Clinical trials: Genetic Conditions
Trigeminal Nerve Injuries, Post-herpetic Neuralgia
Transcranial Magnetic Stimulation for Facial Pain
Complex oro-facial pain is a poorly diagnosed and poorly treated cause of significant suffering and disability. This study will explore the ability of fMRI to identify patients with complex oro-facial pain who respond to…
- Conditions
- Trigeminal Nerve Injuries, Post-herpetic Neuralgia, Facial Pain, Nervus Intermedius Neuralgia
- Trial name
- NCT02633306
- Testing
- transcranial magnetic stimulation
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- University of Minnesota
Osteogenesis Imperfecta
Post-market Observational Study on JTIN Telescopic Nail in Osteogenesis Imperfecta Pediatric Patients
This study has been planned as part of the Orthofix Srl post-market active surveillance plan for the collection of data on both the clinical performance and the safety profile of the JTIN.
- Trial name
- JTIN (NCT05612139)
- Testing
- JTIN implantation surgery
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Orthofix s.r.l.
Headache, Migraine
Comparison of a Drug and Placebo in the Prevention of Migraine Headaches
The purpose of this study is to determine whether ethosuximide works better than placebo in the prevention of episodic migraine among veterans.
- Trial name
- NCT01122381
- Testing
- ethosuximide
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- US Department of Veterans Affairs
Cryopyrin-Associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome
Efficacy and Safety of ACZ885 in Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease
This will provided long-term safety and efficacy data for ACZ885 (a fully human anti-interleukin-1β [anti-IL-1β] monoclonal antibody) given as an injection subcutaneously in patients who participated in the CACZ885A2102…
- Conditions
- Cryopyrin-Associated Periodic Syndromes, Familial Cold Autoinflammatory Syndrome, Muckle Wells Syndrome, Neonatal Onset Multisystem Inflammatory Disease
- Trial name
- NCT00685373
- Testing
- Canakinumab (ACZ885)
- Where
- 28 sites in United States, Belgium and 7 more
- Volunteers
- 166 people
- Sponsor
- Novartis
Cystinuria
Lipoic Acid Supplement for Cystine Stone
This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive 1200 mg alpha lipoic acid orally daily for three years, while the other half will receive…
- Trial name
- ALA (NCT02910531)
- Testing
- Alpha lipoic acid
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Thomas Chi, MD
Metastatic Breast Cancer
A Phase III Study of BKM120 With Fulvestrant in Patients With HR+,HER2-, AI Treated, Locally Advanced or Metastatic Breast Cancer Who Progressed on or After mTORi
This study was a multicenter, randomized, double-blind, placebo-controlled Phase III study to determine the efficacy and safety of treatment with Buparlisib plus Fulvestrant vs.
- Trial name
- BELLE-3 (NCT01633060)
- Testing
- Fulvestrant, BKM120
- Where
- 197 sites in United States, Argentina and 20 more
- Volunteers
- 432 people
- Sponsor
- Novartis Pharmaceuticals
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics
Obesity
Communicating Risk Information for Obesity
This study will examine the impact of providing genetic risk information for obesity on people's attitudes and beliefs about obesity, health behaviors and weight outcomes.
- Trial name
- NCT01355224
- Testing
- Genetic, Lifestyle
- Where
- United States
- Volunteers
- 798 people, healthy volunteers welcome
- Sponsor
- Boston University
Cystic Fibrosis, Irritable Bowel Syndrome
Neuromodulation for Children With Cystic Fibrosis Experiencing Chronic Abdominal Pain
Chronic abdominal pain is extremely common in individuals with Cystic Fibrosis (CF). Therapy for chronic abdominal pain is very limited and generally consists of osmotic laxatives or drugs that are used to treat irritable bowel…
- Conditions
- Cystic Fibrosis, Irritable Bowel Syndrome, Chronic Pain
- Trial name
- NCT05515250
- Testing
- Use of neurostimulation device
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Louis University
Cardiomyopathy, Dilated, Arrhythmia
Mental Stress Reduction in Defibrillator Patients
The purpose of this study is to assess the effectiveness of a 10 week program of Stress Management versus control Patient Education sessions on cardiac responses to mental stress in veterans with Implantable…
- Conditions
- Cardiomyopathy, Dilated, Arrhythmia, Anger, Stress
- Trial name
- NCT00624520
- Testing
- Cognitive Behavioral Stress Management (CBSM), Patient Education
- Where
- 2 sites in United States
- Volunteers
- 129 people
- Sponsor
- US Department of Veterans Affairs
Chronic Obstructive Pulmonary Disease, Heart Failure
PATient Navigator to rEduce Readmissions
Staying out of the hospital is valued by patients and their caregivers. Their interests converge with those of hospitals now that high 30-day readmission rates for some conditions place hospitals at risk for financial penalties…
- Conditions
- Chronic Obstructive Pulmonary Disease, Heart Failure, Sickle Cell Disease, Myocardial Infarction
- Trial name
- PArTNER (NCT02114515)
- Testing
- Hospital usual care, Navigator intervention, Peer-led telephone support line
- Where
- United States
- Volunteers
- 1,029 people
- Sponsor
- University of Illinois Chicago
Atopic Dermatitis (AD)
Study of Ruxolitinib Cream in Adolescents With Atopic Dermatitis
The purpose of this study is to evaluate the long-term safety and tolerability of ruxolitinib cream in adolescents with Atopic Dermatitis (AD).
- Trial name
- NCT05456529
- Testing
- Ruxolitinib Cream
- Where
- 38 sites in United States, Canada and 1 more
- Volunteers
- 103 people
- Sponsor
- Incyte Corporation
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Kidney Failure, Chronic, Growth Hormone Deficiency
A Study to Optimize Growth Hormone Dosing in Children With Chronic Kidney Disease by Measuring IGF-1 Levels in Blood
Treatment with growth hormone (GH; a hormone made by the body that stimulates growth) has been shown to be helpful in treating children with chronic kidney disease who fail to grow.
- Trial name
- NCT00212758
- Testing
- Nutropin AQ
- Where
- 8 sites in United States
- Volunteers
- 17 people
- Sponsor
- Oregon Health and Science University
Heart Failure, Coronary Artery Disease
COVID-19 Testing in Underserved and Vulnerable Populations
As part of National Institutes of Health Rapid Acceleration of Diagnostics-Underserved Populations (RADx-UP) program, the goal of the RADxUP study is to develop, test, and evaluate a rapid, scalable capacity building project to…
- Conditions
- Heart Failure, Coronary Artery Disease, Cancer, Chronic Kidney Diseases
- Trial name
- NCT05315908
- Testing
- Community outreach method
- Where
- United States
- Volunteers
- 9,120 people
- Sponsor
- Jesse Nodora
Alpha-1 Antitrypsin Deficiency
A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype
The purpose of this study evaluates the efficacy and safety of VX-864 in participants with the PiZZ genotype over 48 weeks.
- Trial name
- NCT05643495
- Testing
- VX-864
- Where
- 13 sites in United States, Germany and 2 more
- Volunteers
- 14 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Fanconi Anemia
Eltrombopag for Fanconi Anemia
Background: Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly.
- Trial name
- NCT03206086
- Testing
- Eltrombopag
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome
Distribution of Cell-cell Junction Proteins in Arrhythmic Disorders
Every week in the UK, 12 apparently healthy and fit individuals under the age of 35 die suddenly, a tragic event known as sudden cardiac death (SCD).
- Conditions
- Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome, Cardiac Channelopathy
- Trial name
- NCT04257994
- Testing
- obtaining a buccal smear sample
- Where
- United Kingdom
- Volunteers
- 26 people
- Sponsor
- St. George's Hospital, London
Retinitis Pigmentosa, Usher Syndrome Type 2
Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa
This clinical trial is a single-site, 30 patient study for participants who have early stage retinitis pigmentosa, or Usher syndrome (type 2 or 3). Funding Source - FDA OOPD and Foundation Fighting Blindness.
- Conditions
- Retinitis Pigmentosa, Usher Syndrome Type 2, Usher Syndrome Type 3
- Trial name
- NCT01530659
- Testing
- NT-501
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Neurotech Pharmaceuticals
Gout
Efficacy, Safety And Tolerability Of PF-06743649 In Gout Subjects.
The purpose of the study is to assess the effect of PF-06743649 in lowering serum uric acid in subjects suffering from gout following 14 days of dosing, as well as assessing safety and tolerability.
- Trial name
- NCT02187029
- Testing
- PF-06743649
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Pfizer