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Results for “Morquio Syndrome A”

69 trials

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Showing 20 of 69 results

Testing effectiveness (Phase 2)Ended earlyNCT05116046
What this trial is testing

Continuation Study of Long-term Safety, Tolerability, Pharmacokinetics and Efficacy of Recifercept in Achondroplasia

Who this might be right for
Achondroplasia
Pfizer 35
Not applicableNot Yet RecruitingNCT07516587
What this trial is testing

Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants

Who this might be right for
Digestive HealthAbdominal DiscomfortGas and Bloating
Arrae 100
Testing effectiveness (Phase 2)UnknownNCT03632213
What this trial is testing

Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI

Who this might be right for
Mucopolysaccharidosis IV AMucopolysaccharidosis VIMucopolysaccharidoses+6 more
Hospital de Clinicas de Porto Alegre 10
Not applicableStudy completedNCT07230821
What this trial is testing

Radicle GI Health RDH™: A Study Assessing the Impact of Health and Wellness Products on Gastrointestinal (GI) Health and Related Health Outcomes

Who this might be right for
Gas and BloatingDiarrheaConstipation
Radicle Science 500
Not applicableEnded earlyNCT01733615
What this trial is testing

Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA

Who this might be right for
Mucopolysaccharidosis Type IVA
Emory University 3
Not applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
Large-scale testing (Phase 3)Study completedNCT06164951
What this trial is testing

Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

Who this might be right for
Achondroplasia
QED Therapeutics, a BridgeBio company 114
Early research (Phase 1)Study completedNCT05846009
What this trial is testing

A First-in-human Single and Repeated Dose Escalation Study of SAR442501 in Healthy Adults Subjects

Who this might be right for
Osteochondrodysplasia
Sanofi 76
Not applicableStudy completedNCT07371975
What this trial is testing

Evaluate the Effects of a Butyrate-Polyphenol Formulation on Gut Health and Associated Symptoms

Who this might be right for
Gut Health
Supplement Formulators, Inc. 124
Not applicableStudy completedNCT02125565
What this trial is testing

Radial ABG Sampling: A RCT of Lidocaine Local Anaesthesia

Who this might be right for
Vascular Access
Mr Ryckie George Wade 43
Not applicableStudy completedNCT02208661
What this trial is testing

Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study)

Who this might be right for
Morquio A SyndromeMucopolysaccharidosis IV A
Nadia Ali, PhD 12
Testing effectiveness (Phase 2)Study completedNCT00884949
What this trial is testing

Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA

Who this might be right for
MPS IV A
BioMarin Pharmaceutical 20
Not applicableLooking for participantsNCT07591584
What this trial is testing

A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating

Who this might be right for
IBS (Irritable Bowel Syndrome)BloatingAbdominal Pain+1 more
Kiwi Health Inc 150
Testing effectiveness (Phase 2)Study completedNCT01887834
What this trial is testing

Efficacy of a Multi-strain Probiotic in the Treatment of Irritable Bowel Syndrome

Who this might be right for
Irritable Bowel SyndromeDigestive System DiseasesColonic Diseases, Functional+2 more
The Canadian College of Naturopathic Medicine 100
Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)Enrolling By InvitationNCT06873035
What this trial is testing

An Interventional Study of Infigratinib in Children With Hypochondroplasia

Who this might be right for
Hypochondroplasia
QED Therapeutics, a BridgeBio company 24
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Large-scale testing (Phase 3)Study completedNCT01415427
What this trial is testing

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

Who this might be right for
Mucopolysaccharidosis IV AMorquio A SyndromeMPS IVA
BioMarin Pharmaceutical 173
Large-scale testing (Phase 3)Study completedNCT01966029
What this trial is testing

BMN 110 Phase 3B in Australian Patients

Who this might be right for
Mucopolysaccharidosis IVA (Morquio A Syndrome)
BioMarin Pharmaceutical 13
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