Clinical trials: SIDS
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Sudden Infant Death Syndrome (SIDS)
Comparing Children's Book to Brochures for Safe Sleep Education in a Home Visiting Program
This randomized controlled trial compares a specially-designed children's book to standard brochures for safe sleep education and reduction of Sudden Infant Death Syndrome (SIDS) risk in a high-risk population of young…
- Trial name
- NCT02376673
- Testing
- Children's Book, Standard Brochures
- Where
- United States
- Volunteers
- 282 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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
Hunter Syndrome
Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age
This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth…
- Trial name
- NCT02455622
- Testing
- Elaprase for intravenous (IV) infusion
- Where
- 8 sites in United States, Dominican Republic and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Clinical Trial of Growth Hormone in MPS I, II, and VI
The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT00748969
- Testing
- Somatropin (DNA origin)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Sudden Infant Death, Sudden Unexplained Infant Death
Improving Pediatrician Counseling About Infant Safe Sleep Using the Electronic Medical Record
The study team will evaluate the impact of an Infant Sleep Assessment (ISA) tool with motivational interviewing (MI) communication training on clinician-parent communication during 2-month Well Baby Visits (WBV) and parent…
- Trial name
- NCT05503771
- Testing
- ISA-MI, Standard of Care (SOC)
- Where
- United States
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Hunter Syndrome
A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India
The main aim of this study is to learn more about the safety profile of Elaprase in Indian children and adults with hunter syndrome.
- Trial name
- NCT05058391
- Testing
- Elaprase
- Where
- 5 sites in India
- Volunteers
- 5 people
- Sponsor
- Takeda
Vagal Syncope
Validation of a Non Invasive Blood Marker of SIDS and Vagal Disorders
Context The investigators recently demonstrated a highly significant increase in muscarinic receptor density in the myocardium of infants deceased from sudden infant death syndrome (SIDS) compared to those of infants deceased…
- Trial name
- NCT01358461
- Testing
- Blood sampling, ECG-Holter 24 h tape and sino-carotid stimulation test
- Where
- France
- Volunteers
- 240 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Stillbirths, Sudden Infant Death
Magnetic Resonance Imaging Autopsy Study
The aim of this project is to establish whether magnetic resonance (MR) imaging can provide a minimally invasive approach for post-mortem assessment of the fetus, infant and child, with similar detection rates for anomalies and…
- Trial name
- MARIAS (NCT01417962)
- Where
- 2 sites in United Kingdom
- Volunteers
- 400 people
- Sponsor
- Thayyil, Sudhin
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
Breastfeeding, SUID
Social Media and Risk Reduction Teaching-Enhanced Reach
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) through direct education of mothers…
- Conditions
- Breastfeeding, SUID, SIDS
- Trial name
- SMARTER (NCT04387552)
- Testing
- Prenatal Safe Sleep Mobile Health (mHealth) Messaging, Prenatal Breastfeeding Mobile Health (mHealth) Messaging, Postnatal Safe Sleep Mobile Health (mHealth) Messaging
- Where
- United States
- Volunteers
- 2,093 people
- Sponsor
- Boston University
Sudden Infant Death Syndrome, Apnea
Incidence and Severity of Cardiorespiratory Events in Infants at Increased Epidemiological Risk for Sudden Infant Death Syndrome (SIDS)
OBJECTIVES: I. Assess and compare the incidence and severity of cardiorespiratory events documented by home monitoring in infants at increased epidemiological risk for sudden infant death syndrome (SIDS). II.
- Trial name
- NCT00004392
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Mucopolysaccharidoses
Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network
Mucopolysaccharidosis (MPS) syndromes are disorders characterized by enzyme deficiencies, and they have been linked to heart health complications.
- Trial name
- NCT01586871
- Where
- 2 sites in United States
- Volunteers
- 1,147 people
- Sponsor
- University of Minnesota
Hunter Syndrome
An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment
This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV)…
- Trial name
- NCT01506141
- Testing
- Idursulfase-IT, Elaprase
- Where
- 9 sites in United States, Canada and 1 more
- Volunteers
- 15 people
- Sponsor
- Takeda
Mucopolysaccharidosis II
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
This is a six-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity…
- Trial name
- NCT04007536
- Testing
- No Intervention
- Where
- 6 sites in United States, Netherlands and 1 more
- Volunteers
- 18 people
- Sponsor
- Denali Therapeutics Inc.
Adrenoleukodystrophy, Batten Disease
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
The primary objective of the study is to determine the safety and feasibility of intrathecal administration of DUOC-01 in patients who are undergoing standard treatment with umbilical cord blood transplant (UCBT) for inborn…
- Conditions
- Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell
- Trial name
- DUOC-01 (NCT02254863)
- Testing
- DUOC-01
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Joanne Kurtzberg, MD
Obstruction, Airway, Sudden; Death, Infant
The Inclined Position in Case of Respiratory Discomfort in the One Year Less Infant : Study on the Profits and the Risks in the Home
The inclined position at 30 ° in case of transient respiratory discomfort is recommended since the consensus conference of September 2000. This recommendation is based on a low level of evidence (grade C).
- Trial name
- PROCLIVE (NCT03482804)
- Testing
- Questionnaires
- Where
- 2 sites in France
- Volunteers
- 90 people
- Sponsor
- Hospices Civils de Lyon
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
Hunter Syndrome
Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701)
The purpose of this non-interventional, observational study which is conducted in Mexico is to evaluate the safety profile of elaprase (idulsurfase) in participants with hunter syndrome (mucopolysaccharydosis II) being treated…
- Trial name
- NCT03582449
- Where
- 6 sites in Mexico
- Volunteers
- 5 people
- Sponsor
- Shire