Mucopolysaccharidosis II Clinical Trial
Official title:
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
MPS II is a rare, X-linked, lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase. Because of this deficiency, glycosaminoglycans (GAG) accumulate in multiple tissues and organs, resulting in progressive cellular and organ system dysfunction. The purpose of this study is to determine if one year of therapy with iduronate-2-sulfatase enzyme replacement therapy, at a dose of 0.5mg/kg, weekly or every other week, is safe, and results in clinically meaningful improvement in multiple organ function, compared with a placebo group. Upon completion of the study, patients will be eligible to enroll in an open-label maintenance study. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Withdrawn |
NCT05238324 -
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II
|
Phase 1 | |
| Completed |
NCT03529786 -
Mucopolysaccharidosis Type II Natural History
|
||
| Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
| Terminated |
NCT01675674 -
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
|
N/A | |
| Enrolling by invitation |
NCT06075537 -
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
|
Phase 2/Phase 3 | |
| Recruiting |
NCT05422482 -
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ
|
Phase 1 | |
| Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
| Active, not recruiting |
NCT04348136 -
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
|
Phase 2/Phase 3 | |
| Completed |
NCT04007536 -
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
|
||
| Completed |
NCT00004454 -
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT04628871 -
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
|
||
| Terminated |
NCT00748969 -
Clinical Trial of Growth Hormone in MPS I, II, and VI
|
Phase 2/Phase 3 | |
| Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
||
| Completed |
NCT01301898 -
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
|
Phase 1/Phase 2 | |
| Terminated |
NCT03041324 -
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
|
Phase 1/Phase 2 | |
| Enrolling by invitation |
NCT05368038 -
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
|
||
| Completed |
NCT03128593 -
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
|
Phase 1/Phase 2 | |
| Withdrawn |
NCT04591834 -
Mucopolysaccharidosis Type II Observational
|
||
| Enrolling by invitation |
NCT04597385 -
Long-term Follow-Up for RGX-121
|
||
| Active, not recruiting |
NCT03153319 -
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
|
Phase 1/Phase 2 |