Clinical trials: Effect Neonatal
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.
Enterocolitis, Necrotizing, Bacteremia
Antibiotic "Dysbiosis" in Preterm Infants
Prolonged antibiotic use in preterm neonates has significant consequences on the developing intestinal microbiome, metabolome and host response, predisposing the neonate to various major morbidities, including necrotizing…
- Conditions
- Enterocolitis, Necrotizing, Bacteremia, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage
- Trial name
- NCT02784821
- Testing
- Antibiotic, Gastric fluid, Breast milk
- Where
- United States
- Volunteers
- 98 people
- Sponsor
- University of Florida
Smith-Lemli-Opitz Syndrome
SLOS: The Effect of Simvastatin in Patients Receiving Cholesterol Supplementation
The purpose of this study is to determine if simvastatin improves development and behavior in patients with Smith Lemli-Opitz syndrome (SLOS) receiving dietary cholesterol supplementation.
- Trial name
- NCT01434745
- Testing
- Simvastatin, Lactose
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Oregon Health and Science University
Hypoplastic Left Heart Syndrome, Hypoplastic Right-sided Heart Complex
Ambrisentan in Single Ventricle
Purpose: To evaluate the pharmacokinetics, bioavailability and hemodynamic efficacy of ambrisentan after Fontan surgical palliation of single ventricle heart defects.
- Trial name
- NCT02080637
- Testing
- Ambrisentan
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Kevin Hill
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.
Cystinuria
Lipoic Acid Supplement for Cystine Stone
This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive 1200 mg alpha lipoic acid orally daily for three years, while the other half will receive…
- Trial name
- ALA (NCT02910531)
- Testing
- Alpha lipoic acid
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Thomas Chi, MD
Atopic Dermatitis
A Study to Determine the Efficacy of ZPL-3893787 in Subjects With Atopic Dermatitis
This was a randomized, double blind, placebo controlled, parallel group study in approximately 90 subjects with moderate-severe AD Eczema Area and Severity Index (EASI) ≥12 and ≤ 48 (0-72 scale).
- Trial name
- NCT02424253
- Testing
- ZPL-3893787
- Where
- 17 sites in Belgium, Germany and 2 more
- Volunteers
- 98 people
- Sponsor
- Ziarco Pharma Ltd
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Phenylketonuria
Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients
This double-blind, placebo-controlled, randomized study is designed to evaluate the safety and therapeutic effects of sapropterin dihydrochloride on neuropsychiatric symptoms in subjects with PKU.
- Trial name
- NCT01114737
- Testing
- Sapropterin dihydrochloride
- Where
- 36 sites in United States, Canada
- Volunteers
- 206 people
- Sponsor
- BioMarin Pharmaceutical
Lennox-Gastaut Syndrome
Study of Rufinamide in Pediatric Subjects 1 to Less Than 4 Years of Age With Lennox-Gastaut Syndrome Inadequately Controlled With Other Anti-epileptic Drugs
This study was designed to evaluate the cognitive effect, safety, and pharmacokinetics (PK) of rufinamide on Lennox-Gastaut Syndrome (LGS) inadequately controlled in pediatric participants already taking other anti-epileptic…
- Trial name
- NCT01405053
- Testing
- Rufinamide, Any other approved Antiepileptic Drug
- Where
- 47 sites in United States, Canada and 5 more
- Volunteers
- 37 people
- Sponsor
- Eisai Inc.
Hypophosphatasia
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP).
- Trial name
- NCT05890794
- Testing
- Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- AM-Pharma
Ototoxicity
SPI-1005 for Prevention and Treatment of Tobramycin Induced Ototoxicity
The primary objective of this study is to determine the safety and efficacy of SPI-1005 treatment in CF patients with active pulmonary exacerbation that are receiving an IV course of tobramycin, determined by comparing hearing…
- Trial name
- NCT02819856
- Testing
- SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sound Pharmaceuticals, Incorporated
ASCVD, Elevated Cholesterol
Trial to Assess the Effect of Long Term Dosing of Inclisiran in Subjects With High CV Risk and Elevated LDL-C
The purpose of this extension study was to evaluate the efficacy, safety, and tolerability of long-term dosing of Inclisiran. The study was a global multicenter study.
- Conditions
- ASCVD, Elevated Cholesterol, Heterozygous Familial Hypercholesterolemia, Homozygous Familial Hypercholesterolemia
- Trial name
- ORION-8 (NCT03814187)
- Testing
- Inclisiran Sodium
- Where
- 238 sites in United States, Canada and 11 more
- Volunteers
- 3,275 people
- Sponsor
- Novartis Pharmaceuticals
Huntington's Disease
The Effect of Video Game Exercise on Dynamic Balance and Gait in Individuals With Huntington's Disease
To examine the benefits of using a video-game, Dance, Dance, Revolution, as an exercise modality to improve gait and balance in individuals with Huntington's disease.
- Trial name
- NCT01735981
- Testing
- Video game exercise using Dance Dance Revolution, hand-held video game
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Anne Kloos
Anesthesia; Adverse Effect, Congenital Heart Disease
The Effect of Anesthesia on Neurodevelopmental Outcome (NDO)
The purpose of this study is to assess whether the type of anesthesia, narcotic-based versus inhalational anesthesia administered during cardiopulmonary bypass (CPB) surgery contributes to the wide variation in neurologic…
- Conditions
- Anesthesia; Adverse Effect, Congenital Heart Disease, Neurodevelopmental Disorders
- Trial name
- NDO (NCT03882788)
- Testing
- Isoflurane, Fentanyl (high dose), Fentanyl (low dose)
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- Stanford University
Cystic Fibrosis
Effect of Lucinactant on Mucus Clearance in Cystic Fibrosis Lung Disease
Mucus clearance is impaired in cystic fibrosis. Inhaled surfactants may reduce adhesive forces between mucus and airway surfaces and improve mucus clearance. This in turn my improve lung health.
- Trial name
- NCT00934362
- Testing
- Lucinactant first
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of North Carolina, Chapel Hill
Dermatitis Atopic
Dupilumab Effect on Pruritus Neuro-mechanisms in Patients With Atopic Dermatitis
Primary Objective: Assess change in neuronal architecture following long term treatment with dupilumab in skin biopsies from atopic dermatitis (AD) participants with chronic pruritus.
- Trial name
- DIFFEREN-STAD (NCT04823130)
- Testing
- Dupilumab (SAR231893)
- Where
- 3 sites in United States, Germany
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Sanofi
Skin Abnormalities
Evaluation of Effects of HP828-101 for Moisture Associated Skin Damage
This study evaluates HP828 in the management of moisture associated skin damage after 15 days of use.
- Trial name
- NCT01108523
- Testing
- HP828-101
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Healthpoint
Pain, Sickle Cell Disease
Choosing Opioid Management for Pain and Analyzing Acute Chest Syndrome (ACS) Rates Equally
The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive…
- Trial name
- COMPARE (NCT01380197)
- Testing
- Morphine, Nubain
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Children's Healthcare of Atlanta
Effects of Different Meals on the QT/QTc Interval, Insulin and Oral Hypoglycemic [Antidiabetic] Drugs Causing Adverse Effects in Therapeutic Use
Food and Insulin Effect on QT/QTC Interval of ECG
Moxifloxacin is routinely used as a probe to confirm assay sensitivity in thorough electrocardiogram (ECG) studies. It has been shown that a meal shortens the QT interval, which may affect pharmacokinetics (PK) and/or…
- Conditions
- Effects of Different Meals on the QT/QTc Interval, Insulin and Oral Hypoglycemic [Antidiabetic] Drugs Causing Adverse Effects in Therapeutic Use, C-Peptide Effects on the QT/QTc Interval, Moxifloxacin ECG Profile in Fed and Fasted State
- Trial name
- C11035 (NCT01642485)
- Testing
- Moxifloxacin 400 mg fasted, FDA breakfast, Continental breakfast
- Where
- United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Richmond Pharmacology Limited