Clinical trials: Natural History
Limb-Girdle Muscular Dystrophy Type 2A, Limb-Girdle Muscular Dystrophy, Type 2E
Natural History of Limb Girdle Muscular Dystrophy Type 2A and Type 2E
This is an observational study, no drug (marketed or investigational) will be provided as part of the study, and the study procedures will have no impact on the medical care delivered to patients participating in this study.
- Trial name
- NCT03488784
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Lindsay Alfano
Multiple System Atrophy
Observational Study in Multiple System Atrophy
Talisman is global clinical study (20058N) in Multiple System Atrophy (MSA) patients. It will be conducted in two regions (China and the European Union [EU]).
- Trial name
- TALISMAN (NCT05453058)
- Testing
- plasma NfL and brain MRI (vMRI, DTI, and ASL [if feasible]) only for the EU cohort
- Where
- 8 sites in China
- Volunteers
- 90 people
- Sponsor
- H. Lundbeck A/S
Fibrodysplasia Ossificans Progressiva
A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and…
- Trial name
- NCT02322255
- Where
- 7 sites in United States, Argentina and 4 more
- Volunteers
- 114 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Stuttering
Genetic Linkage Studies of Stuttering
Stuttering is an abnormality in speech that affects the rhythm of speech. People who stutter know what they wish to say, but at the time are unable to say it because of involuntary repetition, unnecessary lengthening…
- Trial name
- NCT00001604
- Where
- 3 sites in United States, Japan and 1 more
- Volunteers
- 3,044 people
- Sponsor
- National Institute on Deafness and Other Communication Disorders (NIDCD)
Leukemia, Lymphoma
Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant
Background: After an allogeneic hematopoietic stem cell transplant (HSCT), the donor genome is found in the recipient s circulation and tissues.
- Conditions
- Leukemia, Lymphoma, Hematologic Malignancy
- Trial name
- NCT06856226
- Testing
- Arm 1
- Where
- United States
- Volunteers
- 88 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Osteogenesis Imperfecta
Registry of Osteogenesis Imperfecta
ROI is a retrospective and prospective registry, finalized for care and research purposes. It is articulated in main sections - strongly related and mutually dependent on each other - corresponding to different data domains…
- Trial name
- ROI (NCT04115774)
- Testing
- bisphosphonates
- Where
- Italy
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Luca Sangiorgi
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)
COVID-19 Infection
Send-In Sample Collection for Comprehensive Analyses of Innate and Adaptive Immune Responses During Acute COVID-19 and Convalescence
Background: Severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) causes coronavirus disease 2019 (COVID-19). The global outbreak of COVID-19 is a major public health problem. COVID-19 causes a wide range of symptoms.
- Trial name
- NCT04582903
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Mild-to-moderate COVID-19
A Clinical Study to Assess the Natural History of COVID-19 and Effects of KB109 and Supportive Self-care in Outpatients With Mild-to-moderate COVID-19
This randomized, open-label, prospective, parallel-group controlled clinical study that aims to explore the natural history of COVID-19 illness and the safety of KB109, a novel glycan, plus SSC versus SSC alone and measures of…
- Trial name
- NCT04414124
- Testing
- KB109 + Self Supportive Care (SSC), Self Supportive Care (SSC) Alone
- Where
- 16 sites in United States
- Volunteers
- 350 people
- Sponsor
- Kaleido Biosciences
Non-Medullary Thyroid Cancer
Clinical and Genetic Studies in Familial Non-medullary Thyroid Cancer
Background: Researchers are studying types of thyroid cancer that seem to cluster in families. Non-medullary thyroid cancer accounts for the vast majority of all types of thyroid cancer, but little is known about possible genes…
- Trial name
- NCT01109420
- Where
- United States
- Volunteers
- 500 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Mitochondrial Neurogastrointestinal Encephalomyopathy
Natural History Study of MNGIE
Non-interventional, prospective, multicenter, natural history study of patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)
- Trial name
- NCT04245917
- Testing
- Non-interventional
- Where
- 5 sites in United States, Turkey (Türkiye)
- Volunteers
- 60 people
- Sponsor
- Entrada Therapeutics, Inc.
Acute on Chronic Liver Failure
To Study the Influence of GCSF on Natural History of Acute On Chronic Liver Failure After the Acute Phase
All consecutive ACLF (Acute on Chronic Liver failure) patients presenting to the institute of liver and biliary sciences, irrespective of the etiology , who have survived the acute phase (i.e.
- Trial name
- NCT02788240
- Testing
- Peg GCSF, 20% Albumin, Nutrition
- Where
- India
- Volunteers
- 14 people
- Sponsor
- Institute of Liver and Biliary Sciences, India
Coronary Artery Disease Progression, Radial Wall Strain
Natural History of Coronary Atherosclerosis
The present study sought to explore the predictive value of radial wall strain (RWS, derived solely from angiograms) for coronary artery lesion progression compared with lesion vulnerability assessed by optical coherence…
- Conditions
- Coronary Artery Disease Progression, Radial Wall Strain, Atherosclerotic Lesion, Optical Coherence Tomography
- Trial name
- NASCENT (NCT06040073)
- Where
- China
- Volunteers
- 125 people
- Sponsor
- China National Center for Cardiovascular Diseases
Mitochondrial, Leukoencephalopathies
Natural History and Genotype-Phenotype Correlations in Mitochondrial Leukoencephalopathies
Mitochondrial disorders (MDs) are among the most common inherited metabolic diseases, caused by genetic defects in mitochondrial or nuclear DNA that impair cellular energy production.
- Conditions
- Mitochondrial, Leukoencephalopathies, Leukoencephalopathy
- Trial name
- Leuko Mito (NCT07868796)
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
GNAO1
GNAO1 Natural History Study
The purpose of the GNAO1 Natural History Study is to establish the clinical phenotype of GNAO1 associated neurologic disease, its association with genotype, and areas of clinical importance within the disease.
- Trial name
- GNAO1 (NCT04950946)
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Washington University School of Medicine
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
Pseudoxanthoma Elasticum
Pseudoxanthoma Elasticum (PXE) Natural History Biomarkers in PXE Individuals and Their Biological Non-PXE Siblings
This PXE biomarker study aims to characterize the levels of inorganic pyrophosphate (PPi), pyridoxal 5´-phosphate (PLP), and other biomarkers relevant to PXE and ectopic calcification in both PXE patients and their biological…
- Trial name
- NCT05025722
- Testing
- Non Interventional
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Daiichi Sankyo
Multiple Sclerosis
Pathological Basis of MRI Signal Changes in Multiple Sclerosis
Background: Multiple sclerosis (MS) is a disease that damages the central nervous system (brain and spinal cord). This leads to increased physical disability over time. The disease is lifelong once it begins.
- Trial name
- NCT02659956
- Where
- United States
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Pregnancy Outcome
NIH Intramural Research Program s Pregnancy Registry Protocol for Subjects and Their Partners
Background: In 2018, the NIH intramural research program had almost 23,000 people taking part in active clinical research studies. Half of those people were female. More than a third were of child-bearing age.
- Trial name
- NCT04977466
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Dengue Fever
Investigating Vector-Borne Determinants of Aedes Transmitted Arboviral Infections in Cambodia: An Observational Longitudinal Cohort Study in Children
Background: Some mosquitos carry viruses that can cause disease. Some examples are dengue and Zika. The mosquitos spread disease by biting people and infecting them with the virus.
- Trial name
- NCT03534245
- Where
- Cambodia
- Volunteers
- 775 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
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