Clinical trials: Birth Defect
Pompe Disease
Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease
A Phase 1/2, open-label, multicenter, multiple dose escalation study of BMN 701 administered by intravenous infusion every 2 weeks over a 24-week treatment period to patients with late-onset Pompe disease.
- Trial name
- NCT01230801
- Testing
- BMN 701
- Where
- 10 sites in United States, Australia and 3 more
- Volunteers
- 22 people
- Sponsor
- BioMarin Pharmaceutical
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease.
- Trial name
- NCT01794000
- Testing
- Prasugrel
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 341 people
- Sponsor
- Eli Lilly and Company
Respiratory Syncytial Virus Infection, Premature Birth
Palivizumab for Prevention of Severe Respiratory Syncytial Virus Infection in Russian Children
100 Russian children of 2 years of age and less in high-risk populations (preterm, and/or with heart and lung problems) will receive palivizumab (Synagis) 15 mg/kg intramuscularly as prophylaxis to severe respiratory syncytial…
- Conditions
- Respiratory Syncytial Virus Infection, Premature Birth, Bronchopulmonary Dysplasia, Congenital Heart Disease
- Trial name
- NCT01006629
- Testing
- palivizumab
- Where
- 19 sites in Russia
- Volunteers
- 100 people
- Sponsor
- Abbott
Cystic Fibrosis
To Assess the Safety, Tolerability and Pharmacokinetics of AZD5634 Following Inhaled and Intravenous (IV)Dose Administration
This is a Phase 1, first-in-human (FIH) single ascending dose study being conducted to better understand the safety, tolerability and pharmacokinetics of AZD5634 in healthy subjects
- Trial name
- NCT02679729
- Testing
- AZD5634 for inhalation, AZD5634 for infusion
- Where
- 2 sites in United States
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
Neonatal Abstinence Syndrome
A Trial to Determine Weight Gain Benefits of Caloric Supplementation for NAS Infants
A randomized clinical study in NAS infants managed via the Eat, Sleep, Console (ESC) approach comparing early weight loss on a standard-caloric density versus high-caloric density feeding regimen.
- Trial name
- NCT04419857
- Testing
- High-calorie formula
- Where
- United States
- Volunteers
- 39 people
- Sponsor
- Yale University
Lennox-Gastaut Syndrome
Transcranial Direct Current Stimulation, Treatment of Childhood Drug-Resistant Lennox-Gastaut Syndrome, A Pilot Study
Background: Lennox-Gastaut syndrome (LGS) is a severe childhood epileptic syndrome with high pharmacoresistance. The treatment outcomes are still unsatisfied.
- Trial name
- NCT02731300
- Testing
- tDCS
- Volunteers
- 22 people
- Sponsor
- Khon Kaen University
Cystic Fibrosis
Effect of Sulforaphane in Broccoli Sprouts on Nrf2 Activation
The purpose of this study to investigate the effect of sulforaphane from macerated broccoli sprouts in humans and to evaluate less invasive methods of assessing potential anti-inflammatory drugs in CF.
- Trial name
- NCT01315665
- Testing
- Broccoli sprouts
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Sickle Cell Disease
Vaporized Cannabis for Chronic Pain Associated With Sickle Cell Disease
Our primary objective is to assess whether inhaling vaporized cannabis ameliorates chronic pain in patients with sickle cell disease (SCD).
- Trial name
- Cannabis-SCD (NCT01771731)
- Testing
- Cannabis
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of California, San Francisco
Coarctation of the Aorta
Statins and Endothelial Function in Patients With Coarctation of the Aorta
Coarctation of the aorta (CoA) accounts for approximately 8% of all congenital heart problems. Patients with CoA are well known to have an increased rates of early heart disease, high blood pressure, heart failure and stroke.
- Trial name
- NCT00767572
- Testing
- atorvastatin, Sugar pill
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of California, San Francisco
Thyroid Eye Disease
A Safety, Tolerability, and Efficacy Study of Veligrotug (VRDN 001) in Participants With Thyroid Eye Disease (TED)
The investigational drug, veligrotug (VRDN-001), is a monoclonal antibody that inhibits the activity of a cell surface receptor called insulin-like growth factor-1 receptor (IGF-1R).
- Trial name
- THRIVE (NCT05176639)
- Testing
- Veligrotug
- Where
- 53 sites in United States, Australia and 5 more
- Volunteers
- 113 people
- Sponsor
- Viridian Therapeutics, Inc.
Cystic Fibrosis
Using Rheological Methods to Characterize Cystic Fibrosis (CF) Sputum and the Effects of Mucoactive Agents
The purpose of this study is to characterize the rheological properties of cystic fibrosis (CF) and healthy sputum and to examine the effects of mucoactive agents on the rheology of CF and healthy sputum.
- Trial name
- GIST (NCT00758771)
- Where
- United States
- Volunteers
- 38 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Bronchopulmonary Dysplasia
A Safety Study of IV Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for BPD
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at <27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).
- Trial name
- NCT03857841
- Testing
- UNEX-42, Phosphate-buffered saline
- Where
- 6 sites in United States
- Volunteers
- 3 people
- Sponsor
- United Therapeutics
Prader-Willi Syndrome, Hyperphagia
Effects of Livoletide (AZP-531) on Food-related Behaviors in Patients With Prader-Willi Syndrome
This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).
- Trial name
- ZEPHYR (NCT03790865)
- Testing
- Livoletide
- Where
- 37 sites in United States, Australia and 6 more
- Volunteers
- 158 people
- Sponsor
- Millendo Therapeutics SAS
Primary Hypercholesterolemia and Homozygous Familial Hypercholesterolemia (HoFH)
Vytorin Reexamination Study (0653A-174)
This survey is conducted for preparing application material for re-examination under the Pharmaceutical Affairs Laws and its Enforcement Regulation, its aim is to reconfirm the clinical usefulness of VYTORIN through collecting…
- Trial name
- NCT01070966
- Volunteers
- 2,089 people
- Sponsor
- Organon and Co
Congenital Heart Defect
The Effects of Inhaled Nitric Oxide After Fontan Operation
Pleural effusions continue to be a post-operative complication seen after Fontan operation. This study will examine the use inhaled nitric oxide (iNO) given for 54 hours after Fontan operation to improve fluid balance.
- Trial name
- NCT00945529
- Testing
- inhaled nitric oxide (iNO)
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Ellen Spurrier
Chronic Myeloproliferative Disorders, Leukemia
Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and busulfan, before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- Bu Flu TBI (NCT00245037)
- Testing
- therapeutic allogeneic lymphocytes, busulfan, cyclosporine
- Where
- United States
- Volunteers
- 147 people
- Sponsor
- OHSU Knight Cancer Institute
Cystic Fibrosis
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have an Ivacaftor-Responsive CFTR Mutation
The purpose of this study was to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and metabolites in participants with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an…
- Trial name
- NCT02725567
- Testing
- IVA
- Where
- 23 sites in United States, Australia and 3 more
- Volunteers
- 57 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gout, Low Medication Adherence
SToRytelling to Improve DiseasE Outcomes in GOut: The STRIDE-GO2 Study
The objective is to test the efficacy of a patient-centered, culturally relevant narrative intervention, or "storytelling," based on the solid conceptual foundation of the narrative communication theory and the constructs of the…
- Conditions
- Gout, Low Medication Adherence, Health Related Quality of Life
- Trial name
- STRIDE-GO2 (NCT02741700)
- Testing
- Gout Storytelling Video Intervention
- Where
- 3 sites in United States
- Volunteers
- 306 people
- Sponsor
- VA Office of Research and Development
Mucopolysaccharidosis IVA, Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01697319
- Testing
- BMN 110
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Hereditary Spastic Paraplegia, Adrenomyeloneuropathy
Repetitive Transcranial Magnetic Stimulation as Therapy in Hereditary Spastic Paraplegia and Adrenomyeloneuropathy
Hereditary spastic paraplegia (HSP) is the group of inherited disorders, characterized by progressive gait disturbance. There is no established therapy. Adrenoleukodystrophy (AMN) is an x-linked hereditary disease.
- Trial name
- NCT03627416
- Testing
- rTMS
- Where
- Poland
- Volunteers
- 15 people
- Sponsor
- Jakub Antczak