Clinical trials: Congenital Disorder
Cystic Fibrosis
"Safety, Tolerability and Pharmacokinetics of MP-376 Administered for 14 Days to Stable Pediatric (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
- NCT00840333
- Testing
- MP-376 (Levofloxacin solution for Inhalation)
- Where
- 6 sites in United States
- Volunteers
- 27 people
- Sponsor
- Amgen
Muscular Dystrophy, Duchenne
Safety Study of Eteplirsen to Treat Advanced Stage Duchenne Muscular Dystrophy
The primary objective of this study is to explore safety and tolerability of eteplirsen in participants with advanced stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.
- Trial name
- NCT02286947
- Testing
- Eteplirsen
- Where
- 9 sites in United States
- Volunteers
- 24 people
- Sponsor
- Sarepta Therapeutics, Inc.
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Long-Term Treatment and Follow up of Subjects Completing 24 Months of Treatment With Tesevatinib on Study KD019-101
Subjects who received tesevatinib in Study KD019-101 and completed 24 months of treatment will continue on the dose of tesevatinib they were receiving at 24 months on the KD019-101 study.
- Trial name
- NCT02616055
- Testing
- tesevatinib
- Where
- 6 sites in United States
- Volunteers
- 15 people
- Sponsor
- Kadmon Corporation, LLC
Cystic Fibrosis
MP-376 (Aeroquin™, Levofloxacin for Inhalation) in Patients With Cystic Fibrosis
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
- NCT01180634
- Testing
- Aeroquin
- Where
- 112 sites in United States, Australia and 3 more
- Volunteers
- 330 people
- Sponsor
- Amgen
Dilated Cardiomyopathy
Safety and Feasibility of Algisyl-LVR™ as a Method of Left Ventricular Restoration in Patients With DCM Undergoing Open-heart Surgery
This is a pilot study to evaluate the feasibility and safety of the Algisyl-LVR™ device. The purpose of this study is to investigate Algisyl-LVR™ employed as a method of left ventricular restoration in patients with dilated…
- Trial name
- NCT00847964
- Testing
- Algisyl-LVR
- Where
- 3 sites in Germany, Poland
- Volunteers
- 11 people
- Sponsor
- LoneStar Heart, Inc.
Muscular Dystrophies
Phase II Doubleblind Exploratory Study of GSK2402968 in Ambulant Subjects With Duchenne Muscular Dystrophy
The purpose of this study is to determine whether GSK2402968 given as a continuous dose and as an intermittent dose is effective and safe in the treatment of Duchenne muscular dystrophy.
- Trial name
- NCT01153932
- Testing
- GSK2402968
- Where
- 13 sites in Australia, Belgium and 7 more
- Volunteers
- 53 people
- Sponsor
- GlaxoSmithKline
Atopic Dermatitis
Efficacy of KAM2904 Face Cream and KAM3008 Body Lotion Treatment in Children With Atopic Dermatitis (AD)
The main purpose of this study is to demonstrate the safety and efficacy of KAM2904 Face Cream and KAM3008 Body Lotion in reducing the symptoms of mild to moderate AD.
- Trial name
- NCT01781663
- Testing
- KAM2904 Face Cream, KAM3008 Body Lotion, petrolatum-based moisturizer
- Where
- Spain
- Volunteers
- 56 people
- Sponsor
- Kamedis Ltd.
Dermatitis, Atopic
Safety, Tolerability, Pharmacokinetics and Efficacy of WOL071-007 in Atopic Dermatitis Patients
Purpose of the study is the local tolerability and systemic safety of a novel k-opioid receptor agonist proven to inhibit inflammation and pruritus in preclinical model of dermatitis.
- Trial name
- NCT02576093
- Testing
- 0.1% WOL071-007, 0.3% WOL071-007, 1.0% WOL071-007
- Where
- Germany
- Volunteers
- 58 people
- Sponsor
- Dr. August Wolff GmbH & Co. KG Arzneimittel
Huntington's Disease
Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study.
- Trial name
- NCT04120493
- Testing
- intra-striatal rAAV5-miHTT
- Where
- 12 sites in United States
- Volunteers
- 43 people
- Sponsor
- UniQure Biopharma B.V.
Eczema
Test to Determine the Effectiveness of Moisturizing Balm Used on Babies With Dry, Itchy Skin
The purpose of this 3 week study is to test the effectiveness of an over-the-counter (OTC) skin balm in infants and toddlers with mild to moderate dry, itchy skin.
- Trial name
- NCT02404493
- Testing
- EpiCeram Skin Barrier Emulsion, 1% Colloidal Oatmeal Balm
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)
Cystic Fibrosis
Study of Denufosol Tetrasodium in Patients With Cystic Fibrosis Lung Disease
The objectives of this study are to evaluate the safety, tolerability and preliminary efficacy of two dose levels of INS37217 (denufosol tetrasodium) Inhalation Solution in patients with cystic fibrosis (CF) lung disease.
- Trial name
- NCT00103714
- Testing
- denufosol tetrasodium (INS37217)
- Volunteers
- 72 people
- Sponsor
- Merck Sharp & Dohme LLC
Fabry Disease
Replagal Enzyme Replacement Therapy for Children With Fabry Disease
Primary Objective(s): To assess the safety of Replagal at a dose of 0.2 mg/kg administered over 40 (+/-10) minutes in children with Fabry disease To assess the effect of Replagal on heart rate variability in patients 7 to 17…
- Trial name
- NCT00084084
- Testing
- Agalsidase alfa
- Where
- 14 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Turner Syndrome, Renal Insufficiency, Chronic
Study to Define Optimal IGF-1 Monitoring in Children Treated With NutropinAq
The main purpose of this study is to establish an optimal monitoring regimen in NutropinAq treated children, using newly developed capillary blood spot IGF-1 measurement technology.
- Conditions
- Turner Syndrome, Renal Insufficiency, Chronic, Pituitary Diseases, Dwarfism
- Trial name
- OPTIMA (NCT00234533)
- Testing
- Somatropin (rDNA origin)
- Where
- 45 sites in Belgium, Czechia and 12 more
- Volunteers
- 251 people
- Sponsor
- Ipsen
Wilson's Disease
International Wilson's Disease Patient Registry (iWilson Registry)
Longitudinal, observational, non-interventional, standard of care Registry. Data will be collected from the routinely scheduled WD clinic visits at approximately 6-12 month intervals.
- Trial name
- NCT05239858
- Where
- 16 sites in Belgium, France and 5 more
- Volunteers
- 500 people
- Sponsor
- Orphalan
Congenital Bleeding Disorder, Healthy
Dose Response to Recombinant Factor VIIa When Administered for Bleed
This trial is conducted in the United States of America (USA). The purpose of this trial is to investigate the dose response to recombinant factor VIIa in healthy volunteers when administered for bleed.
- Trial name
- NCT00104455
- Testing
- activated recombinant human factor VII
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
Hypertension, Angina Pectoris
Amlodipine/Atorvastatin (Caduet®) Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
In this survey, to collect the safety and efficacy information of Amlodipine /Atorvastatin (Caduet® Combination Tablets) in daily medical practice will be examined.
- Conditions
- Hypertension, Angina Pectoris, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- NCT01107743
- Testing
- Amlodipine/Atorvastatin
- Volunteers
- 1,291 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Huntington Disease
Wearable Sensors for Quantitative Assessment of Motor Impairment in Huntington's Disease Huntington's Disease
The principal means of measuring motor impairment in Huntington disease (HD) is the Unified Huntington's Disease Rating Scale (UHDRS) total motor score, which is subjective, categorical, requires significant training to…
- Trial name
- NCT03599076
- Testing
- HD Wear
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- BioSensics
Von Hippel Lindau Disease
MSD Belzutifan PAS
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice.
- Trial name
- NCT06554730
- Testing
- Belzutifan
- Sponsor
- Merck Sharp & Dohme LLC
Atopic Dermatitis (AD)
A Phase III Clinical Study of QY201 Tablet in Subjects With Moderate to Severe Atopic Dermatitis.
To evaluate the efficacy of QY201 tablets in the treatment of adult patients with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT07180511
- Testing
- 10mg QY201 tablet, 20mg QY201 tablet
- Where
- China
- Volunteers
- 460 people
- Sponsor
- E-nitiate Biopharmaceuticals (Hangzhou) Co., Ltd.
Cystic Fibrosis
A Study of VX-445 in Healthy Subjects and Subjects With Cystic Fibrosis
This is a first-in-human and proof-of-concept study of VX-445. The study includes 6 parts. Parts A, B, and C were conducted in healthy subjects.
- Trial name
- NCT03227471
- Testing
- IVA, TEZ/IVA, VX-445
- Where
- 38 sites in United States, Australia and 2 more
- Volunteers
- 225 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
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