Clinical trials: Effect Neonatal
Adrenoleukodystrophy
Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
OBJECTIVES: I. Evaluate the clinical efficacy of combination glyceryl trierucate and glyceryl trioleate (Lorenzo's Oil) therapy in boys with X-linked adrenoleukodystrophy. II.
- Trial name
- NCT00004418
- Testing
- glyceryl trierucate/glyceryl trioleate
- Where
- 2 sites in United States
- Volunteers
- 126 people
- Sponsor
- Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sickle Cell Anemia, Sickle Cell-Beta0-Thalassemia
The Effect of Rivaroxaban in Sickle Cell Disease
The primary study hypothesis is that inhibition of factor Xa with rivaroxaban will reduce inflammation, coagulation and endothelial cell activation, and improve microvascular blood flow in patients with sickle cell disease (SCD)…
- Trial name
- NCT02072668
- Testing
- rivaroxaban
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- University of North Carolina, Chapel Hill
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Alpha-1 Antitrypsin Deficiency
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT).
- Trial name
- NCT02363946
- Testing
- ARC-AAT Injection, Diphenhydramine
- Where
- 4 sites in Australia, Germany and 2 more
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Muscular Dystrophy, Duchenne
Effect of EPA and DHA in the Inflammation and Metabolic Disorders in DMD/DMB Patients
The purpose of this study is to evaluate the effect of docosahexaenoic fatty acid and eicosapentaenoic fatty acid supplementation for six months on the inflammation state as well as the process of muscular regeneration and the…
- Trial name
- NCT01826422
- Testing
- EPA and DHA
- Where
- Mexico
- Volunteers
- 40 people
- Sponsor
- Coordinación de Investigación en Salud, Mexico
Sickle Cell Disease
Evaluation of Different Dose Regimens of Aes-103 Given for 28 Days to Subjects With Stable Sickle Cell Disease
Sickle cell disease (SCD) is a genetic blood disorder characterized by the presence of sickle-shaped red blood cells. In the U.S. and the U.K. this occurs primarily in persons of African origin.
- Trial name
- NCT01987908
- Testing
- Aes-103
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- Baxalta now part of Shire
Joint Damage
Effect of Febuxostat on Joint Damage in Hyperuricemic Subjects With Early Gout
This purpose of this study is to assess the effect of febuxostat, once daily (QD), on joint damage in patients with elevated serum urate levels and gout.
- Trial name
- NCT01078389
- Testing
- Febuxostat
- Where
- 56 sites in United States
- Volunteers
- 314 people
- Sponsor
- Takeda
Myotonic Dystrophy
Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)
The aim of this study is to investigate the safety and feasibility of daily subcutaneous injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a treatment for muscle wasting and weakness in…
- Trial name
- NCT00233519
- Testing
- SomatoKine/IPLEX
- Where
- United States
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Sickle Cell Disease
Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD
This registry is an observational study designed to evaluate the effect of Oxbryta in individuals with SCD in a real-world setting.
- Trial name
- PROSPECT (NCT04930445)
- Testing
- Oxbryta® (voxelotor) 500mg Tablets
- Where
- 43 sites in United States
- Volunteers
- 265 people
- Sponsor
- Pfizer
Heart Defects, Congenital, Cardiopulmonary Bypass
Nesiritide Use Following Cardiac Surgery in Infants
The purpose of this study is to determine the effects of nesiritide on urine output and hemodynamics following cardiopulmonary bypass in infants. Safety and pharmacokinetic data will also be obtained.
- Trial name
- NCT00281671
- Testing
- nesiritide
- Where
- United States
- Volunteers
- 9 people
- Sponsor
- Boston Children's Hospital
Sickle Cell Disease
SIKAMIC (SIklos on Kidney Function and AlbuMInuria Clinical Trial)
The purpose of this phase IIb, international, multicentre, double-blind, randomised, placebo-controlled study is to determine the effect of hydroxycarbamide on albuminuria after 6 months of treatment in SCD adult patients.
- Trial name
- SIKAMIC (NCT03806452)
- Testing
- Hydroxycarbamide
- Where
- 21 sites in Côte d’Ivoire, France and 4 more
- Volunteers
- 86 people
- Sponsor
- Theravia
Cystic Fibrosis
Effects of Sildenafil on CFTR-dependent Ion Transport Activity
Dehydrated airway surfaces resulting from sodium hyperabsorption and lack of chloride secretion are critical to the pathology that leads to the morbidity and mortality from Cystic Fibrosis (CF) lung disease.
- Trial name
- NCT01132482
- Testing
- Sildenafil
- Where
- United States
- Volunteers
- 19 people
- Sponsor
- National Jewish Health
Atopic Dermatitis, Eczema
Steroid-reducing Effects of Crisaborole
Atopic dermatitis, or eczema, is a chronic skin condition affecting many children. Crisaborole is a non-steroid topical medication which is FDA approved for children aged 2 and older for eczema.
- Trial name
- NCT03832010
- Testing
- Crisaborole, Hydrocortisone Ointment, Triamcinolone ointment
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Dyslipidemias, Hypercholesterolemia
Study of Obicetrapib & Ezetimibe Fixed Dose Combination on Top of Maximum Tolerated Lipid-Modifying Therapies
The study is a placebo-controlled, double-blind, randomized, phase 3 study in participants with heterozygous familial hypercholesterolemia (HeFH) and/or atherosclerotic cardiovascular disease (ASCVD) or multiple ASCVD risk…
- Conditions
- Dyslipidemias, Hypercholesterolemia, Familial Hypercholesterolemia, ASCVD
- Trial name
- TANDEM (NCT06005597)
- Testing
- Combination Therapy, Monotherapy obicetrapib, Monotherapy ezetimibe
- Where
- 45 sites in United States
- Volunteers
- 407 people
- Sponsor
- NewAmsterdam Pharma
Dilated Cardiomyopathy
Safety and Efficacy Study of Stem Cell Transplantation to Treat Dilated Cardiomyopathy
Several studies have documented that transplantation of bone marrow-derived cells (BMC) following acute myocardial infarction is associated with a reduction in infarct scar size and improvements in left ventricular function and…
- Trial name
- NCT00629018
- Testing
- CD34+ autologous stem cell transplantation, Bone Marrow Stimulation, SC therapy
- Where
- Slovenia
- Volunteers
- 110 people
- Sponsor
- University Medical Centre Ljubljana
Hypothalamic Obesity
Energy Balance & Weight Loss in Craniopharyngioma-related or Other Hypothalamic Tumors in Hypothalamic Obesity
The proposed multicenter study will test the effect of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (ExQW, Bydureon®) on clinical outcomes and metabolic parameters in a double-blind…
- Trial name
- ECHO (NCT02664441)
- Testing
- Exenatide
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- Seattle Children's Hospital
Obsessive-Compulsive Disorder, Tic Disorders
Effects of Ondansetron in Obsessive-compulsive and Tic Disorders
This project investigates the use of 4 weeks of 24 mg/day ondansetron as compared to placebo on symptoms and brain functioning in patients with obsessive-compulsive disorder (OCD) and tic disorders (TD).
- Conditions
- Obsessive-Compulsive Disorder, Tic Disorders, Tourette Syndrome
- Trial name
- NCT03239210
- Testing
- Ondansetron
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- NYU Langone Health
Obesity, Sedentary
Effects of Obesity and Physical Inactivity on Pregnancy Outcomes
Regular maternal physical activity leads to the delivery of lighter, leaner infants. Higher birth weights and childhood obesity are both strong predictors for adult obesity, suggesting that the impact of maternal physical…
- Trial name
- NCT02039414
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Washington University School of Medicine
Non-ischemic Dilated Cardiomyopathy
PercutaneOus StEm Cell Injection Delivery Effects On Neomyogenesis in Dilated CardioMyopathy (The POSEIDON-DCM Study)
The technique of transplanting progenitor cells into a region of damaged myocardium, termed cellular cardiomyoplasty1, is a potentially new therapeutic modality designed to replace or repair necrotic, scarred, or dysfunctional…
- Trial name
- PoseidonDCM (NCT01392625)
- Testing
- Autologous hMSCs, Allogeneic hMSCs
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Joshua M Hare
Fetal Hypoxia, Fetal Distress
Trans-abdominal Fetal Pulse Oximetry
The Raydiant Oximetry Sensing System (Lumerah) is a non-invasive fetal pulse oximeter that measures fetal arterial oxygen saturation using safe, non-invasive, transabdominal near-infrared spectroscopy.
- Conditions
- Fetal Hypoxia, Fetal Distress, Fetal Acidemia, Fetal or Neonatal Effect of Complication of Labor
- Trial name
- NCT05147584
- Testing
- Dual fetal oxygen sensors
- Where
- United States
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Raydiant Oximetry, Inc.