Clinical trials: Genetics
Facial Angiofibroma
Topical Rapamycin and Calcitriol for Angiofibroma of Tuberous Sclerosis
The aim of the study is to determine the effect and safety of topical rapamycin or calcitriol and their combination for the treatment of TSC-associated facial angiofibroma.
- Trial name
- NCT03140449
- Testing
- Rapamycin, Calcitriol, Rapamycin-calcitriol combination
- Volunteers
- 52 people
- Sponsor
- National Taiwan University Hospital
Analgesia
A Study Investigating the Pharmacokinetic Profiles of Four Extended Release Paracetamol Formulations
A study investigating the pharmacokinetic profiles of four extended release paracetamol formulations
- Trial name
- NCT01568749
- Testing
- Paracetamol, Paracetamol formulation 1, Paracetamol Formulation 2
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
X-Linked Adrenoleukodystrophy, Adrenomyeloneuropathy
Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
The purpose of this study is to assess the safety, tolerance, pharmacokinetics, and pharmacodynamics of sobetirome, a selective thyroid hormone analog, in adult male X-ALD patients.
- Trial name
- NCT01787578
- Testing
- Sobetirome
- Where
- United States
- Sponsor
- Thomas S. Scanlan
Atopic Dermatitis
Safety, Tolerability, Pharmacokinetics and Efficacy of Twice Daily Application of Topical BioLexa in Adult Healthy Subjects and Patients With Mild to Moderate Atopic Dermatitis
This is a Phase 1, randomised, double-blind, vehicle controlled study to determine the safety, tolerability, PK and efficacy of twice daily application of topical BioLexa™ lotion, administered for 28 days in adult healthy…
- Trial name
- NCT04544943
- Testing
- BioLexa- Cohort 1, BioLexa- Cohort 2, Gentamicin
- Where
- Australia
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Hoth Therapeutics, Inc.
Heart Failure, Dilated Cardiomyopathy
Myocardial Telomere Recapping Study for Dilated Cardiomyopathy
The aim of this study is to determine the safety and feasibility of giving an adeno-associated viral vector expressing a modified telomerase protein (TERT), driven by cardiac troponin T promoter (AAV9-cTnT-modTERT), to 12 dilated…
- Trial name
- MERCURY-DCM (NCT05837143)
- Testing
- JV001
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai East Hospital
Pregnancy Related, Atopic Dermatitis
An Observational Retrospective Cohort Study Being Conducted in Women With Atopic Dermatitis Exposed to Ruxolitinib Cream Versus Topical Corticosteroids During Pregnancy: A US Claims Database Analysis
This study is to assess infant outcomes of women with AD who were exposed to ruxolitinib cream during pregnancy compared with a control cohort of women with AD who were exposed to a topical corticosteroid (TCS) during pregnancy.
- Trial name
- NCT06627335
- Testing
- Ruxolitinib Cream, TCS
- Where
- United States
- Volunteers
- 5,621 people
- Sponsor
- Incyte Corporation
Idiopathic Cardiomyopathy, Left Ventricular (LV) Systolic Dysfunction
Performance of the Cardiac Microcurrent (C-MIC) System With a Less Invasively Placed Left Ventricular Lead
Patients with idiopathic dilated cardiomyopathy in heart failure (NYHA class III - IV) with a baseline left ventricular ejection fraction between ≥25% and ≤35%, and patients with non-ischemic cardiomyopathy in heart failure (NYHA…
- Conditions
- Idiopathic Cardiomyopathy, Left Ventricular (LV) Systolic Dysfunction, Non-ischemic Cardiomyopathy
- Trial name
- NCT06920030
- Testing
- Subcutaneous cardiac microcurrent treatment
- Where
- 4 sites in Bosnia and Herzegovina, Serbia
- Volunteers
- 22 people
- Sponsor
- Berlin Heals GmbH
Atopic Dermatitis
Open-label Study to Investigate Systemic Exposure in Adult and Pediatric Atopic Dermatitis Patients Treated 8.5 Days With Pimecrolimus Cream 1% Under Occlusion
This study will access the degree to which pimecrolimus is absorbed from pimecrolimus cream 1% into the blood when applied repeatedly under occlusion (i.e.
- Trial name
- NCT00925730
- Testing
- Pimecrolimus
- Where
- 2 sites in Germany
- Volunteers
- 20 people
- Sponsor
- Novartis
Cystic Fibrosis, Non-Cystic Fibrosis Bronchiectasis
A Clinical Study to Investigate Safety, Tolerability and Distribution of CHF 6333 After One or After Repeated Inhalation in Patients With Cystic Fibrosis (CF) and in Patients With Non Cystic Fibrosis (NCFB) Bronchiectasis
CHF 6333 is a medicinal product on development for the treatment of cystic fibrosis and non-CF bronchiectasis and undergoing clinical testing. It has not yet been approved by the authorities for the treatment of these diseases.
- Trial name
- NCT04010799
- Testing
- CHF 6333
- Where
- Germany
- Volunteers
- 68 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Sickle Cell Disease
Uganda Sickle Surveillance Study (US-3)
It is estimated that over 250,000 babies are born with sickle cell disease (SCD) annually in sub-Saharan Africa, and only 10% - 50% of them survive beyond five years of age.
- Trial name
- NCT06301893
- Where
- 4 sites in Uganda
- Volunteers
- 1,000,000 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cardiomyopathy, Obstructive Hypertrophic Cardiomyopathy
Patients' Long-Term Survival of Obstructive Hypertrophic Cardiomyopathy (HCM)
Background: HCM is a genetic heart disease. It can cause fatigue, chest pain, or even death. For more than 50 years, a surgery called septal myectomy has been used to help people with this disease. Dr. Andrew G.
- Trial name
- NCT04603521
- Where
- United States
- Volunteers
- 325 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Cystic Fibrosis Related Diabetes
Insulin Glargine Vs Standard Insulin Therapy
This Study is designed to determine whether treatment of CFRD with glargine insulin will improve hemoglobin A1c, weight and muscle mass compared to the traditional regimen of bedtime NPH insulin.
- Trial name
- NCT00222521
- Testing
- Glargine insulin
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Minnesota
Lynch Syndrome
Lynch Syndrome Integrative Epidemiology and Genetics
The vision of the "Lynch syndrome INtegrative Epidemiology And GEnetics" (LINEAGE) Consortium is to collaboratively improve the lives and longevity of individuals and families with Lynch syndrome.
- Trial name
- LINEAGE (NCT06582914)
- Where
- 2 sites in United States
- Volunteers
- 5,000 people
- Sponsor
- University of Colorado, Denver
Phenylketonurias
Study to Evaluate the Effect on Nitrogen Retention of Two Different Posology Schemes of PKU GOLIKE PLUS 3-16 and Free AAs in Patients With Phenylketonuria
This is an open-label, randomized, 4-way crossover, monocentric, controlled study in patients (3 ≤ years of age ≤ 20 )) with phenylketonuria (PKU).
- Trial name
- NCT05229549
- Testing
- GOLIKE PLUS 3-16, Free AAs
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- APR Applied Pharma Research s.a.
Thyroid Associated Ophthalmopathies
Treatment of Soft Tissue Expansion in Inactive Thyroid Eye Disease Patients Using Drops of Prostaglandin Analogues
Prostaglandin analogues eye drops are common and effective treatment for decreasing Intra-Ocular Pressure (IOP) in Glaucoma patients.
- Trial name
- NCT02155049
- Testing
- bimatoprost 0.03%
- Where
- Israel
- Volunteers
- 10 people
- Sponsor
- Rabin Medical Center
Cystic Fibrosis
A Study to Evaluate Multiple Doses of GLPG2222 in Adult Subjects With Cystic Fibrosis
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate 4 different doses of GLPG2222 administered for 4 weeks to adult subjects with a confirmed diagnosis of CF and…
- Trial name
- NCT03119649
- Testing
- GLPG2222 50 mg, GLPG2222 100 mg, GLPG2222 200 mg
- Where
- 23 sites in United States, Belgium and 4 more
- Volunteers
- 59 people
- Sponsor
- Lakefront Biotherapeutics NV
Atopic Dermatitis
A Study to Evaluate the Safety of Lebrikizumab Compared to Topical Corticosteroids in Adult Patients With Atopic Dermatitis
The primary objective for this study is to evaluate the safety of lebrikizumab compared with Topical Corticosteroids (TCS) alone in patients with persistent moderate to severe Atopic Dermatitis (AD) that is inadequately…
- Trial name
- NCT02465606
- Testing
- Lebrikizumab, Topical Corticosteroid Creams (Triamcinolone acetonide 0.1% and Hydrocortisone 2.5%)
- Where
- 19 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Hoffmann-La Roche
Cerebral Necrosis, Nashopharyngeal Cancer
Treatment of Cerebral Radiation Necrosis (CRN) With Nerve Growth Factor (NGF)
Cerebral radiation necrosis (CRN) is a well-documented late complication of radiation therapy for cancers, and may have a devastating effect on the patient's quality of life (QOL).
- Conditions
- Cerebral Necrosis, Nashopharyngeal Cancer, Nerve Growth Factor
- Trial name
- NCT02032147
- Testing
- NGF group
- Where
- China
- Volunteers
- 30 people
- Sponsor
- Fudan University
Orthopaedic Surgery
Pharmacokinetic, Pharmacodynamic and Pharmacogenetic of Morphine After Surgery
The main objective of this study is to improve our knowledge on the pharmacodynamic, pharmacokinetic, and pharmacogenetic relationships of morphine administered to relief severe postoperative pain.
- Trial name
- NCT00822549
- Testing
- intravenous morphine titration
- Where
- France
- Volunteers
- 438 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris