Clinical trials: Natural History
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.
Depression
Characterization and Treatment of Adolescent Depression
This research study seeks to find causes and treatments of depression in teenagers. The study goals are to increase our knowledge of treatments for depression and understand how the brain changes when teenagers have depression.
- Trial name
- NCT03388606
- Testing
- Growth Mindset
- Where
- 3 sites in United States
- Volunteers
- 4,100 people, healthy volunteers welcome
- Sponsor
- National Institute of Mental Health (NIMH)
Fertility Perservation, Fertility
Fertility Attitudes of Adolescents and Young Adults With Turner Syndrome and Their Parents/Guardians (The Fertility ConcepTS Study)
Background: Turner syndrome (TS) is caused by the partial or complete absence of one of the two X chromosomes in all cells or a portion of cells.
- Trial name
- NCT05078892
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
EBV, HSV
The Natural History of Severe Viral Infections and Characterization of Immune Defects in Patients Without Known Immunocompromise
Background: Infections caused by viruses are common causes of illnesses: the common cold, many ear infections, sore throats, chicken pox, and the flu are caused by different viruses.
- Conditions
- EBV, HSV, VZV, HPV
- Trial name
- NCT01011712
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Open Pilon Fractures
Operatively Treated Open Pilon Fractures
A chart review will be performed to extrapolate information including sex, height, weight, date/time admitted to HMC, injury data, comorbidities, fracture pattern, antibiotic usage, surgical history, wound history, fracture…
- Trial name
- NCT00689429
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Milton S. Hershey Medical Center
Von Hippel-Lindau Disease, Central Nervous System Hemangioblastoma
Pediatric Von Hippel-Lindau Disease: Natural History, Predictive Factors, and Long-Term Functional Outcomes of Central Nervous System Hemangioblastomas
Von Hippel-Lindau (VHL) disease is a rare hereditary cancer predisposition syndrome associated with the development of central nervous system hemangioblastomas from childhood.
- Trial name
- VHL-PED-CHB (NCT07705529)
- Where
- 3 sites in France
- Volunteers
- 25 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Fear, Stress
Epi-Genetic Modulators of Fear Extinction in Alcohol Dependence
Background: Researchers want to learn if people with alcohol dependence have more difficulty learning to feel calm, or learn to fear things more easily.
- Conditions
- Fear, Stress, Alcohol Dependence
- Trial name
- NCT02438969
- Where
- United States
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- National Institute on Alcohol Abuse and Alcoholism (NIAAA)
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)
Malaria
Laboratory Evaluation of Pregnancy Malaria Vaccine Candidates/In-vitro Testing of Pregnancy Malaria Vaccine Candidates
Background: Malaria is a disease that affects many people in African countries. It is caused by germs that are spread by mosquito bites. It can be fatal if not diagnosed and treated right away.
- Trial name
- NCT02471378
- Where
- Mali
- Volunteers
- 7,476 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Abortion, Habitual, Infertility Unexplained
Autologous Decidual-like Natural Killer Cells Therapy for Infertility or Adverse Pregnancy History
The aim of this study is preliminary exploration of the effectiveness and duration of autologous decidual-like NK cells therapy in improving uterine NK cells dysfunction.
- Conditions
- Abortion, Habitual, Infertility Unexplained, Embryo Implantation, Pre-Eclampsia
- Trial name
- NCT06821685
- Testing
- autologous decidual-like NK cells
- Where
- China
- Volunteers
- 14 people
- Sponsor
- The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
Sickle Cell Disease, Solid Tumor
Validating Pain Scales in Children and Young Adults
Background: Assessing pain levels is important to improve treatments for different illnesses. Most pain rating scales are used to determine pain levels in adults. Pain is also a common symptom among children who have cancer.
- Conditions
- Sickle Cell Disease, Solid Tumor, Leukemia, Neurofibromatosis
- Trial name
- NCT01639950
- Where
- United States
- Volunteers
- 252 people
- Sponsor
- National Cancer Institute (NCI)
Mild Traumatic Brain Injury
Deployment Related Mild Traumatic Brain Injury (mTBI)
The study will provide evidence on the long term outcomes of mTBI in service members returning from Afghanistan and Iraq. In addition, the study will provide evidence on mTBI incidence, and symptom patterns.
- Trial name
- NCT01847040
- Where
- 2 sites in United States
- Volunteers
- 750 people, healthy volunteers welcome
- Sponsor
- The Defense and Veterans Brain Injury Center
RAS Mutation, Neurofibromatosis 1
RASopathy Biorepository
The RASopathies are a group of developmental disorders caused by genetic changes in the genes that compose the Ras/mitogen activated protein kinase (MAPK) pathway. New RASopathies are being diagnosed frequently.
- Conditions
- RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines
- Trial name
- NCT04395495
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Lung Diseases, Sleep Apnea Syndromes
Sleep Apnea: Age Effects On Prevalence and Natural History
To identify the prevalence of sleep apnea in men and its relationship to age, assess the natural history of the disorder, and predict those men at risk.
- Trial name
- NCT00005296
- Volunteers
- 741 people
- Sponsor
- Milton S. Hershey Medical Center
Late Infantile Metachromatic Leukodystrophy
Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy
High-field MRI and diffusion tensor imaging with 3D reconstruction of the myelin tracks, in combination with multivoxel proton spectroscopy, will allow to precise more accurately the evolution of the white matter lesions in…
- Trial name
- HCIT-MLD (NCT01325025)
- Testing
- High-field MRI (3 Teslas)
- Where
- 3 sites in France
- Volunteers
- 29 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Healthy Volunteer, Traumatic Brain Injury
Estimating Brain Biomechanics Using MRI
Objective: In this study we will develop and apply imaging techniques to perform the first three-dimensional (3-D) measurements of brain biomechanics during mild head movement in healthy human subjects.
- Conditions
- Healthy Volunteer, Traumatic Brain Injury, Brain Mapping, Craniocerebral Trauma
- Trial name
- NCT01633268
- Where
- United States
- Volunteers
- 101 people, healthy volunteers welcome
- Sponsor
- National Institutes of Health Clinical Center (CC)
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Angelman Syndrome
Angelman Syndrome Natural History Study
The goal of this study is to conduct a prospective, longitudinal natural history study of children and adults with Angelman Syndrome using investigator-observed and parent-reported outcome measures to obtain data that will be…
- Trial name
- NCT04507997
- Where
- 11 sites in United States, Canada
- Volunteers
- 300 people
- Sponsor
- Boston Children's Hospital
Hereditary Hemochromatosis
Cardiac Function in Patients With Hereditary Hemochromatosis
This study will examine the effect of iron buildup in the hearts of patients with hereditary hemochromatosis (HH), a genetic disease that causes the body to accumulate excess amounts of iron.
- Trial name
- NCT00068159
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Isolated Distal DVT, Proximal DVT
Natural History of Isolated Deep Vein Thrombosis of the Calf
Whether isolated distal DVT (IDDVT), DVT confined to the calf, should be looked for and diagnosed to allow them to be treated with anticoagulants remains one of the still unsolved issues in vascular medicine, especially because…
- Conditions
- Isolated Distal DVT, Proximal DVT, Pulmonary Embolism
- Trial name
- CALTHRO (NCT00816920)
- Testing
- Ultrasound examination of leg veins
- Where
- 2 sites in Italy
- Volunteers
- 500 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
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