Clinical trials: Abnormalities
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Cystic Fibrosis (CF)
Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00677365
- Testing
- MP-376
- Where
- 50 sites in United States, Germany and 1 more
- Volunteers
- 151 people
- Sponsor
- Amgen
Pain, Post-operative
Evaluation of N1539 Following Bunionectomy Surgery
The primary objective of this study is to evaluate the analgesic efficacy of N1539 in subjects with acute moderate to severe pain following unilateral bunionectomy.
- Trial name
- NCT02675907
- Testing
- N1539
- Where
- 4 sites in United States
- Volunteers
- 201 people
- Sponsor
- Baudax Bio
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Huntington's Disease
Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease
The pace of basic science research defining the mechanisms of selective neuronal degeneration in Huntington disease (HD) has far exceeded the pace of translation of this information into clinically effective treatments for the…
- Trial name
- HD (NCT00670709)
- Where
- United States
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- University of California, Los Angeles
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Open-label MNS for Tourette Syndrome
A recent report (Morera Maiquez et al 2020) described reduced tic severity in people with Tourette syndrome during 1-minute epochs of median nerve stimulation (MNS) at 10 Hz.
- Trial name
- NCT05016765
- Testing
- Active, self-directed electrical stimulation of the median nerve
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Washington University School of Medicine
Atopic Dermatitis
Cetaphil Restoraderm Effect on Young Children With Atopic Dermatitis
The purpose of this study is to evaluate the effect of Cetaphil® Restoraderm® skin restoring moisturizer in reducing the signs and symptoms of very dry atopic skin in young children.
- Trial name
- NCT02589392
- Testing
- Cetaphil® Restoraderm® moisturizer + Cetaphil® Restoraderm® body wash, Cetaphil® Restoraderm® body wash
- Where
- 8 sites in China, Philippines
- Volunteers
- 120 people
- Sponsor
- Galderma R&D
Heart Defects, Congenital
Safety and Performance of a Vascular Patch in Pediatric Patients Undergoing Bidirectional Cava-pulmonary Anastomosis
Xeltis developed biodegradable patch prosthesis, the Vascular Patch Model COR-VP-001, to be used as a vascular patch to augment the pulmonary trunk and pulmonary arteries.
- Trial name
- XEL-CR-06 (NCT02377700)
- Testing
- Xeltis Vascular Patch, Model COR-VP-001
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Xeltis
Mixed Hyperlipidemia
Lipid Efficacy and Safety in Participants With Mixed Hyperlipidemia (MK-0524B-024)
This is a 12-week clinical trial in participants with mixed hyperlipidemia to study the effects of MK-0524B on lipids.The primary hypothesis is that MK-0524B (dosed as MK-0524A coadministered with simvastatin) will be superior to…
- Trial name
- NCT00289900
- Testing
- MK-0524A, Atorvastatin, Simvastatin
- Volunteers
- 2,340 people
- Sponsor
- Merck Sharp & Dohme LLC
Skin Laxity
Evaluation of the Evoke Radiofrequency Device for Improvement of Skin Appearance
Evaluation of the Evoke Radiofrequency Device for Improvement of Skin Appearance
- Trial name
- NCT04721600
- Testing
- Evoke Radiofrequency Device
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- InMode MD Ltd.
Autism Spectrum Disorder, EEG With Periodic Abnormalities
Safety and Efficacy of tPBM for Epileptiform Activity in Autism
For this study, the proposed intervention will be noninvasively delivered near infra-red (NIR) light - transcranial Photobiomodulation (tPBM) - to the brains of autistic children with abnormal EEGs with epileptiform discharges or…
- Conditions
- Autism Spectrum Disorder, EEG With Periodic Abnormalities, Epilepsy, Neurodevelopmental Disorders
- Trial name
- tPBM (NCT06352372)
- Testing
- noninvasively delivered near infra-red (NIR) light - transcranial Photobiomodulation (tPBM)
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Richard Frye
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Atopic Dermatitis
Efficacy and Safety of IDP-124 Lotion for the Treatment of Moderate to Severe Atopic Dermatitis in Pediatric and Adult Subjects (301)
Efficacy and Safety of IDP-124 Lotion for the Treatment of Moderate to Severe Atopic Dermatitis in Pediatric and Adult Subjects
- Trial name
- NCT03002571
- Testing
- IDP-124 Lotion, IDP-124 Vehicle Lotion
- Where
- United States
- Volunteers
- 338 people, healthy volunteers welcome
- Sponsor
- Bausch Health Americas, Inc.
Prostate Cancer, Prostatic Neoplasms
Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration
This trial is designed to investigate the safety, dosing schedule, and efficacy of the combination treatment of Panobinostat (a histone deacetylase inhibitor) and hormone therapy for recurrent prostate cancer.
- Trial name
- NCT00878436
- Testing
- Panobinostat, Bicalutamide
- Where
- 4 sites in United States
- Volunteers
- 52 people
- Sponsor
- NYU Langone Health
Tourette Syndrome, Chronic Tic Disorder
Proof of Concept Study of an Oral Orthotic to Reduce Tic Severity in Chronic Tic Disorder and Tourette Syndrome
The purpose of this study is to assess the feasibility of a trial on an oral orthotic for reducing tic severity in children ages 7-25 years with Tourette syndrome (TS) or Chronic Tic Disorder (CTD).
- Trial name
- NCT02067819
- Testing
- Active Oral Orthotic Treatment
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Weill Medical College of Cornell University
Atopic Dermatitis
Targeted Investigation of Microbiome Elimination
The purpose of this study is to examine the pharmacokinetics or survival of new product containing commensal infection fighting bacteria, on the skin of patients with Atopic Dermatitis (AD), after a single application.
- Trial name
- TIME-1 (NCT05177328)
- Testing
- S. hominis A 9 Product
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Down Syndrome, Trisomy 21
Down Syndrome Metabolic Health Study
The purpose of this research study is to determine which measures best capture cardiovascular disease (CVD) risk and type 2 diabetes (T2DM) risk in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01821300
- Where
- 2 sites in United States
- Volunteers
- 257 people, healthy volunteers welcome
- Sponsor
- Children's Hospital of Philadelphia
Prehypertension, Gout
Study of Febuxostat Effect on Blood Pressure in Patients With High Normal Blood Pressure
High normal blood pressure, also called prehypertension, is a significant problem. High uric acid in the blood, the same thing that causes gout, may cause higher blood pressures.
- Conditions
- Prehypertension, Gout, Pulse Wave Velocity, Hypertension
- Trial name
- NCT01472692
- Testing
- Febuxostat
- Where
- United States
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- University of California, San Diego
Duchenne Muscular Dystrophy
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)
The objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or…
- Trial name
- NCT01247207
- Testing
- Ataluren
- Where
- 34 sites in United States, Canada
- Volunteers
- 265 people
- Sponsor
- PTC Therapeutics