Clinical trials: Newborn
Cystic Fibrosis
Safety and Efficacy Study of Aztreonam for Inhalation Solution (AZLI) in Cystic Fibrosis Patients With P. Aeruginosa
The purpose of this study was to evaluate the safety and efficacy of aztreonam for inhalation solution (AZLI) in patients with cystic fibrosis (CF) and lung infection due to Pseudomonas aeruginosa (PA).
- Trial name
- AIR-CF2 (NCT00104520)
- Testing
- AZLI 75 mg two times a day (BID)/three times a day (TID)
- Where
- 56 sites in United States
- Volunteers
- 211 people
- Sponsor
- Gilead Sciences
Birt-Hogg-Dube Syndrome
Assessment of Safety of Air Travel in Patients With Birt-Hogg-Dube Syndrome
The aim of this study is to conduct survey-based assessments for the safety of air travel in patients with Birt-Hogg-Dube syndrome (BHD).
- Trial name
- NCT03040115
- Testing
- Non-Interventional Study
- Where
- United States
- Volunteers
- 104 people
- Sponsor
- University of Cincinnati
Diabetes Mellitus, Type 1
A Research Trial of Aralast in New Onset Diabetes (RETAIN)
The drug Alpha-1 Antitrypsin (AAT, Aralast NP) is being tested in this study as an anti-inflammatory drug (a medication that decreases inflammation, which is part of the body's normal ability to fight infection and respond to…
- Trial name
- RETAIN (NCT01183468)
- Testing
- Aralast NP 45 mg dose, Aralast NP 90 mg dose, Aralast NP 180 mg dose
- Where
- 15 sites in United States
- Volunteers
- 17 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
High-Frequency Ventilation, Intermittent Positive-Pressure Ventilation
Nasal HFOV Versus Nasal SIPPV in Neonate Following Extubation: RCT Crossover Study
Mechanical ventilation was introduced to treat respiratory failure in preterm infants or sick neonates then improvements in survival (1,2).
- Conditions
- High-Frequency Ventilation, Intermittent Positive-Pressure Ventilation, Newborn Morbidity, Noninvasive Ventilation
- Trial name
- nHFOnSIPPV (NCT04323397)
- Testing
- Non-invasive ventilation
- Where
- Thailand
- Volunteers
- 133 people
- Sponsor
- Prince of Songkla University
Dent Disease
Role Of Phosphorus And FGF 23 In Patients With Dent Disease
Patients with Dent disease have suppressed levels of FGF 23 which contributes to hypercalciuria, kidney stones, nephrocalcinosis and renal failure. Supplementation with phosphorus may reduce hypercalciuria.
- Trial name
- NCT02016235
- Testing
- Phosphorus Supplement, Observation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Mayo Clinic
Hypothalamic Obesity
Open-Label Study of Setmelanotide in Hypothalamic Obesity
Open-label, single-arm study designed to evaluate the body weight response to setmelanotide administered subcutaneously (SC) daily in participants with hypothalamic obesity (HO).
- Trial name
- NCT04725240
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Pulmonary Stenosis, Pulmonary Regurgitation
Pulmonary Artery Repair With Covered Stents
The Covered Cheatham-Platinum Stent (CCPS) is being study for repair of tears that occur in the pulmonary artery during dilation (enlargement) of a conduit (passageway) connecting the right ventricle of the heart to the pulmonary…
- Conditions
- Pulmonary Stenosis, Pulmonary Regurgitation, Tetralogy of Fallot
- Trial name
- PARCS (NCT01824160)
- Testing
- Repair of RV-PA Conduit Disruption
- Where
- 38 sites in United States
- Volunteers
- 50 people
- Sponsor
- Johns Hopkins University
Hereditary Angioedema
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab.
- Trial name
- SPRING (NCT04070326)
- Testing
- Lanadelumab
- Where
- 17 sites in United States, Canada and 3 more
- Volunteers
- 21 people
- Sponsor
- Shire
Down Syndrome, Cognitive Dysfunction
Evaluating The Efficacy And Safety Of Donepezil Hydrochloride (Aricept) In The Treatment Of The Cognitive Dysfunction Exhibited By Children With Down Syndrome, Aged 11 To 17
The purpose of this study is to determine the efficacy and safety of donepezil hydrochloride (Aricept) in the treatment of the cognitive dysfunction shown by children with Down syndrome, aged 11 to 17.
- Trial name
- NCT00754052
- Testing
- Aricept (donepezil hydrochloride)
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Eisai Inc.
Type 3 Von Willebrand's Disease
Type 3 Von Willebrand International Registries Inhibitor Prospective Study
International Registries and Prospective Study on Type 3 Von Willebrand's Disease (VWD3), aimed to assess number, types and risk factors for bleeding and the efficacy and safety of plasma-derived and/or recombinant Von Willebrand…
- Trial name
- 3WINTERS-IPS (NCT02460458)
- Testing
- Von Willebrand Factor
- Where
- 25 sites in Finland, France and 8 more
- Volunteers
- 265 people
- Sponsor
- Fondazione Angelo Bianchi Bonomi
Cardiac Transplant, Patent Ductus Arterious
The Pharmacology and Hemodynamics of Dexmedetomidine in Children With Congenital Heart Disease
The purpose of this study is to examine the pharmacokinetics, pharmacodynamics, and pharmacogenomics of dexmedetomidine in the following three pediatric patient populations: patients with bi-directional cavopulmonary anastomosis…
- Conditions
- Cardiac Transplant, Patent Ductus Arterious, Atrial Septal Defect, Bidirectional Cavopulmonary Anastomosis
- Trial name
- NCT00480740
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Children's National Research Institute
Congenital Heart Disease, Bradycardia Sinus
Specialized Pacing for Patients With Congenital Heart Disease
The closed-loop stimulation (CLS) algorithm is a novel sensor-based technology that relies on the change in myocardial systolic impedance for modulation of the heart rate during physical and emotional stress.3 The pacing…
- Trial name
- NCT03361189
- Testing
- Closed loop stimulation, Standard rate response
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of California, Los Angeles
Chronic Hepatitis C Infection
Open-Labeled Study of PSI-7977 and RBV With and Without PEG-IFN in Treatment-Naïve Patients With HCV GT2 or GT3
This study is to assess the safety and tolerability of sofosbuvir (SOF) 400 mg with and without ribavirin (RBV) and/or with and without pegylated interferon alfa-2a (PEG) in subjects with genotype 1, 2 or 3 hepatitis C (HCV)…
- Trial name
- NCT01260350
- Testing
- SOF, RBV, PEG
- Where
- 2 sites in New Zealand
- Volunteers
- 292 people
- Sponsor
- Gilead Sciences
Physical Disability
A Community Wellness Program for Adults Living With Long-term Physical Disability
For people living with long-term physical disabilities, such as spinal cord injury or multiple sclerosis, middle-age (45-64) is a period of great vulnerability for losses in function and participation.
- Trial name
- EW-D (NCT03653390)
- Testing
- EnhanceWellness for Disability (EW-D), Wellness Education
- Where
- United States
- Volunteers
- 516 people
- Sponsor
- University of Washington
Sly Syndrome, MPS VII
Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age
The primary objective was to evaluate the effect of UX003 treatment in pediatric MPS VII participants less than 5 years of age on safety, tolerability, and efficacy as determined by the reduction of urinary glycosaminoglycans…
- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Trial name
- NCT02418455
- Testing
- UX003
- Where
- 5 sites in United States, Portugal and 1 more
- Volunteers
- 8 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Gastroschisis
Evaluation of Glycerin Suppositories to Improve Bowel Function in Gastroschisis
Gastroschisis is a congenital defect of the abdominal wall that leads to evisceration of various amounts of the abdominal organs.
- Trial name
- NCT03355326
- Testing
- Glycerin Suppository
- Where
- United States
- Volunteers
- 16 people, healthy volunteers welcome
- Sponsor
- Indiana University
Fragile X Syndrome
Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Fragile X Syndrome (FXS) is the most common known inherited form of mental impairment, developmental disability and autism. Minocycline is an antibiotic that has recently been used to treat the mouse model for Fragile X, and was…
- Trial name
- NCT00858689
- Testing
- Minocycline
- Where
- Canada
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- FRAXA Research Foundation
Hemophilia, Factor XIII Deficiency
Clinical Trial of Factor XIII (FXIII) Concentrate
Congenital deficiency of Factor XIII is a rare but potentially life threatening disorder. It is inherited in an autosomal recessive fashion.
- Trial name
- NCT00640289
- Testing
- Fibrogammin P
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- Children's Hospital of Orange County
Hemophilia A
A Study of PEGylated Recombinant Factor VIII (BAX855) in Previously Untreated Young Children With Severe Hemophilia A
This study is for young children with severe hemophilia A who have previously not been treated with BAX855 or other FVIII concentrates. The main aim of the study is to check for side effects from treatment with BAX855.
- Trial name
- NCT02615691
- Testing
- PEGylated Recombinant Factor VIII, ITI
- Where
- 91 sites in United States, Austria and 21 more
- Volunteers
- 120 people
- Sponsor
- Baxalta now part of Shire
Atopic Dermatitis
A Study to Learn How Well the Study Treatment Zabedosertib (BAY1834845) Works and How Safe it is Compared to Placebo in Adult Participants With Moderate-to-severe Atopic Dermatitis
Researchers are looking for a better way to treat atopic dermatitis (AD), an often long-lasting inflammation of the skin. Atopic dermatitis, also called eczema, is causing patches of skin to become swollen, red, cracked, and…
- Trial name
- Damask (NCT05656911)
- Testing
- Zabedosertib (BAY1834845)
- Where
- 22 sites in United States, Czechia and 5 more
- Volunteers
- 77 people
- Sponsor
- Bayer
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