Clinical trials: Congenital Anomaly
Vascular Anomaly
Efficacy and Safety of Sirolimus to Vascular Anomalies
To evaluate the safety and efficacy of Sirolimus in complicated vascular anomalies in Chinese children
- Trial name
- NCT03583307
- Testing
- Sirolimus
- Where
- China
- Volunteers
- 126 people
- Sponsor
- West China Hospital
Infant, Premature, Congenital Heart Disease
Splanchnic and Renal Tissue Oxygenation During Enteral Feedings in Neonates With Patent Ductus Arteriosus
Patent ductus arteriosus (PDA) is a common problem in the neonatal intensive care unit and can be secondary to prematurity or congenital heart disease (CHD).
- Conditions
- Infant, Premature, Congenital Heart Disease, Patent Ductus Arteriosus, Necrotizing Enterocolitis
- Trial name
- NCT03551600
- Testing
- Near-infrared spectroscopy (NIRS)
- Where
- 3 sites in United States
- Volunteers
- 64 people
- Sponsor
- University of Utah
Overweight and Obesity, Men
Evaluation of a Supplement for Weight Management in Obese and Overweight Individuals
The purpose of this trial is to examine the effects of a novel, orally consumed dietary supplement (composed of naturally occurring components) on weight management and metabolic rate in individuals who are overweight or obese…
- Conditions
- Overweight and Obesity, Men, Women
- Trial name
- NCT03812211
- Testing
- Active Supplement
- Where
- Canada
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- McMaster University
Cardiomyopathy, Hypertrophic, Familial
Signs and Symptoms of Genetic Abnormalities Linked to Inherited Heart Disease
Genetically inherited heart diseases (familial cardiomyopathies) are conditions affecting the heart passed on to family members through abnormalities in genetic information.
- Trial name
- NCT00001881
- Where
- United States
- Volunteers
- 9,999 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Comparing Three Different Combination Chemotherapy Regimens in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia
This randomized phase II trial is comparing three different combination chemotherapy regimens to see how well they work in treating patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT00634244
- Testing
- alvocidib, mitoxantrone hydrochloride, carboplatin
- Where
- 20 sites in United States, Israel
- Volunteers
- 92 people
- Sponsor
- National Cancer Institute (NCI)
Post Term Pregnancy, Premature Rupture of Membrane
Dilapan-S®: A Multicenter US E-registry
Objective of this registry is to collect a representative set of real world data on the use of Dilapan-S® for pre-induction cervical ripening in daily clinical practice.
- Conditions
- Post Term Pregnancy, Premature Rupture of Membrane, Fetal Growth Retardation, Oligohydramnios
- Trial name
- NCT04451109
- Testing
- Dilapan-S
- Where
- United States
- Volunteers
- 1,200 people
- Sponsor
- Medicem Technology s.r.o.
Spinal and Bulbar Muscular Atrophy
Safety, Tolerability, and Efficacy of BVS857 in Patients With Spinal and Bulbar Muscular Atrophy
The purpose of this study was to determine if BVS857 is safe, tolerable and increases thigh muscle thickness in patients with spinal bulbar and muscular atrophy (SBMA).
- Trial name
- NCT02024932
- Testing
- BVS857
- Where
- 6 sites in United States, Denmark and 2 more
- Volunteers
- 37 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
Improving Outcome Measures For Adult CF ACT Trials
This is a randomised controlled cross-over trial involving adult cystic fibrosis (CF) patients from the Royal Brompton Hospital, London investigating outcome measures used in airway clearance trials.
- Trial name
- NCT02721498
- Testing
- Airway clearance session using the Active Cycle of Breathing Techniques (ACBT), Rest period
- Where
- United Kingdom
- Volunteers
- 68 people
- Sponsor
- Royal Brompton & Harefield NHS Foundation Trust
Ventricular Septal Defect, Closure; Interventricular Septum
The Effect of Transcatheter Ventricular Septal Defect Closure on Heart Rate Variability Parameters
In this study; we evaluated the heart rate variability parameters of pediatric patients whose VSDs were closed with the transcatheter method before and after, and compared with the control group.
- Conditions
- Ventricular Septal Defect, Closure; Interventricular Septum, Autonomic Nervous System Imbalance
- Trial name
- NCT04859036
- Testing
- 24 hours ambulatory Holter monitoring
- Where
- Turkey (Türkiye)
- Volunteers
- 37 people, healthy volunteers welcome
- Sponsor
- Kayseri City Hospital
Intraventricular Hemorrhage, Spina Bifida
Micro Ribonucleic Acid (miRNA) Markers of Hydrocephalus in Intraventricular Hemorrhage (IVH)
A collection of biological samples (cerebrospinal fluid [CSF] and blood) from patients under 6 years of age who are diagnosed with intraventricular hemorrhage or spina bifida.
- Trial name
- NCT02386228
- Testing
- collection of CSF and blood
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- St. Joseph's Hospital and Medical Center, Phoenix
Arnold-Chiari Malformation, Type 1, Chiari Malformation Type I
Posterior Fossa Decompression With or Without Duraplasty for Chiari Type I Malformation With Syringomyelia
The purpose of this study is to determine whether a posterior fossa decompression or a posterior fossa decompression with duraplasty results in better patient outcomes with fewer complications and improved quality of life in…
- Conditions
- Arnold-Chiari Malformation, Type 1, Chiari Malformation Type I, Type I Arnold-Chiari Malformation, Syringomyelia
- Trial name
- NCT02669836
- Testing
- Posterior fossa decompression, Dural Augmentation
- Where
- United States
- Volunteers
- 162 people
- Sponsor
- Washington University School of Medicine
Apparent Mineralocorticoid Excess
An Investigation Into the Effect of Liquorice Ingestion on the Salivary Cortisol to Cortisone Molar Ratio
Aldosterone, the major mineralocorticoid hormone and cortisol, the major glucocorticoid hormone are produced in the adrenal gland.
- Trial name
- NCT02939144
- Testing
- Liquorice
- Where
- United Kingdom
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- The Royal Wolverhampton Hospitals NHS Trust
Atopic Dermatitis
A Single-ascending Dose (Part A) and Repeat-dose (Part B) Study to Investigate the Safety, Pharmacokinetics and Efficacy (Part B Only) of UCB1381 in Healthy Study Participants (Part A) and in Study Participants With Moderate to Severe Atopic Dermatitis (Part B)
The purpose of the study is to investigate the safety and tolerability of single-ascending doses of UCB1381 (intravenous and subcutaneous) in healthy study participants and after repeat intravenous dosing in study participants…
- Trial name
- NCT05277571
- Testing
- UCB1381
- Where
- 16 sites in United States
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- UCB Biopharma SRL
Rett Syndrome, RTT
Efficacy and Safety of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
To evaluate the efficacy of cannabidiol oral solution (GWP42003-P, CBD-OS) in reducing symptom severity when compared with placebo, in participants with Rett syndrome.
- Trial name
- ARCH (NCT03848832)
- Testing
- GWP42003-P
- Where
- 26 sites in United States, Italy and 2 more
- Volunteers
- 29 people
- Sponsor
- Jazz Pharmaceuticals
Sickle Cell Disease
Assessment of Algorithm-Based Hydroxyurea Dosing on Fetal Hemoglobin Response, Acute Complications, and Organ Function in People With Sickle Cell Disease
Background: Sickle cell disease (SCD) is a blood disease. The drug hydroxyurea (HU) is approved to prevent pain crises in people with SCD. Researchers want to see how higher doses of HU affect the blood.
- Trial name
- NCT02225132
- Testing
- Hydroxyurea
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Molar Incisor Hypomineralization, Anesthesia, Local
Effect of Silver Diamine Fluoride on Local Anesthesia in Hypomineralized Molars
The purpose of this randomized, controlled clinical study is to evaluate if application of 38% SDF increases the efficacy of local anesthesia during operative dentistry on permanent molars affected by molar-incisor hypoplasia…
- Trial name
- NCT05964387
- Testing
- SDF
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Michigan
Friedreich Ataxia
A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia
The primary objective of the study is to assess the pharmacokinetics (PK) and safety of vatiquinone administered in participants with Friedreich ataxia (FA) younger than 7 years.
- Trial name
- NCT05485987
- Testing
- Vatiquinone
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- PTC Therapeutics
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital…
- Trial name
- ZEBRHA 2 (NCT07416604)
- Testing
- NXT007, Emicizumab
- Where
- 59 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Hoffmann-La Roche
Muscular Dystrophy, Duchenne and Becker Types
Effectiveness of a Multimodal Physiotherapy Program With Virtual Reality Glasses in Duchenne and Becker.
Duchenne's Muscular Dystrophy and Becker Dystrophy, hereafter DMD and BMD, is a serious and progressive disease that affects 1 in 3,500-6,000 males born alive.
- Trial name
- RVDuchenne (NCT03879304)
- Testing
- multimodal physiotherapy program with RV, traditional physiotherapy program
- Where
- Spain
- Volunteers
- 12 people
- Sponsor
- University of Malaga
Pseudoxanthoma Elasticum
A Study of DS-1211b in Individuals With PseudoXanthoma Elasticum
This study was designed to evaluate the safety, tolerability, pharmacodynamics (PD) of DS-1211b, and pharmacokinetics (PK) in individuals with Pseudoxanthoma elasticum (PXE).
- Trial name
- NCT05569252
- Testing
- DS-1211b
- Where
- 7 sites in United States, Netherlands
- Volunteers
- 65 people
- Sponsor
- Daiichi Sankyo