Clinical trials: Delivery Systems
Duchenne Muscular Dystrophy
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study.
- Trial name
- EMBARK (NCT05096221)
- Testing
- delandistrogene moxeparvovec
- Where
- 42 sites in United States, Belgium and 7 more
- Volunteers
- 126 people
- Sponsor
- Sarepta Therapeutics, Inc.
Concussion, Brain, Concussion, Mild
Mobile Subthreshold Exercise Program for Concussion--R01
Approximately 1.9 million youth sustain a concussion each year, and up to 30% experience persistent post-concussive symptoms (PPCS) such as headache, dizziness, and difficulty focusing that continue for weeks or months.
- Conditions
- Concussion, Brain, Concussion, Mild, Brain Injuries, Traumatic, Persistent Post Traumatic Headache
- Trial name
- MSTEP-R01 (NCT04688255)
- Testing
- MSTEP, Stretching
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Seattle Children's Hospital
Induction of Labour
REgistry of MisOprostol 200 µg Vaginal dElivery System
The design of the study is post-marketing, observational, multi-centre and open-label. The study does not provide treatment; only patients to whom misoprostol 200 µg vaginal delivery system (VDS) is prescribed may be included.
- Trial name
- REMOVE (NCT02242214)
- Testing
- Misoprostol
- Where
- Netherlands
- Volunteers
- 106 people
- Sponsor
- Ferring Pharmaceuticals
Pancreatic Pseudocyst Infection, Pancreatic Pseudocyst
Hot AXIOS System Japan Post Market Survey
To detect information of Adverse Events and Device Malfunctions under real world medical condition in Japan.
- Conditions
- Pancreatic Pseudocyst Infection, Pancreatic Pseudocyst, Walled Off Necrosis Infection, Walled Off Necrosis
- Trial name
- NCT03797209
- Testing
- EUS-guided fistulization AXIOS
- Where
- 40 sites in Japan
- Volunteers
- 120 people
- Sponsor
- Boston Scientific Japan K.K.
Zinc Deficiency, Diarrhea
Water-based Zinc Intervention Trial in Zinc Deficient Children
The overall aim of this trial is to determine whether the use of the household-based water treatment device Life Straw Family (LSF) can be an effective strategy to improve zinc status and health status of vulnerable populations…
- Trial name
- NCT01472211
- Testing
- zinc enriching filter (LifeStrawFamily), disinfection tablets
- Where
- Benin
- Volunteers
- 317 people, healthy volunteers welcome
- Sponsor
- Swiss Federal Institute of Technology
Premature Birth, NEC
Prenatal Administration of Spermine Promotes Maturation of Premature Fetal Gut Epithelial Tight Junction: Experimental Study on Fetal Rabbit
Infections, particularly on the gastrointestinal tract, has been known to be one of the leading causes of death in preterm infants. This is due to the immaturity of the intestinal epithelial cells.
- Trial name
- NCT04004091
- Testing
- Spermine
- Where
- Indonesia
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- dr. Riana Pauline Tamba, SpB(K)BA
Weight, Birth, Premature Birth
Effects of Olfactory and Gustatory Stimulus on the Nutrition of Premature Babies
The objective of this clinical trial is to test the effects of olfactory and taste stimulation with milk or water in premature newborns with a gestational age between 28 and 32 weeks.
- Trial name
- NCT06305052
- Testing
- Milk, Water
- Where
- Brazil
- Volunteers
- 63 people, healthy volunteers welcome
- Sponsor
- Professor Fernando Figueira Integral Medicine Institute
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
COVID-19, Maternal Vaccine Exposure
Priming of the NEonatal Immune System by Transfer of Maternal Immunity
Newborn babies and infants are susceptible to infections as their immune system is still immature. Maternal immune factors for example antibodies and immune cells mitigate this vulnerability.
- Conditions
- COVID-19, Maternal Vaccine Exposure, Respiratory Infections in Children
- Trial name
- NEST (NCT05429047)
- Testing
- Tozinameran, COVID-19, No SARS-CoV-2 exposure
- Where
- Germany
- Volunteers
- 560 people, healthy volunteers welcome
- Sponsor
- Hannover Medical School
Feeding; Difficult, Newborn, Growth Failure
Prophylactic Probiotics to Extremely Low Birth Weight Prematures
Mortality and incidence of severe complications is still high among extremely premature infants. Common causes of severe complications in this population are poor nutrition, necrotizing enterocolitis, and severe infections.
- Conditions
- Feeding; Difficult, Newborn, Growth Failure, Necrotizing Enterocolitis, Sepsis
- Trial name
- PROPEL (NCT01603368)
- Testing
- Lactobacillus reuteri
- Where
- 2 sites in Sweden
- Volunteers
- 134 people, healthy volunteers welcome
- Sponsor
- Ostergotland County Council, Sweden
Type 1 Diabetes Mellitus
Assessment of Quality of Life and Satisfaction With Medical Care of Pediatric Type 1 Diabetes Mellitus Patients
The purpose of this study is to evaluate the change score for the PedsQL short form Diabetes and Core Modules and the Patient Assessment of Chronic Illness Care measured at baseline (before changing care delivery models) and 12…
- Trial name
- NCT00450489
- Where
- United States
- Sponsor
- Children's Mercy Hospital Kansas City
Preterm, Extreme Prematurity
Effectiveness of Home Based Early Intervention of Extremely Premature Infant by Parent
This study is to prove the effectiveness of home based early intervention of extremely premature infant by Parent.
- Conditions
- Preterm, Extreme Prematurity, Early Intervention, Developmental Delay
- Trial name
- NCT05334550
- Testing
- Home Based Early Intervention by Parent
- Volunteers
- 110 people
- Sponsor
- Samsung Medical Center
Autism Spectrum Disorder
Improving the Part C Early Intervention Service Delivery System for Children With ASD
Despite strong consensus that early, specialized intervention for children with autism spectrum disorder (ASD) can have a dramatic impact on outcomes, the public health system's capacity to provide such services is severely…
- Trial name
- NCT05114538
- Testing
- Reciprocal Imitation Training
- Where
- 4 sites in United States
- Volunteers
- 440 people
- Sponsor
- Rush University Medical Center
Cleft Lip, Cleft Palate
Systematic Pediatric Care for Oral Clefts - South America
Cleft lip and palate are a significant component of morbid human birth defects in the developing world. This study measures the impact of having a child born with a cleft lip on subsequent maternal/infant family health, and…
- Conditions
- Cleft Lip, Cleft Palate, Congenital Defects
- Trial name
- NCT00097149
- Testing
- Systematic pediatric care
- Where
- Brazil
- Volunteers
- 696 people
- Sponsor
- NICHD Global Network for Women's and Children's Health
Immunization Reminder System
Accountable Care Organizations/ Public Health Collaborative
This study focuses on implementing and evaluating an evidence-based collaborative method of increasing immunization rates among preschool children, adolescents and adults within an Accountable Care Organization-Public Health…
- Trial name
- ACO/PH (NCT02133391)
- Testing
- Centralized Reminder/Recall (R/R), Practice-based Reminder/Recall
- Where
- United States
- Volunteers
- 78,354 people
- Sponsor
- University of Colorado, Denver
Premature Baby, Pulmonary Hypertension of Newborn
Measuring Heart Health in Both Term, Preterm and Unwell Newborn Babies With an Advanced Ultrasound Method: Speckle Tracking Echocardiography
This study aims to improve how neonatologists check the heart function of newborn babies, especially those who are sick. While standard heart ultrasound scans are useful, a more advanced and sensitive technique called 2D speckle…
- Conditions
- Premature Baby, Pulmonary Hypertension of Newborn, Hypoxic Ischaemic Encephalopathy (HIE), Congenital Diaphragmatic Hernia
- Trial name
- NCT07364682
- Where
- United Kingdom
- Volunteers
- 190 people, healthy volunteers welcome
- Sponsor
- Birmingham Women's NHS Foundation Trust
Dysphagia
The Effects of Cold Liquids on the Swallowing Mechanism in Preterm Infants
The purpose of this study is to see if cold liquids improve the swallowing mechanisms in premature infants with swallowing difficulties (dysphagia).
- Trial name
- NCT01863264
- Testing
- Cold Thin Liquid Barium
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- NYU Langone Health
Necrotizing Enterocolitis
Role of Probiotics for Prevention of NEC in Preterm VLBW Infants
This prospective randomized double-blind control trial is carrying out in the neonatal unit of Sylhet MAG Osmani Medical College Hospital, Sylhet between July 2012 to December 2015 with the diagnosis of preterm (<33 weeks…
- Trial name
- Probiotics (NCT02650869)
- Testing
- Probiotics, Breast milk
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Sylhet M.A.G.Osmani Medical College
Spinal Muscular Atrophy
A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
The primary objective is to examine the clinical efficacy of multiple doses of nusinersen (ISIS 396443) administered intrathecally to participants with Infantile-Onset Spinal Muscular Atrophy (SMA).
- Trial name
- NCT01839656
- Testing
- nusinersen
- Where
- 4 sites in United States, Canada
- Volunteers
- 21 people
- Sponsor
- Biogen
Asthma
To Compare the Pharmacokinetics of Budesonide Delivered by BDA MDI to Budesonide Delivered by Pulmicort Respules in Children With Asthma Aged 4 to 8 Years.
To compare the pharmacokinetics of budesonide delivered by BDA MDI to budesonide delivered by Pulmicort Respules in children with Asthma aged 4 to 8 years.
- Trial name
- BLANC (NCT04848662)
- Testing
- BDA MDI (PT027) 160/180 μg, Pulmicort Respules 0.5 MG/ML Inhalation Suspension
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Bond Avillion 2 Development LP
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