Clinical trials: Effect Neonatal
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
The purpose of this study is to compare the bioavailability of the Test Product, Nitisinone 10 mg Tablet, under fasting and fed conditions (food-effect).
- Trial name
- NCT02750332
- Testing
- Nitisinone
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Congenital Heart Defect, Surgery-Induced Tissue Adhesions
Effects of CoSeal on Bleeding & Adhesions in Pediatric Heart Surgery
This is a prospective, randomized, open-label, blinded-evaluator study that will evaluate the effectiveness of a surgical sealant (CoSeal) composed of biocompatible polyethylene glycol on the formation of mediastinal and…
- Conditions
- Congenital Heart Defect, Surgery-Induced Tissue Adhesions, Hemorrhage
- Trial name
- NCT01330433
- Testing
- CoSeal Surgical Spray Group
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Loma Linda University
Bicuspid Aortic Valve, Thoracic Aortic Aneurysm
The Effect of Losartan in Bicuspid Aortic Valve Patients
The specific aims of this study are to: Establish baseline levels of circulating MMP-2 and -9 , TIMP-1and- 2 and TGFB levels in individuals with bicuspid aortic valve and ascending aortic or aortic sinus measurements >40mm.
- Trial name
- NCT01390181
- Testing
- Cozaar
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Michigan
Pseudohypoparathyroidism Type 1a, Albright Hereditary Osteodystrophy
Theophylline as a Treatment for Children With Pseudohypoparathyroidism Type 1a (Albright Hereditary Osteodystrophy)
This study will test an investigational drug, theophylline, in children with pseudohypoparathyroidism type 1a (PHP1a). This study involves a 3 day visit to the Vanderbilt Clinical Research Center.
- Trial name
- NCT02463409
- Testing
- Theophylline
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Vanderbilt University Medical Center
Sickle Cell Disease
Voxelotor Neurocognitive Function Study
This is a Phase 3b, randomized, double-blind, placebo-controlled, multicenter study to assess the treatment effect of voxelotor on neurocognitive function as assessed by the National Institute of Health (NIH) Toolbox Cognition…
- Trial name
- NCT05228834
- Testing
- Voxelotor Only Product in Oral Dose Form
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pfizer
Single-ventricle
Simple Non-invasive Breathing Device to Improve Pulmonary Flow Pusatility in Single Ventricle Post Fontan
This is a pilot study using a novel, minimal risk, portable, hands-free oral-positive pressure device (oPEP) in patients with Fontan palliation that will examine whether using this device in both the acute and chronic phase will…
- Trial name
- NCT05634226
- Testing
- Hands-free oral-positive pressure device (oPEP)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Tarek Alsaied
Prematurity
The Effect of Lipid Emulsions on Free Fatty Acids and Free Bilirubin in Premature Newborns
The goal of this research study is to compare the levels of free fatty acids and free bilirubin between two intravenous lipid emulsion products in premature newborns.
- Trial name
- NCT03857646
- Testing
- Intralipid, 20% Intravenous Emulsion, Soybean oil Medium-chain triglycerides Olive oil Fish Oil (SMOF) lipid
- Where
- United States
- Volunteers
- 66 people
- Sponsor
- The University of Texas Health Science Center, Houston
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Hypertension, Congestive Cardiomyopathy
The Acute Effects of the Angiotensin-converting Enzyme Inhibitor Enalaprilat on Flow Distribution
The primary objective of this study is to study the acute effects of angiotensin-converting enzyme inhibitor (ACEI) on systemic, pulmonary and cerebral blood flow in post bidirectional cavopulmonary connection (BCPC) patients.
- Trial name
- NCT00741156
- Testing
- Enalaprilat
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- The Hospital for Sick Children
Osteogenesis Imperfecta
Multicenter,Single-arm Study to Evaluate Efficacy, Safety, & Pharmacokinetics of Denosumab in Children w/ OI
This is a prospective, multicenter, single-arm study in children 2 to 17 years of age with OI to evaluate efficacy and safety of denosumab.
- Trial name
- OI (NCT02352753)
- Testing
- Denosumab
- Where
- 49 sites in United States, Australia and 11 more
- Volunteers
- 153 people
- Sponsor
- Amgen
Huntington's Disease
A Study to Evaluate the Effect of SAGE-718 on Cognitive Function in Participants With Huntington's Disease (HD)
The primary purpose of this study is to evaluate the effect of SAGE-718 on cognitive performance and functioning in participants with HD.
- Trial name
- NCT05107128
- Testing
- SAGE-718
- Where
- 49 sites in United States, Australia and 2 more
- Volunteers
- 189 people
- Sponsor
- Supernus Pharmaceuticals, Inc.
Atopic Dermatitis
Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5)
The purpose of this trial is to test if treatment with the trial drug, tralokinumab, can affect the body's immune response to vaccines.
- Trial name
- ECZTRA 5 (NCT03562377)
- Testing
- Tralokinumab, Tdap vaccine, Meningococcal vaccine
- Where
- 51 sites in United States, Canada
- Volunteers
- 215 people
- Sponsor
- LEO Pharma
Huntington Disease
Effect of Tetrabenazine on Stroop Interference in HD
Tetrabenazine has been shown to improve gating of abnormal visual stimuli and improve postural stability in Huntington disease (HD) patients as measured by computerized dynamic posturography testing.
- Trial name
- NCT01834911
- Testing
- Tetrabenazine withdrawal
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- New York Medical College
Cystic Fibrosis
Clinical Effects of Exercise Program Added to Pulmonary Rehabilitation in Patients With Cystic Fibrosis
The aim of this study is to investigate the effects of postural exercise program added to pulmonary rehabilitation program on quality of life, exercise tolerance and postural stability in children with Cystic Fibrosis.
- Trial name
- NCT03295201
- Testing
- Active cycle of breathing techniques (ACBT), Postural Exercise
- Where
- Turkey (Türkiye)
- Volunteers
- 22 people
- Sponsor
- Marmara University
Melanoma
The Effects of Treatment With Vemurafenib on the Immune System in Advanced Melanoma
Approximately 40-60 % of cutaneous melanomas select for a mutation in a protein called BRAF which is part of a signaling pathway called the Mitogen Activated Protein Kinase (MAPK) pathway.
- Trial name
- NCT01942993
- Testing
- Vemurafenib
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Philip Friedlander
Lipid Metabolism, Inborn Errors, Hyperlipidemias
Measure Liver Fat Content After ISIS 301012 (Mipomersen) Administration
This study will assess what, if any, effect that ISIS 301012 (mipomersen) has on liver triglyceride content in multiple groups of subjects with varying degrees of risk for hepatic steatosis.
- Conditions
- Lipid Metabolism, Inborn Errors, Hyperlipidemias, Metabolic Diseases, Hypolipoproteinemia
- Trial name
- NCT00362180
- Testing
- mipomersen
- Where
- Netherlands
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- Kastle Therapeutics, LLC
Hemophilia A
Russian Kogenate Pediatric Study
A prospective study to evaluate the effect of rFVIII-FS in different prophylactic regimens on bleeding events frequency and development of arthropathy in Previously Treated and Minimally treated Hemophilia A pediatric population.
- Trial name
- NCT00632814
- Testing
- rFVIII-FS (Kogenate FS, BAY14-2222) 70 IU/kg, dosing once per week, rFVIII-FS (Kogenate FS, BAY14-2222), 70 IU/kg twice per week (30 IU/kg + 40 IU/kg), rFVIII-FS (Kogenate FS, BAY14-2222) 75 IU/kg, dosing three times per week (3 x 25 IU/kg)
- Where
- 4 sites in Russia
- Volunteers
- 32 people
- Sponsor
- Bayer
Huntington Disease
Within Subject Crossover Study of Cognitive Effects of Neflamapimod in Early-Stage Huntington Disease
This is a double-blind, placebo-controlled 2-period 10-week treatment within-subject crossover study of neflamapimod in early-stage Huntington disease (HD).
- Trial name
- NCT03980938
- Testing
- neflamapimod
- Where
- United Kingdom
- Volunteers
- 15 people
- Sponsor
- EIP Pharma Inc
Spinal Muscular Atrophy Type 3
Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) are one of the common physical disabilities in childhood. For SMA, progressive muscle weakness and early fatigue hamper the mobility of the sufferers.
- Trial name
- NCT03056144
- Testing
- whole body vibration therapy
- Where
- Hong Kong
- Volunteers
- 1 people
- Sponsor
- The Hong Kong Polytechnic University
Breastfeeding
Effect of Early Skin to Skin Contact on Breast Feeding Behaviour in Term Newborns: A Randomized Controlled Trial
Aims and Objectives Aim To determine the effect of delivery room Skin To Skin contact on breast feeding behavior of term neonates born by normal vaginal delivery at 36-48 hours.
- Trial name
- NCT00776789
- Testing
- skin to skin contact, Control group
- Where
- India
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- All India Institute of Medical Sciences