Clinical trials: Sudden Infant Death
MPS II, Hunter Syndrome (MPS II)
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
The objective of this study is to assess the long-term safety and efficacy of Hunterase for approximately 1 year in subjects who completed the end of study (EOS) visit (Visit 54) tests in the Phase 3 Hunterase study and patients…
- Conditions
- MPS II, Hunter Syndrome (MPS II), Hunter Syndrome, Hunterase
- Trial name
- NCT07344376
- Testing
- Hunterase
- Where
- South Korea
- Volunteers
- 30 people
- Sponsor
- GC Biopharma Corp
MLD, Krabbe Disease
Longitudinal Study of Neurodegenerative Disorders
The purpose of this study is to understand the course of rare genetic disorders that affect the brain. This data is being analyzed to gain a better understanding of the progression of the rare neurodegenerative disorders and the…
- Conditions
- MLD, Krabbe Disease, ALD, MPS I
- Trial name
- NCT03333200
- Testing
- Palliative Care, Hematopoetic Stem Cell Transplantation
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- University of Pittsburgh
Hunter Syndrome
An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients
The objective of this study is to evaluate the effect of anti-idursulfase antibodies on idursulfase safety (measured by infusion related adverse events) between patients who develop anti-idursulfase antibodies and patients who do…
- Trial name
- NCT00882921
- Testing
- Idursulfase
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 26 people
- Sponsor
- Shire
Sudden Infant Death
Investigation of the Genetic Diseases in Infants With Unknown Cause of Death
Sudden infant death syndrome (SIDS) is a disease of an infant under one year of age, whose sudden death occurred unexpectedly, which the cause of death cannot be determined despite macro-autopsy, and toxicological, pathological…
- Trial name
- SIDS (NCT06060249)
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people
- Sponsor
- Ankara University
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Safe Sleep Education, SUID
Social Media and Risk-reduction Training for Preterm Infant Care Practices
SMART Preemie is a cluster randomized trial that will investigate the effectiveness of NICU-based and post-discharge interventions to improve adherence to safe sleep practices among mothers of preterm infants.
- Conditions
- Safe Sleep Education, SUID, Sudden Infant Death Syndrome (SIDS)
- Trial name
- SMART Preemie (NCT07420686)
- Testing
- Safe Sleep NICU Quality Improvement Campaign, Shared Reading NICU Quality Improvement Campaign, Safe Sleep TodaysNICUBaby videos and messages
- Where
- United States
- Volunteers
- 1,600 people, healthy volunteers welcome
- Sponsor
- University of Massachusetts, Worcester
Infant Death, Sudden Infant Death
Get Social Media and Risk-Reduction Training
This study examines the implementation of the TodaysBaby™ mobile safe sleep intervention program in US hospitals. The main aims of this study are to examine: 1) differences in program penetration and the equity of penetration…
- Trial name
- GETSMART (NCT06618586)
- Testing
- TodaysBaby
- Where
- United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- University of Virginia
Stillbirth, Infant Mortality
Impact of Smoke-free Legislation on Early-life Mortality and Low Birth Weight in England
The purpose of this study is to investigate whether there has been a change in low birth weight and perinatal and infant mortality following the July 2007 introduction of a ban on smoking in public places and workplaces in…
- Conditions
- Stillbirth, Infant Mortality, Sudden Infant Death, Infant, Low Birth Weight
- Trial name
- NCT02039583
- Testing
- Smoke-free legislation
- Where
- United Kingdom
- Volunteers
- 10,291,118 people, healthy volunteers welcome
- Sponsor
- University of Edinburgh
Mucopolysaccharidosis (MPS)
Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)
Mucopolysaccharidosis (MPS) type II (MPS II; Hunter syndrome) is a rare, X-linked disease caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S) and occurs almost exclusively in boys, with an incidence of…
- Trial name
- NCT02095015
- Where
- 14 sites in United States, Canada and 5 more
- Volunteers
- 159 people
- Sponsor
- Shire
Breastfeeding, Infant Death
Support Via Online Social Networks to Promote Safe Infant Care Practices
This study aims to improve adherence to American Academy of Pediatrics safe sleep (SS) recommendations and improve rates of initiation and duration of partial and exclusive breastfeeding (BF); and reduce Black/White disparities…
- Conditions
- Breastfeeding, Infant Death, Sudden Infant Death Syndrome Without Mention of Autopsy
- Trial name
- SUPERSONIC (NCT05767658)
- Testing
- Safe Sleep, Breastfeeding, Early Brain Development and Parent-Child Relationships
- Where
- United States
- Volunteers
- 3,000 people
- Sponsor
- University of Virginia
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
MPS II, Mucopolysaccharidosis II
MPS II Immunophenotyping
The purpose of this study is to investigate how participant's body's immune system responds to idursulfase, an enzyme replacement therapy (ERT) and find out which types of immune cells are involved in causing untoward responses…
- Conditions
- MPS II, Mucopolysaccharidosis II, Hunter Syndrome
- Trial name
- NCT04976231
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Duke University
Lactation
Influence of Unrestricted Introduction of Pacifiers on Breastfeeding Duration
We will enroll 1010 mother-infant dyads in a randomized trial exploring the effect of distribution of the pacifier during the first days of life on breastfeeding prevalence and duration. .
- Trial name
- NCT03981133
- Testing
- offer a pacifier, no pacifier
- Where
- 6 sites in Argentina
- Volunteers
- 1,010 people
- Sponsor
- Fundación para el Estudio y Prevención de la Muerte Infantil y Perinatal
Sudden Unexplained Infant Death, Child Rearing
Comparing Children's Books to Brochures for Safe Sleep and Infant Reading Education During Prenatal Care
The aim of this randomized trial is to compare a specially designed children's book to brochures for safe sleep education via clinical providers at a third trimester prenatal obstetric visit.
- Conditions
- Sudden Unexplained Infant Death, Child Rearing, Literacy
- Trial name
- NCT04031235
- Testing
- Sleep Baby, Safe and Snug, Read Baby, Every Day
- Where
- United States
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Mucopolysaccharidosis II
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
The purpose of this study is to evaluate the safety and efficacy of GC1111 (recombinant human iduronate-w-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) patients
- Trial name
- NCT01301898
- Testing
- GC1111_0.5mg/kg, GC1111_1.0mg/kg, Elaprase_0.5mg/kg
- Where
- South Korea
- Volunteers
- 31 people
- Sponsor
- Green Cross Corporation
Mucopolysaccharidosis Type II (MPS II)
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system.
- Trial name
- NCT03566043
- Testing
- RGX-121
- Where
- 5 sites in United States, Brazil
- Volunteers
- 48 people
- Sponsor
- REGENXBIO Inc.
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
- Trial name
- NCT05208281
- Testing
- GNR-055 1.0-2.0-3.0 mg/kg, GNR-055 2.0 mg/kg, GNR-055 3.0 mg/kg
- Where
- 5 sites in Russia
- Volunteers
- 32 people
- Sponsor
- AO GENERIUM
Sudden Infant Death Syndrome
Parental Knowledge and Beliefs About Infant Sleep Position
In the United States, Sudden Infant Death Syndrome (SIDS)is the leading cause of death in infants between the ages of 1 and 12 months.
- Trial name
- NCT00005567
- Testing
- Parental knowledge and beliefs about infant sleep position
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Center for Research Resources (NCRR)
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT
The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant…
- Conditions
- Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias
- Trial name
- HSCT+RIC (NCT01962415)
- Testing
- Hydroxyurea, Alemtuzumab, Fludarabine
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Paul Szabolcs
Sudden Unexplained Infant Death, Apnea of Newborn
Advanced Wireless Sensors for Neonatal Care in the Delivery Room
The goals of this observational study is to assess whether a new advanced wireless skin sensor vital sign monitoring system can effectively monitor the vital signs of healthy newborn infants (≥ 35 weeks gestational age).
- Conditions
- Sudden Unexplained Infant Death, Apnea of Newborn, Newborn Morbidity, Newborn Asphyxia
- Trial name
- AWARD (NCT06693817)
- Testing
- Wireless skin sensors vital sign monitoring system, Wired vital sign monitoring system
- Where
- 5 sites in Brazil, Canada and 2 more
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Guilherme Sant'Anna, MD