene.
- Conditions
- TNNT1-associated Myopathy, Infantile-onset Nemaline Rod Myopathy, Myopathies, Nemaline, Myopathy
- Trial name
- WiTNNess (NCT06374719)
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Clinic for Special Children
Mitochondrial Disease
Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
Background: Mitochondrial disease is a rare disorder. It can cause poor growth, developmental delays, muscle weakness, and other symptoms. The disease is usually inherited. It can be present at birth or develop later in life.
- Trial name
- NCT04419870
- Where
- United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
Recurrent Respiratory Papillomatosis, Pulmonary Neoplasm
The Natural History and Biological Study of Pulmonary Recurrent Respiratory Papillomatosis (pRRP)
Recurrent respiratory papillomatosis (RRP) is an orphan disease that affects approximately 20,000 people in the United States and is caused by infection with human papillomavirus (HPV) types 6 and 11.
- Trial name
- NCT06412172
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Massachusetts General Hospital
Viral Infections
Retrospective Study of Viral Reactivation Across All Bone Marrow Transplant Protocols Since 2010
Background: Some blood and immune disorders can be helped with HSCT. This is allogeneic hematopoietic stem cell transplantation. The person who gets the stem cells has their immune system suppressed.
- Trial name
- NCT03111745
- Where
- United States
- Volunteers
- 730 people
- Sponsor
- National Cancer Institute (NCI)
Multicentric Castleman s Disease
Natural History of KSHV-Associated Multicentric Castleman s Disease
Background: Kaposi s sarcoma herpes virus (KSHV) causes several kinds of cancer, Kaposi sarcoma (KS), a form of Multicentric Castleman s Disease (MCD) and a type of lymphoma known as Primary Effusion Lymphoma (PEL).
- Trial name
- NCT04968288
- Where
- United States
- Volunteers
- 195 people
- Sponsor
- National Cancer Institute (NCI)
Parkinson's Disease
Natural History of Levodopa-Induced Dyskinesia (LID)
Levodopa is the main drug treatment for Parkinson's disease. Levodopa can cause unwanted and uncontrolled movements called dyskinesias (LID). The severity of these movements can range from subtle to extremely debilitating.
- Trial name
- NCT01003002
- Testing
- Levodopa (delivered intravenously)
- Where
- United States
- Sponsor
- Oregon Health and Science University
Vascular Dysfunction, Genetic Mutations
Vascular Disease Discovery Protocol
Background: Some genetic diseases put increase the risk of heart and blood diseases, which are the number one cause of death and disability in the U.S. Researchers want to study diseases of the heart and/or blood vessels.
- Conditions
- Vascular Dysfunction, Genetic Mutations, Genetic Predisposition
- Trial name
- NCT03538639
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Idiopathic Anaphylaxis, Drug Anaphylaxis
Cause of Unexplained Anaphylaxis
This study will explore the possible cause of unexplained, or idiopathic, anaphylaxis. Anaphylaxis is a rapid, life-threatening, severe reaction that occurs suddenly after contact with an allergy-causing substance, usually a…
- Conditions
- Idiopathic Anaphylaxis, Drug Anaphylaxis, Venom Anaphylaxis, Food Anaphylaxis
- Trial name
- NCT00719719
- Where
- United States
- Volunteers
- 115 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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