Clinical trials: MPS - Mucopolysaccharidosis
MPS IV A
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This Phase 3 study will evaluate the efficacy and safety of 2.0 mg/kg/week BMN 110 and 2.0 mg/kg/every other week BMN 110 in patients with mucopolysaccharidosis IVA (Morquio A Syndrome).
- Trial name
- NCT01275066
- Testing
- BMN 110 Weekly, BMN 110 Every Other Week
- Where
- 28 sites in United States, Argentina and 15 more
- Volunteers
- 177 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis Type 1
Efficacy and Safety of YW17 (Laronidase-CinnaGen) Compared to Aldurazyme® in MPS I Patients
The purpose of this phase III study is to assess the efficacy and safety of YW17 produced by CinnaGen Company compared to Aldurazyme® in mucopolysaccharidosis type I (MPS I) patients.
- Trial name
- NCT06406153
- Testing
- Laronidase, Antihistamine, Antipyretic
- Where
- 4 sites in Iran
- Volunteers
- 12 people
- Sponsor
- Cinnagen
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
Sanfilippo Syndrome Type A (MPS IIIA)
A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients
MPS IIIA, also known as Sanfilippo A, is an inherited lysosomal storage disease (LSD). MPS IIIA is caused by a deficiency in sulfamidase, one of the enzymes involved in the lysosomal degradation of the glycosaminoglycan (GAG)…
- Trial name
- NCT03423186
- Testing
- SOBI003
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 6 people
- Sponsor
- Swedish Orphan Biovitrum
Digestive Health, Abdominal Discomfort
Study to Investigate the Efficacy of a Digestive Health Dietary Supplement on Post-meal Bloating in Healthy Participants
The goal of this crossover clinical trial is to evaluate the efficacy and safety of Bloat Gummies on postprandial bloating and related gastrointestinal discomfort in healthy adults with recurrent bloating.
- Conditions
- Digestive Health, Abdominal Discomfort, Gas and Bloating
- Trial name
- NCT07516587
- Testing
- Bloat Gummies
- Where
- United States
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Arrae
Mucopolysaccharidosis II
Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome
AGT-182 is a fusion protein containing idursulfase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT02262338
- Testing
- AGT-182
- Where
- 5 sites in United States, Germany and 1 more
- Volunteers
- 6 people
- Sponsor
- ArmaGen, Inc
Morquio Syndrome, Accumulation of Mucopolysaccharides
Biomarker for Morquio Disease (BioMorquio)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Morquio disease from plasma
- Conditions
- Morquio Syndrome, Accumulation of Mucopolysaccharides, Morquio Syndrome A, Morquio B Disease
- Trial name
- BioMorquio (NCT01457456)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Sanfilippo Syndrome Type D
A Natural History Study of Sanfilippo Syndrome Type D
Sanfilippo syndrome type D is a ultra rare syndrome with limited available natural history data. This study is planned to document, through retrospective and prospective data collection, syndrome progression in children and young…
- Trial name
- MPSIIID (NCT05648851)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Phoenix Nest
Hunter Syndrome
Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome
As the HGT-HIT-046 (NCT01506141) and SHP609-302 (NCT02412787) studies are completed and closed, this post-trial access (PTA) program provides TAK-609 to participants in these studies for whom the benefit:risk ratio of continued…
- Trial name
- NCT05795361
- Testing
- Idursulfase-IT
- Where
- 22 sites in United States, Australia and 3 more
- Sponsor
- Takeda
Mucopolysaccharidosis I, Mucopolysaccharidosis VI
Stem Cell Transplantation for Hurler
The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for Hurler syndrome…
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
- Trial name
- NCT00176917
- Testing
- Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Morquio Syndrome A, Maroteaux Lamy Syndrome
Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome
The purpose of this study is to identify patients with Morquio syndrome type A (MPS IVA) and Maroteaux-Lamy syndrome (MPS VI) who may have been missed or misdiagnosed due to atypical clinical features, a milder course, and/or…
- Conditions
- Morquio Syndrome A, Maroteaux Lamy Syndrome, MPS IVA, MPS VI
- Trial name
- NCT01961518
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Greenwood Genetic Center
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
MPS I
The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan
MPS I newborn screening has been executed in Taiwan nationwide since August 2015. Infants who failed the recheck at recall were referred to MacKay Memorial Hospital for a detailed confirmatory diagnosis.
- Trial name
- NCT04958070
- Testing
- MPS I
- Where
- Taiwan
- Volunteers
- 20 people
- Sponsor
- Mackay Memorial Hospital
Mucopolysaccharidosis I
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
AGT-181 is a fusion protein containing alpha-L-Iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT02371226
- Testing
- AGT-181 (HIRMAb-IDUA)
- Where
- 6 sites in United States
- Volunteers
- 3 people
- Sponsor
- ArmaGen, Inc
Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant.
- Trial name
- NCT05634512
- Testing
- Laronidase therapy and a stem cell transplant
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Mucopolysaccharidosis I
Immune Tolerance Study With Aldurazyme® (Laronidase)
The purpose of this study is to see if treatment with an antigen-specific immunosuppressive can decrease or stop an antibody response to laronidase (Aldurazyme®) during enzyme replacement therapy with laronidase in severe…
- Trial name
- NCT00741338
- Testing
- Laronidase, Cyclosporine A (CsA), Azathioprine (Aza)
- Where
- 2 sites in Brazil, Russia
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Hunter Syndrome
A Study of GC1111 in Hunter Syndrom Patients
The objective of this study is to evaluate the efficacy of GC1111 in Hunter Syndrome Patients
- Trial name
- NCT03920540
- Testing
- GC1111, Comparator
- Where
- South Korea
- Volunteers
- 32 people
- Sponsor
- Green Cross Corporation
High Altitude, Inhalation; Gas
Effect of Inhalation of Low Dose CO2 on Exercise Performance at High Altitude
High altitude (>2400 m) is associated with decreased atmosphere pressure leading to hypoxia which in turn impairs exercise capacity and causes acute mountain sickness (AMS).
- Conditions
- High Altitude, Inhalation; Gas, Acute Mountain Sickness
- Trial name
- NCT05798416
- Testing
- Exercise under inhalation CO2, Exercise under inhalation ambient air
- Where
- China
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- State Key Laboratory of Respiratory Disease
Morquio Disease, MPS - Mucopolysaccharidosis
Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
The present study seeks to interview women with Morquio A and Morquio B syndrome, to explore their concerns surrounding pregnancy and the impact of ERT on their perspectives, in comparison with the control group of Morquio B…
- Trial name
- MorqPreg (NCT03150069)
- Testing
- Interview
- Where
- United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Emory University
MPS II, Hunter Syndrome (MPS II)
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system.
- Trial name
- NCT07236606
- Testing
- RGX-121-3102
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- REGENXBIO Inc.