Mucopolysaccharidosis IV: all 101 studies
Page 1 of 2. Open to participants first (27), then studies with results posted, then the rest; newest registration first within each.
- A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported BloatingRecruitingNCT07591584Not applicable
- A Study to Investigate the Safety, Pharmacokinetics (PK), and Efficacy of Garetosmab in Children and Adolescents With Fibrodysplasia Ossificans Progressiva (FOP)Not yet recruitingNCT07559513Phase 3
- Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy ParticipantsNot yet recruitingNCT07516587Not applicable
- Open-Label, Long-Term, Extension Study of Infigratinib in Children With HypochondroplasiaEnrolling by invitationNCT07393373Phase 2
- Cardiac Structure and Function in MPSRecruitingNCT07361536
- Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)RecruitingNCT07169279Phase 2
- An Interventional Study of Infigratinib in Children With HypochondroplasiaEnrolling by invitationNCT06873035Phase 2/3
- Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic SequencingRecruitingNCT06749366
- Assessment of the Impact of Intestinal Gas Emission Quality on the Postoperative Course After Abdominal SurgeryRecruitingNCT06675097
- Effect of Chronic Feeding of Inulin And Methylcellulose on Colonic FermentationRecruitingNCT06551961Not applicable
- Prospective Clinical Assessment Study in Children With HypochondroplasiaRecruitingNCT06410976
- Efficacy of BIS Monitoring in Deep Hypnotic StateNot yet recruitingNCT06177847Not applicable
- MPS (RaDiCo Cohort) (RaDiCo-MPS)RecruitingNCT06036693
- Assessment of Artificial Intelligence for Treatment Decision Recommendation of Adult Skeletal Class III PatientsRecruitingNCT06002373
- Decoding the Genetic Landscape of Skeletal DiseasesRecruitingNCT05876416
- Multidisciplinary Evaluation and a Genome-wide Analysis in a Cohort of Idiopathic Short Stature PatientsRecruitingNCT05858606Not applicable
- Registry of Patients Diagnosed With Lysosomal Storage DiseasesRecruitingNCT05619900
- A Natural History Study in Children With a Type II Collagen Disorder With Short StatureRecruitingNCT05408715
- ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening ProgramEnrolling by invitationNCT05368038
- Non-invasive Functional Assessment and Pathogenesis of Morquio ARecruitingNCT05284006
- Data Collection of Patients With Rare Bone DiseasesRecruitingNCT05247645
- Extension Study of Infigratinib in Children With Achondroplasia (ACH)Enrolling by invitationNCT05145010Phase 2
- Study of Skeletal DisordersRecruitingNCT05031507
- Registry GangliosidosesRecruitingNCT04624789
- PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)RecruitingNCT04532047Phase 1
- Effects of Bisphosphonates on OI-Related Hearing LossRecruitingNCT04152551Phase 4
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT03333200
- Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in AchondroplasiaTerminatedNCT05116046Phase 2Results posted
- Efficacy and Safety of mAnnitol in Bowel Preparation During Elective Colonoscopy and Comparison With Moviprep®CompletedNCT04759885Phase 2/3Results posted
- A Study Of Safety, Tolerability And Effectiveness Of Recifercept In Children With AchondroplasiaTerminatedNCT04638153Phase 2Results posted
- North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2CompletedNCT03548779Not applicableResults posted
- Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited AmbulationTerminatedNCT01697319Phase 2Results posted
- Safety and Exercise Study of Two Doses of BMN 110 for Morquio A SyndromeTerminatedNCT01609062Phase 2Results posted
- Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01515956Phase 2Results posted
- A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel LeakageCompletedNCT01475474Not applicableResults posted
- Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01415427Phase 3Results posted
- A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)CompletedNCT01275066Phase 3Results posted
- A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)TerminatedNCT01242111Phase 1/2Results posted
- Efficacy and Safety Evaluation of Recombinant Human Growth Hormone (r-hGH), Saizen®, on a Population of Children With Hypochondroplasia, Treated at Least 3 Years or Until Near Final Height, When Applicable, in Comparison With a Historic Cohort of Non-treated ChildrenCompletedNCT01111019Phase 2Results posted
- Pregabalin and Colonic FunctionCompletedNCT01094808Phase 4Results posted
- A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVACompletedNCT00884949Phase 1/2Results posted
- Effect of Abdominal Massage on Gastrointestinal Symptoms After Head and Neck Cancer SurgeryCompletedNCT07889011Not applicable
- Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal OutcomesCompletedNCT07408518Not applicable
- A Study to Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated SymptomsCompletedNCT07371975Not applicable
- Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health OutcomesCompletedNCT07230821Not applicable
- Effectiveness of a Butyrate Formulation and Butyrate + Polyphenol Formulation on Gut Health, Permeability and Associated SymptomsCompletedNCT06419231Not applicable
- CO2 Supplement for Treatment of Acute Mountain SicknessCompletedNCT06393998Not applicable
- Sevoflurane Wash in Method Using Conventional Flows Versus Low Flows of 0.5LCompletedNCT06209112
- A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With AchondroplasiaCompletedNCT06164951Phase 3
- A Non-Interventional Study of Clinical Characteristics and Mortality of US Patients With Fibrodysplasia Ossificans Progressiva (FOP)CompletedNCT06064656
- Extreme Morphology and Metabolic HealthActive, not recruitingNCT05991609
- Safety and Efficacy of Voxzogo for Growth Deficits in MPS IVA and VIActive, not recruitingNCT05845749Phase 1/2
- Effect of Inhalation of Low Dose CO2 on Exercise Performance at High AltitudeCompletedNCT05798416Not applicable
- Effectiveness Verification of Using Hydrogen Inhalation to Improve Fatigue, Pain and Quality of Life in Cancer PatientsStatus unknownNCT05728112Not applicable
- Baby Detect : Genomic Newborn ScreeningCompletedNCT05687474
- Effect of Rate Of Delivery on Colonic Fermentation of inuliN (EON)CompletedNCT05619341Not applicable
- The Effect of Paula Method Exercises on Post-cesarean Section DistensionStatus unknownNCT05283707Not applicable
- Effects of Resistant Potato Starch on the Gut MicrobiotaCompletedNCT05242913Not applicable
- Awareness in Old Aged Patients During Laryngoscopy and Intubation Using Isolated Forearm TechniqueCompletedNCT05019560Phase 4
- Assessment and Validation of Electronic Gas and Bloating DiaryStatus unknownNCT04684849
- Natural History of Morquio B and Late-Onset of GM1 GangliosidosisStatus unknownNCT04320329
- Study of Infigratinib in Children With AchondroplasiaCompletedNCT04265651Phase 2
- End Tidal Anesthetic Concentration in Different Anesthesia Techniques Where Depth of Anesthesia Adjusted With EntropyCompletedNCT04203290Not applicable
- CARbon Impact of aNesthesic GasCompletedNCT04163848
- Prospective Clinical Assessment Study in Children With Achondroplasia (ACH)CompletedNCT04035811
- Clinical Evaluation of Acuity 200 (Fluoroxyfocon A) RGP Contact LensCompletedNCT03971227Not applicable
- Establishment of Human Cellular Disease Models for Morquio DiseaseCompletedNCT03872713
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
- Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VIStatus unknownNCT03632213Phase 2
- Natural History of Atypical Morquio A DiseaseCompletedNCT03204370
- Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?CompletedNCT03150069
- A Study to Evaluate OPTI-BIOME™ Bacillus Subtilis MB40 on Abdominal Discomfort, Gas and Bloating in a Healthy PopulationCompletedNCT02950012Phase 2
- Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related SymptomsCompletedNCT02936713Not applicable
- Small Intestinal Bacterial Overgrowth ObeseCompletedNCT02819037Not applicable
- Identification and Characterization of Bone-related Genetic Variants in FamiliesTerminatedNCT02762318
- A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)CompletedNCT02294877
- Probiotic Supplementation Among Adults With Recurrent Gastrointestinal SymptomsCompletedNCT02277431Phase 2/3
- Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)CompletedNCT02208661
- Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVaWithdrawnNCT02153255
- Radial ABG Sampling: A RCT of Lidocaine Local AnaesthesiaCompletedNCT02125565Not applicable
- Ultrasound Findings of Finger, Wrist and Knee Joints in MucopolysaccharidosisCompletedNCT02067650
- BMN 110 Phase 3B in Australian PatientsCompletedNCT01966029Phase 3
- Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy SyndromeCompletedNCT01961518
- Gait Analysis in MPS IVACompletedNCT01920828
- Efficacy of a Multi-strain Probiotic in the Treatment of Irritable Bowel SyndromeCompletedNCT01887834Phase 1/2
- Longitudinal Studies of Brain Structure and Function in MPS DisordersCompletedNCT01870375
- BMN 110 US Expanded Access ProgramApproved for marketingNCT01858103
- Safety and Effect of L. Reuteri on Biomarkers of Inflammation in Healthy Infants With ColicCompletedNCT01849991Phase 1
- Psychological Concomitants of Morquio Syndrome (The MAP Study)CompletedNCT01752296
- Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVATerminatedNCT01733615
- Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip DiseaseCompletedNCT01707433
- Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia ClinicsTerminatedNCT01675674
- Biomarker for Morquio Disease (BioMorquio)WithdrawnNCT01457456
- Efficacy of a Multi-strain Probiotic in the Treatment of Irritable Bowel Syndrome (IBS)CompletedNCT01176227Phase 1/2
- Observation of Gas Exchange in the Middle EarStatus unknownNCT00794105
- A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)TerminatedNCT00787995
- Curcumin (Tumeric) in the Treatment of Irritable Bowel Syndrome: A Randomized-Controlled TrialCompletedNCT00779493Phase 4
- Morquio's Syndrome: a Case StudyTerminatedNCT00609440Phase 4
- Stromal Therapy of Osteodysplasia After Allogeneic Bone Marrow TransplantationCompletedNCT00186914Phase 1
- Genetic Analysis of the Chiari I MalformationCompletedNCT00004738