Mucopolysaccharidosis II Clinical Trial
Official title:
A Prospective, Longitudinal Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
This is a six-part prospective, multicenter, multiregional observational study of patients with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, to assess biomarkers potentially related to disease severity and/or treatment response and prospectively assess the progression of disease in participants with MPS II who are aged ≤30 years at the time of enrollment.
This clinical trial information was submitted voluntarily under the applicable law and, therefore, certain submission deadlines may not apply. (That is, clinical trial information for this applicable clinical trial was submitted under section 402(j)(4)(A) of the Public Health Service Act and 42 CFR 11.60 and is not subject to the deadlines established by sections 402(j)(2) and (3) of the Public Health Service Act or 42 CFR 11.24 and 11.44.). ;
| 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| Withdrawn |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 | |
| Completed |
NCT00069641 -
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
|
Phase 2/Phase 3 | |
| 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 |
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 |