Mucopolysaccharidosis II Clinical Trial
Official title:
Phase 1/2 Study of the Effect of Adalimumab on Physical Function and Musculoskeletal Disease in Mucopolysaccharidosis Types I, II, and VI
Randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI.
This study is a randomized, double-blind, placebo-controlled, parallel-group, single-center study followed by open-label phase, to evaluate the effects of adalimumab compared to placebo on the change from baseline in joint and skeletal disease in children and adults with mucopolysaccharidosis (MPS) I, II or VI. Children and adults diagnosed with MPS I, II or VI, with significant joint restrictions and pain will be randomized to adalimumab treatment or placebo treatment for the first 16 weeks. This will be followed by a 32-week open label adalimumab treatment phase. ;
| 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| Withdrawn |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
|