Clinical trials: MPS - Mucopolysaccharidosis
Mucopolysaccharidosis IVA, Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01697319
- Testing
- BMN 110
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis III-A
A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)
This protocol is a decentralized, single cohort, natural history and biomarker study enrolling up to 20 participants with MPS IIIA (Sanfilippo syndrome).
- Trial name
- NCT05523206
- Testing
- No intervention
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Sanguine Biosciences
IBS (Irritable Bowel Syndrome), Bloating
A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Individuals With Self-Reported Bloating
This study is a randomized, double-blind, placebo-controlled, within-individual crossover trial designed to assess the impact of regular use of a consumer-grade FODMAP-targeting digestive enzyme blend (FODZYME®) on…
- Conditions
- IBS (Irritable Bowel Syndrome), Bloating, Abdominal Pain, Gut Health
- Trial name
- NCT07591584
- Testing
- FODMAP-targeting digestive enzyme blend
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- Kiwi Health Inc
Vascular Access
Radial ABG Sampling: A RCT of Lidocaine Local Anaesthesia
Arterial Blood Gas (ABG) sampling is a common procedure in all hospitals worldwide. Often, ABG samples are obtain from inserting a needle into the radial artery at the wrist - this procedure is often painful and dreaded by…
- Trial name
- NCT02125565
- Testing
- Injected subcutaneous local (lidocaine) anaesthesia
- Where
- United Kingdom
- Volunteers
- 43 people
- Sponsor
- Mr Ryckie George Wade
Abdominal Massage, Enteral Nutrition
Effect of Abdominal Massage on Gastrointestinal Symptoms After Head and Neck Cancer Surgery
This study aimed to evaluate whether abdominal massage could reduce abdominal pain, constipation, and gas/bloating in patients receiving enteral nutrition through a nasogastric tube after surgery for head and neck cancer.
- Conditions
- Abdominal Massage, Enteral Nutrition, Nasogastric Feeding, Head and Neck Surgery
- Trial name
- HNC-AM (NCT07889011)
- Testing
- Abdominal Massage
- Where
- Turkey (Türkiye)
- Volunteers
- 70 people
- Sponsor
- Maltepe University
Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.
- Trial name
- NCT03568175
- Testing
- JR-141
- Where
- 19 sites in Japan
- Volunteers
- 28 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis Type IVA
Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA
The purpose of this study is to find out more about some of the unusual chemicals (called oligosaccharides) that can occur naturally as a result of processes in the body.
- Trial name
- NCT01733615
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Mucopolysaccharidosis II
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II
Phase 1, open-label, sequential ascending dose-escalation study. Designed to evaluate the safety and efficacy of a single IV infusion of investigational gene therapy HMI-203.
- Trial name
- NCT05238324
- Testing
- Genetic HMI-203
- Where
- 6 sites in United States, Canada
- Sponsor
- Homology Medicines, Inc
General Anesthesia, Inhalation; Gas
End Tidal Anesthetic Concentration in Different Anesthesia Techniques Where Depth of Anesthesia Adjusted With Entropy
The aim of this study is to adjust the end tidal inhalation anesthetic concentration by observing the entropy value in patients who will be provided anesthesia maintenance with volatile anesthetics after applying the routine…
- Trial name
- NCT04203290
- Testing
- Bupivacaine, Sevoflurane
- Where
- Turkey (Türkiye)
- Volunteers
- 44 people
- Sponsor
- Istanbul University
Mucopolysaccharidosis Type 3 B
Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB
Open-label, dose-escalation clinical trial of rAAV9.CMV.hNAGLU injected intravenously through a peripheral limb vein
- Trial name
- MPSIIIB (NCT03315182)
- Testing
- rAAV9.CMV.hNAGLU
- Where
- 4 sites in United States, France and 2 more
- Volunteers
- 11 people
- Sponsor
- Abeona Therapeutics, Inc
Ophthalmic Manifestations
Ophthalmic Manifestations of Patients With Mucopolysaccharidosis
Mucopolysaccharidosis (MPSs) are a group of disorders caused by inherited defects in lysosomal enzymes resulting in widespread intra- and extra-cellular accumulation of glycosaminoglycan(1,2).
- Trial name
- NCT04770415
- Where
- Egypt
- Volunteers
- 28 people
- Sponsor
- Shrouk Shaban Bakr
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Achondroplasia
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who…
- Trial name
- NCT05145010
- Testing
- Infigratinib
- Where
- 31 sites in United States, Argentina and 8 more
- Volunteers
- 325 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Enchondromatosis
Uncovering Genes Behind Cartilage Tumors and Vascular Anomalies Using Genomic Sequencing
Background: Ollier disease (OD) and Maffucci syndrome (MS) are rare disorders that increase the risk of cancers in cartilage tissue. These tumors can lead to severe skeletal deformities beginning in childhood.
- Trial name
- NCT06749366
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome)
Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP)
The objectives of this program are: to further characterize the natural progression of MPS VI disease; to generate and disseminate information on the care and management of MPS VI patients to clinical and medical professionals…
- Trial name
- NCT00214773
- Where
- 50 sites in United States, Australia and 11 more
- Volunteers
- 237 people
- Sponsor
- BioMarin Pharmaceutical
Hunter Syndrome
Extension Study of Idursulfase-IT Along With Elaprase in Children and Adults With Hunter Syndrome and Cognitive Impairment
The study is an extension of two previous studies (HGT-HIT-046 [NCT01506141] and SHP609-302 [NCT02412787]). Participants must have completed one of the previous studies.
- Trial name
- NCT06031259
- Testing
- Idursulfase-IT, Elaprase
- Where
- 4 sites in Canada, France
- Volunteers
- 6 people
- Sponsor
- Takeda
MPS I
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I
The purpose of the study is to evaluate the safety, tolerability of ascending doses of SB-318. SB-318 is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing.
- Trial name
- NCT02702115
- Testing
- SB-318
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Sangamo Therapeutics
Mucopolysaccharidosis (MPS), Hunter Syndrome
A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)
The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter…
- Trial name
- NCT05494593
- Testing
- ELAPRASE, Rituximab, Methotrexate
- Where
- 8 sites in United States
- Sponsor
- Takeda
Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B
A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Mucopolysaccharidosis type IIIB (MPS IIIB, also known as Sanfilippo Syndrome Type B) is a severe neurodegenerative disorder. The purpose of this study is to learn more about the health problems in patients with MPS IIIB and how…
- Conditions
- Mucopolysaccharidosis Type IIIB, Mucopolysaccharidosis Type 3 B, MPS III B, MPS 3 B
- Trial name
- NCT02493998
- Where
- 8 sites in United States, Australia and 6 more
- Volunteers
- 22 people
- Sponsor
- Allievex Corporation
Mucopolysaccharidosis IIIB
A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
This is a global, open-label, Phase I/II, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its…
- Trial name
- NCT07640984
- Testing
- JR-446
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 12 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.