Clinical trials: MPS I
Mucopolysaccharidosis I
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate…
- Trial name
- NCT05134571
- Testing
- Laronidase
- Where
- 5 sites in China
- Volunteers
- 12 people
- Sponsor
- Genzyme, a Sanofi Company
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
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
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
Mucopolysaccharidosis Type I, Gargoylism
Biomarker for Hurler Disease (BioHurler)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Hurler disease from plasma. Testing for clinical robustness, specificity and long-term stability of the biomarker.
- Conditions
- Mucopolysaccharidosis Type I, Gargoylism, Metabolism, Inborn Errors
- Trial name
- BioHurler (NCT02298712)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
- Trial name
- NCT05682144
- Testing
- Autologous Plasmablasts (B cells)
- Where
- 2 sites in United States
- Volunteers
- 11 people
- Sponsor
- Immusoft of CA, Inc.
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Mucopolysaccharidosis I
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
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
- NCT02597114
- Testing
- AGT-181
- Volunteers
- 3 people
- Sponsor
- ArmaGen, Inc
Inherited Metabolic Diseases, Lysosomal Storage Disorders
ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Eligible research subjects will receive an unrelated umbilical cord blood transfusion as a possible cure for their inherited metabolic disease.
- Conditions
- Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism
- Trial name
- UCBT-002 (NCT00654433)
- Testing
- ALD-101
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- Aldagen
Mucopolysaccharidosis I
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).
- Trial name
- NCT04227600
- Testing
- JR-171 (lepunafusp alfa)
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 18 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis I
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 (NCT04227600) for the treatment of MPS I
- Trial name
- NCT04453085
- Testing
- JR-171
- Where
- 6 sites in United States, Brazil and 1 more
- Volunteers
- 14 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Mucopolysaccharidosis I
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children 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
- NCT03053089
- Testing
- AGT-181
- Where
- Brazil
- Volunteers
- 21 people
- Sponsor
- ArmaGen, Inc
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
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome
RGX-111 Gene Therapy in Patients With MPS I
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system.
- Conditions
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT03580083
- Testing
- RGX-111
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Inherited Metabolic Disorders (IMD)
MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)
This study is designed to assess the safety and efficacy of using MGTA-456 in patients with Inherited Metabolic Disorders (IMD) undergoing stem cell transplantation.
- Trial name
- NCT03406962
- Testing
- MGTA-456
- Where
- 2 sites in United States
- Volunteers
- 8 people
- Sponsor
- Magenta Therapeutics, Inc.
Mucopolysaccharidosis I, Hurler Syndrome
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease.
- Conditions
- Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
- Trial name
- NCT00258011
- Testing
- Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
- Where
- 2 sites in Japan
- Volunteers
- 3 people
- Sponsor
- Genzyme, a Sanofi Company
Hepato-splenomegaly, Dysostosis Multiplex
Biomarker for Gangliosidosis: BioGM1/BioGM2 (BioGM1/GM2)
Development of a new MS-based biomarker for the ear-ly and sensitive diagnosis of GM1/GM2 from blood
- Conditions
- Hepato-splenomegaly, Dysostosis Multiplex, Seizures, Mental Retardation
- Trial name
- BioGM1/BioGM2 (NCT02298647)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Mucopolysaccharidosis I, Hurler's Syndrome
A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients
The purpose of this study is to determine whether the development of antibodies to laronidase in patients with MPS I receiving Aldurazyme® impairs the clearance of GAG substrate.
- Conditions
- Mucopolysaccharidosis I, Hurler's Syndrome, Hurler-Scheie Syndrome, Scheie's Syndrome
- Trial name
- NCT00144768
- Testing
- laronidase
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Genzyme, a Sanofi Company
Mucopolysaccharidosis I
Long Term Follow-Up for RGX-111
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase…
- Trial name
- NCT06103487
- Testing
- No Intervention
- Where
- 2 sites in United States, Brazil
- Volunteers
- 21 people
- Sponsor
- REGENXBIO Inc.
Mucopolysaccharidosis II, MPS II
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
The purpose of the study is to evaluate the safety, tolerability and effect on leukocyte and plasma Iduronate 2-Sulfatase (IDS) enzyme activity of ascending doses of SB-913.
- Trial name
- NCT03041324
- Testing
- SB-913
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Sangamo Therapeutics