Clinical trials: Neonatal
Fabry Disease
Clinical Investigation on the Blood Oxygenation at the Optic Nerve Head in Fabry Patients
This study aimed to evaluate blood oxygenation at the optic nerve head in relation with visual field losses observed in many Fabry patients. Data collected allowed to evaluate if there is a link between these two entities.
- Trial name
- NCT02023086
- Testing
- Contrast sensitivity measurement, Slit Lamp assessment and intra-ocular pressure measurement, Visual field testing
- Where
- Canada
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Université de Montréal
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
- Trial name
- pathfinder™6 (NCT02137850)
- Testing
- turoctocog alfa pegol
- Where
- 96 sites in United States, Algeria and 21 more
- Volunteers
- 124 people
- Sponsor
- Novo Nordisk A/S
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
Cystic Fibrosis
Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA TC Combination Therapy in Participants With Cystic Fibrosis Who Are 6 Years of Age and Older
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
- Trial name
- NCT04183790
- Testing
- ELX/TEZ/IVA, IVA
- Where
- 21 sites in United States, Australia and 3 more
- Volunteers
- 64 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Gout
Open-Label Lesinurad Monotherapy Extension Study in Gout
This study will assess the serum uric acid lowering effects and safety of lesinurad over a long-term timeframe.
- Trial name
- NCT01650246
- Testing
- lesinurad
- Where
- 75 sites in United States, Australia and 5 more
- Volunteers
- 143 people
- Sponsor
- Ardea Biosciences, Inc.
Hemophilia A, Hemophilia B
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
- Trial name
- NCT02740413
- Volunteers
- 86 people
- Sponsor
- Pfizer
Neonatal Abstinence Syndrome
Efficacy and Outcomes of a Non-Pharmacological Intervention for Neonatal Abstinence Syndrome
The purpose of this study is to examine the efficacy of a specially-constructed crib mattress that delivers gentle vibrations (stochastic vibrotactile stimulation) as a complementary, non-pharmacological intervention for treating…
- Trial name
- NCT02801331
- Testing
- Stochastic Vibrotactile Stimulation (SVS)
- Where
- United States
- Volunteers
- 208 people
- Sponsor
- Elisabeth B Salisbury
Urea Cycle Disorders
To Evaluate the Safety of Long-term Use of HPN-100 in the Management of Urea Cycle Disorders (UCDs)
This was an open-label, long-term safety study of HPN-100 (RAVICTI; glycerol phenylbutyrate) in participants with a urea cycle disorder (UCD) who completed the safety extensions of HPN-100-005 (NCT00947544; HPN-100-005SE)…
- Trial name
- NCT01257737
- Testing
- HPN-100
- Where
- 16 sites in United States, Canada
- Volunteers
- 88 people
- Sponsor
- Amgen
Irritable Bowel Syndrome Constipation Predominant
Effects of ROSE-010 on GI Transit in Constipation Predominant Irritable Bowel Syndrome (C-IBS) Patients
This trial will study the effects of an investigational (not FDA approved) medication, ROSE-010, on the movement of food through the stomach, small intestine and colon in females with constipation predominant irritable bowel…
- Trial name
- NCT01056107
- Testing
- ROSE-010
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Mayo Clinic
Infant, Newborn, Hypoxia, Brain
Late Hypothermia for Hypoxic-Ischemic Encephalopathy
This study is a randomized, placebo-controlled, clinical trial to evaluate whether induced whole-body hypothermia initiated between 6-24 hours of age and continued for 96 hours in infants ≥ 36 weeks gestational age with…
- Conditions
- Infant, Newborn, Hypoxia, Brain, Hypoxia-Ischemia, Brain, Encephalopathy, Hypoxic-Ischemic
- Trial name
- NCT00614744
- Testing
- Hypothermia, Normothermic Control
- Where
- 22 sites in United States
- Volunteers
- 168 people
- Sponsor
- NICHD Neonatal Research Network
Hypoxic-Ischemic Encephalopathy, Brain Injury
Prospective Research in Infants With Mild Encephalopathy
A multicenter observational pilot study will be conducted to determine the natural history of infants with early diagnosis (≤ 6 hrs of age) of mild neonatal encephalopathy (NE) who are not qualified for therapeutic hypothermia.
- Conditions
- Hypoxic-Ischemic Encephalopathy, Brain Injury, Neonatal Seizure
- Trial name
- PRIME (NCT01747863)
- Testing
- Neurologic examination
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 63 people
- Sponsor
- McGill University Health Centre/Research Institute of the McGill University Health Centre
Heart Arrest, Birth Asphyxia
SI + CC Versus 3:1 C:V Ratio During Neonatal CPR
Research question In newborn infants requiring CPR, does CC superimposed by sustained inflation compared to 3:1 compression to ventilation ratio improves return of spontaneous circulation? Overall objective: CC superimposed by…
- Conditions
- Heart Arrest, Birth Asphyxia, Bradycardia
- Trial name
- SURV1VE (NCT02858583)
- Testing
- CC+SI, 3:1 C:V
- Where
- 4 sites in Austria, Canada
- Volunteers
- 27 people
- Sponsor
- University of Alberta
Dermatitis, Atopic
A Study in Patients With Atopic Eczema to Test How Effective BI 655130 is and How Well it is Tolerated
The primary objective of this trial is to investigate the safety, tolerability and efficacy of BI 655130 in patients with Atopic Dermatitis (AD) following repeated intravenous administrations compared to placebo.
- Trial name
- NCT03822832
- Testing
- BI 655130
- Where
- 23 sites in United States, Canada and 1 more
- Volunteers
- 51 people
- Sponsor
- Boehringer Ingelheim
Atopic Dermatitis
Efficacy and Safety Study of CD2027 Ointment 3 Microgram Per Gram (mcg/g) Twice Daily Treatment for Adults With at Least Moderate Atopic Dermatitis
This was a multi-center, randomized, double-blind, parallel group study. Efficacy Objectives: To evaluate the efficacy of CD2027 ointment 3 mcg/g applied twice daily over 4 weeks versus its vehicle in adult participants with at…
- Trial name
- NCT00919763
- Testing
- CD2027 Ointment 3 mcg/g, twice daily, Vehicle Ointment, twice daily
- Where
- 14 sites in United States
- Volunteers
- 102 people
- Sponsor
- Galderma R&D
Thalassemia Major With Severe Transfusional Iron Overload
Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia
We hypothesize that the combination treatment with deferasirox and deferiprone will be well tolerated and will result in significant improvement in cardiac and liver iron levels.
- Trial name
- NCT01709032
- Testing
- Deferasirox and deferiprone
- Where
- 2 sites in United States
- Volunteers
- 9 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
- Trial name
- NCT00179998
- Testing
- Recombinant Human DNase (Pulmozyme)
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Nationwide Children's Hospital
Exocrine Pancreatic Insufficiency
Phase 1 Study to Assess Safety and Efficacy of ANG003
Randomized, parallel, active-treatment Phase 1 study of a single dose of orally administered ANG003 with a test meal in adult subjects with cystic fibrosis-related exocrine pancreatic insufficiency.
- Trial name
- NCT06052293
- Testing
- ANG003
- Where
- 20 sites in United States
- Volunteers
- 57 people
- Sponsor
- Anagram Therapeutics, Inc.
Respiratory Distress Syndrome, Newborn
Aerosolized Surfactant in Neonatal RDS
Respiratory distress syndrome (RDS), caused by surfactant deficiency, is the leading cause of mortality and morbidity in preterm infants.
- Trial name
- AS-02 (NCT02294630)
- Testing
- Surfactant
- Where
- United States
- Volunteers
- 159 people
- Sponsor
- Sood, Beena G., MD, MS
Dermatitis, Atopic
A Study for AR100DP1 in Mild to Moderate Atopic Dermatitis (AD)
This is a Phase I/IIa Open-Label, Dose-Escalation Study to Determine the Safety, Tolerability, and Efficacy of AR100DP1 in Health Subjects, and Subjects with Mild to Moderate Atopic Dermatitis.
- Trial name
- NCT04220411
- Testing
- AR100DP1
- Where
- Taiwan
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- Arjil Pharmaceuticals LLC