Clinical trials: Mucopolysaccharidosis Type IV
Acute Mountain Sickness, High Altitude
CO2 Supplement for Treatment of Acute Mountain Sickness
It was reported that up to 76% people who rapidly ascend to the altitude higher than 4500 m without sufficient acclimatization will have AMS which often develops 6-8 hours after reaching at high altitude.
- Conditions
- Acute Mountain Sickness, High Altitude, Inhalation; Gas
- Trial name
- NCT06393998
- Testing
- Breathing with CO2 during daytime and night at high altitude, Sleep and doing exercise test under inhalation of air
- Where
- China
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- State Key Laboratory of Respiratory Disease
Bowel Incontinence
A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage
The purpose of this study is to determine whether the use of the Renew Insert is effective, tolerable and safe in the treatment of accidental bowel leakage due to bowel incontinence.
- Trial name
- REST (NCT01475474)
- Testing
- Renew Insert
- Where
- 3 sites in United States
- Volunteers
- 97 people
- Sponsor
- Renew Medical
Fibrodysplasia Ossificans Progressiva (FOP)
A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)
Primary Objective(s): To describe the demographic characteristics of people living with FOP and a matched cohort of non-FOP people living in the United States (U.S.) To describe the prevalence of clinical characteristics of…
- Trial name
- NCT06064656
- Testing
- Non-Interventional
- Where
- United States
- Volunteers
- 131 people
- Sponsor
- Regeneron Pharmaceuticals
Normal Middle Ears., Otitis Media Ears.
Observation of Gas Exchange in the Middle Ear
Observation of fluid behavior over a perforated eardrum. A comparison of the behavior of the gas in normal vs. different kinds of otitis media ears.
- Trial name
- NCT00794105
- Where
- Israel
- Sponsor
- Shaare Zedek Medical Center
Postoperative Complication, Recovery, Physiological
Assessment of the Impact of Intestinal Gas Emission Quality on the Postoperative Course After Abdominal Surgery
The recovery of transit after surgery is an important parameter in postoperative evaluation. It generally reflects simple postoperative outcomes and allows the patient to return home.
- Trial name
- FLATQUAL (NCT06675097)
- Where
- 2 sites in France
- Volunteers
- 200 people
- Sponsor
- University Hospital, Angers
MPS IV A
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA
This multicenter, open-label study is designed to assess safety, dose-response using pharmacokinetic (PK) and pharmacodynamic (PD) measures, and clinical efficacy of BMN 110 in subjects between 5 and 18 years of age, diagnosed…
- Trial name
- NCT00884949
- Testing
- BMN 110
- Where
- 3 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Irritable Bowel Syndrome
Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic Fermentation
To determine the effect on tolerance and gas production from chronic feeding of fermentable fibre (inulin) incorporated into a gel forming fibre (methylcellulose) compared with placebo (maltodextrin)
- Trial name
- CINNAMON (NCT06551961)
- Testing
- methylcellulose, inulin, maltodextrin
- Where
- 2 sites in United Kingdom
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Gastric Bypass, Blind Loop Syndrome
Small Intestinal Bacterial Overgrowth Obese
The objective of this research is to determine the incidence of microbial overgrowth of the small intestine before and after a gastric bypass.
- Conditions
- Gastric Bypass, Blind Loop Syndrome, Bacterial Overgrowth Syndrome, Chromatography, Gas
- Trial name
- SIBOB (NCT02819037)
- Testing
- gas chromatography, Stool analysis for detection of malabsorption
- Where
- 2 sites in France
- Volunteers
- 51 people
- Sponsor
- Lille Catholic University
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Gas, Bloating
Assessment and Validation of Electronic Gas and Bloating Diary
The investigator's goal is to improve the patient's compliance and accurate capture of patient reported gas and bloating symptoms, by utilizing a phone application that interactively records each gas and bloating event in…
- Conditions
- Gas, Bloating, Distension, Constipation
- Trial name
- NCT04684849
- Testing
- APP Diary, Paper Diary
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Augusta University
Head and Neck Cancer, Quality of Life
Effectiveness Verification of Using Hydrogen Inhalation to Improve Fatigue, Pain and Quality of Life in Cancer Patients
There are two recruitment methods for this study, which are mainly based on the Co-Principal Investigator Dr. Chih-Jen Huang recommendation of eligible subjects and poster recruitment methods.
- Conditions
- Head and Neck Cancer, Quality of Life, Pain, Sleep
- Trial name
- hydrogen (NCT05728112)
- Testing
- Hydrogen Inhalation, Ordinary Air Inhalation
- Where
- Taiwan
- Volunteers
- 30 people
- Sponsor
- Kaohsiung Medical University Chung-Ho Memorial Hospital
Bloating
A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy Population
The purpose of this study is to investigate the effects of OPTI-BIOME™ (Bacillus subtilis MB40), a probiotic supplement, on bloating, gas and abdominal discomfort symptoms in otherwise healthy adults.
- Trial name
- NCT02950012
- Testing
- OPTI-BIOME™ Bacillus subtilis MB40
- Where
- 3 sites in United States, Canada
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- BIO-CAT Microbials, LLC
SEDC, Hypochondrogenesis
A Natural History Study in Children With a Type II Collagen Disorder With Short Stature
There are relatively few data available on type II collagen disorders, and evidence is lacking on the disease course in relation to symptoms and development of complications, the level of actual disease burden over time as well…
- Conditions
- SEDC, Hypochondrogenesis, Semd, Strudwick Type, Kniest Dysplasia
- Trial name
- ROCKET (NCT05408715)
- Testing
- Natural History Study
- Where
- 2 sites in France, Spain
- Volunteers
- 60 people
- Sponsor
- Innoskel
Achondroplasia
A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With Achondroplasia
Approximately 63 participants will be randomized to one of three doses to receive Recifercept either Low Dose Medium Dose High Dose Participants will will attend the clinic at baseline and at Day 1, 4, 8, 15, 29 \& then Month 2…
- Trial name
- NCT04638153
- Testing
- Recifercept
- Where
- 16 sites in United States, Australia and 6 more
- Volunteers
- 60 people
- Sponsor
- Pfizer
Mucopolysaccharidoses, Mucopolysaccharidosis I
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
This study is being done to learn how many children and young adults who come to pediatric rheumatology clinics may have mucopolysaccharidosis (MPS). The study tests for 4 of the types of MPS: I, II, IVA, and VI.
- Conditions
- Mucopolysaccharidoses, Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV
- Trial name
- NCT01675674
- Testing
- Dried blood spot test for MPS
- Where
- 2 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- National MPS Society
Osteogenesis Imperfecta
Effects of Bisphosphonates on OI-Related Hearing Loss
Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in…
- Trial name
- NCT04152551
- Testing
- Risedronate Oral Tablet
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Hospital for Special Surgery, New York
Mucopolysaccharidosis IV Type A, Morquio A Syndrome
A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)
The objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the…
- Conditions
- Mucopolysaccharidosis IV Type A, Morquio A Syndrome, MPS IVA
- Trial name
- MARS (NCT02294877)
- Testing
- Vimizim® (elosulfase alfa)
- Where
- 79 sites in United States, Australia and 15 more
- Volunteers
- 418 people
- Sponsor
- BioMarin Pharmaceutical
Achondroplasia
Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia
All participants who completed the prior study to assess long-term safety, tolerability, pharmacokinetics and efficacy, and in the opinion of the investigator, continue to have a positive risk:benefit profile, will be offered to…
- Trial name
- NCT05116046
- Testing
- Recifercept
- Where
- 14 sites in United States, Australia and 5 more
- Volunteers
- 35 people
- Sponsor
- Pfizer
Morquio Disease
Establishment of Human Cellular Disease Models for Morquio Disease
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSMORQUIO (NCT03872713)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International