Clinical trials: Mucinoses
Morquio Disease
Establishment of Human Cellular Disease Models for Morquio Disease
Establishment of human cellular disease models for Morquio disease for an individualized therapy development having the capacity to address both hepatic and neurologic forms of the disease
- Trial name
- IPSMORQUIO (NCT03872713)
- Where
- Pakistan
- Volunteers
- 40 people
- Sponsor
- CENTOGENE GmbH Rostock
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
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
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.
Mucopolysaccharidosis II
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
MPS II is a genetic disorder that affects boys. Boys with MPS II are missing a working enzyme known as iduronate-2-sulfatase (IDS) which is needed to break down long sugar chains in the body.
- Trial name
- NCT05665166
- Testing
- Autologous CD34+ HSCs transduced ex vivo with CD11B LV encoding human IDS tagged with ApoEII
- Where
- United Kingdom
- Volunteers
- 5 people
- Sponsor
- University of Manchester
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
Sanfilippo Syndrome
Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease
Sanfilippo syndrome Type A, or Mucopolysaccharidosis (MPS) IIIA, is a rare lysosomal storage disease caused by deficiency of the enzyme heparan N-sulfatase (sulfamidase).
- Trial name
- NCT02060526
- Testing
- Recombinant human heparan N-sulfatase [rhHNS]
- Where
- 10 sites in United States, Argentina and 6 more
- Volunteers
- 21 people
- Sponsor
- Shire
Sanfilippo Syndrome Type A
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including…
- Trial name
- NCT01047306
- Testing
- assessment
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Shire
Microstomia, Scleroderma
Hyaluronidase in Treating Oral Microstomia in Patients With Sclerosing Skin Disease
The principal objective is to measure the degree to which oral microstomia caused by sclerosing skin disease improves after treating patients with local hyaluronidase injections.
- Conditions
- Microstomia, Scleroderma, CREST Syndrome, Scleromyxedema
- Trial name
- NCT04656704
- Testing
- hyaluronidase injected intradermally
- Sponsor
- Brigham and Women's Hospital
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sanfilippo Syndrome Type A
Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhHNS, GC1130A) administered via intracerebroventricular access device…
- Trial name
- NCT06567769
- Testing
- GC1130A
- Where
- 4 sites in United States, South Korea
- Volunteers
- 9 people
- Sponsor
- GC Biopharma Corp
Parkinson's Disease
Study of AAV-GAD Gene Transfer Into the Subthalamic Nucleus for Parkinson's Disease
The purpose of this study is to determine the safety and efficacy of AAV-GAD gene transfer into the subthalamic nucleus (STN) region of the brain.
- Trial name
- NCT00643890
- Testing
- Bilateral surgical infusion of AAV-GAD into the subthalamic nucleus
- Where
- 7 sites in United States
- Volunteers
- 44 people
- Sponsor
- Neurologix, Inc.
Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI
Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease
BACKGROUND/OBJECTIVE: Quantitative urine screening for mucopolysaccharides (MPS) has been the primary method for detecting mucopolysaccharidoses in children.
- Trial name
- NCT01707433
- Testing
- Enzyme testing
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- Children's Hospitals and Clinics of Minnesota
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
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
Mucopolysaccharidoses
Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network
Mucopolysaccharidosis (MPS) syndromes are disorders characterized by enzyme deficiencies, and they have been linked to heart health complications.
- Trial name
- NCT01586871
- Where
- 2 sites in United States
- Volunteers
- 1,147 people
- Sponsor
- University of Minnesota
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
Sanfilippo Syndrome Type B
Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B)
The purpose of this study is to evaluate the natural course of disease progression in Mucopolysaccharidosis Type III (MPS IIIB) patients who are untreated to identify potential surrogate endpoints that may be utilized in future…
- Trial name
- NCT01509768
- Where
- 4 sites in United States, Germany and 1 more
- Volunteers
- 19 people
- Sponsor
- Shire
Mucopolysaccharidosis Type II, Metabolic Diseases
A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers
It is a phase I open-label single-dose, dose-escalation cohort study to evaluate of the tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers
- Trial name
- NCT04539340
- Testing
- GNR-055
- Where
- Russia
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- AO GENERIUM
Morquio Disease, Mucopolysaccharidosis IV
Psychological Concomitants of Morquio Syndrome (The MAP Study)
Mucopolysaccharidosis IV, also known as MPS IV or Morquio disease, is a rare autosomal recessive genetic lysosomal storage disorder.
- Trial name
- MAP (NCT01752296)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Nadia Ali, PhD