Clinical Trials Logo

Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03153319
Study type Interventional
Source Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Contact
Status Active, not recruiting
Phase Phase 1/Phase 2
Start date June 5, 2017
Completion date June 2026

See also
  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Withdrawn NCT05238324 - Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II Phase 1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