Mucopolysaccharidosis II Clinical Trial
Official title:
Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II
A Phase II open-label, randomized, parallel group, 2 sites (Brazil), designed to evaluate the safety and efficacy of 3 doses of study drug for the treatment of the MPS II.
n/a
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Withdrawn |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 |
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
|