Clinical trials: MPS - Mucopolysaccharidosis
Mucopolysaccharidoses, Carotid Disease
Cardiovascular Structure and Function in the Mucopolysaccharidoses
This study's investigators previously demonstrated the potential utility of non-invasive carotid ultrasonography to calculate carotid intima media thickness (cIMT) and stiffness (as measured by the three parameters, carotid…
- Conditions
- Mucopolysaccharidoses, Carotid Disease, Cardiac Disease, Inflammation
- Trial name
- NCT05063435
- Testing
- Carotid ultrasonography, Echocardiography, transthoracic, Venipuncture
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Children's Hospital of Orange County
Mucopolysaccharidosis Type IIIB, MPS IIIB (Sanfilippo B Syndrome)
Early Access Treatment With Tralesinidase Alfa in Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B)
This is an intermediate-size patient population Early Access Program (EAP) providing access to intracerebroventricular (ICV) tralesinidase alfa (TA) for participants with mucopolysaccharidosis type IIIB (MPS IIIB, Sanfilippo…
- Trial name
- EAP (NCT07733856)
- Testing
- Tralesinidase alfa (TA)
- Where
- United States
- Sponsor
- Spruce Biosciences
Mucopolysaccharidosis Type IIIA
Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA)
MPS IIIA is predominantly a central nervous system disease causing cognitive disability, progressive loss of acquired skills, behavioral and sleep disturbance.
- Trial name
- AAVance (NCT03612869)
- Testing
- LYS-SAF302
- Where
- 8 sites in United States, France and 3 more
- Volunteers
- 20 people
- Sponsor
- LYSOGENE
Mucopolysaccharidosis IVA, Morquio A Syndrome
Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This open-label Phase 2 study will evaluate the safety and efficacy of weekly 2.0 mg/kg/wk infusions of BMN 110 in pediatric patients, less than 5 years of age at the time of administration of the first dose of study drug…
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01515956
- Testing
- BMN 110
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 15 people
- Sponsor
- BioMarin Pharmaceutical
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
Digestive Disease, Gut Health
Impact of B. Adolescentis iVS-1 Probiotic on Gastrointestinal Outcomes
The study team is conducting a consumer-driven, decentralized clinical research study to assess the impact of the consumer-grade probiotic supplement, iVS-1, on adults with moderate to severe gastrointestinal discomfort.
- Trial name
- NCT07408518
- Testing
- Probiotic
- Where
- United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Synbiotic Health
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP1903) on day 7 to prevent GVHD.
- Conditions
- Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies
- Trial name
- NCT02231710
- Testing
- BPX-501 and Rimiducid
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Bellicum Pharmaceuticals
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
Sanfilippo Syndrome, MPS3
The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3)
The natural history study of patients with Sanfilippo disease(s) (MPS3)
- Trial name
- NCT05705674
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.
MPS IV A, Mucopolysaccharidosis IVA
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
This multicenter, open-label extension study is designed to assess long-term efficacy and safety of 2.0 milligrams (mg)/kilogram(kg)/week of BMN 110 in patients diagnosed with Mucopolysaccharidosis IVA (MPS IVA).
- Conditions
- MPS IV A, Mucopolysaccharidosis IVA, Morquio A Syndrome
- Trial name
- NCT01242111
- Testing
- BMN 110
- Where
- 4 sites in United Kingdom
- Volunteers
- 20 people
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IIIA, MPS IIIA
Follow-up Study of AAV-Mediated Gene Transfer (UX111; Previously Known as ABO-102) for MPS Type IIIA
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysaccharidosis IIIA (MPS IIIA).
- Conditions
- Mucopolysaccharidosis IIIA, MPS IIIA, Sanfilippo Syndrome, Sanfilippo A
- Trial name
- NCT04360265
- Testing
- No Investigational Product, Adjuvant Immunomodulatory (IM) Therapy
- Where
- 4 sites in United States, Australia and 1 more
- Volunteers
- 41 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
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
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.
MPS-IH (Hurler Syndrome)
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
A multi-center randomized clinical trial to compare OTL-203 (gene therapy) with stem cell transplant (standard of care) in patients with MPS-IH (Hurler syndrome).
- Trial name
- HURCULES (NCT06149403)
- Testing
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Where
- 5 sites in United States, Italy and 2 more
- Volunteers
- 41 people
- Sponsor
- Orchard Therapeutics
Irritable Bowel
Effect of Rate Of Delivery on Colonic Fermentation of inuliN (EON)
To test whether slowing the rate of delivery of inulin with either psyllium or divided dosing of inulin, will reduce colonic gas production as compared to inulin combined with placebo.
- Trial name
- EON (NCT05619341)
- Testing
- psyllium, inulin
- Where
- United Kingdom
- Volunteers
- 17 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
Mucopolysaccharidosis VI
Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI
The purpose of the study is to evaluate the safety, efficacy and pharmacokinetics of two dose levels of weekly intravenous infusions of recombinant human N-acetylgalactosamine 4-sulfatase (rhASB) for a minimum of 24 weeks in…
- Trial name
- NCT00048620
- Testing
- N-acetylgalactosamine 4-sulfatase
- Where
- United States
- Sponsor
- BioMarin Pharmaceutical
Mucopolysaccharidosis IV A
Natural History of Atypical Morquio A Disease
Mucopolysaccharidosis IVA (MPS IVA) (or Morquio A disease) is a rare recessive autosomal lysosomal storage disorder caused by deficiency of N-acetylgalactosamine-6-sulfatase (GALNS) resulting in accumulation of the…
- Trial name
- NCT03204370
- Testing
- Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM]
- Where
- France
- Volunteers
- 7 people
- Sponsor
- GOIZET
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
Mucopolysaccharidosis VI
A Study in MPS VI to Assess Safety and Efficacy of Odiparcil
Mucopolysaccharidoses (MPS) are a group of rare inherited disorders characterized by a deficiency of lysosomal enzymes responsible for the normal degradation of glycosaminoglycans (GAGs).
- Trial name
- iMProveS (NCT03370653)
- Testing
- Odiparcil
- Where
- 4 sites in France, Germany and 2 more
- Volunteers
- 20 people
- Sponsor
- Inventiva Pharma
MPS IIIB (Sanfilippo Syndrome)
A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB
The objective is to perform a retrospective chart review to generate data to evaluate the clinical characteristics and course of disease progression of MPS IIIB.
- Trial name
- NCT02293382
- Where
- 5 sites in United States, Brazil and 3 more
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.