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.
Status | Active, not recruiting |
Enrollment | 14 |
Est. completion date | June 2026 |
Est. primary completion date | December 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 5 Years and older |
Eligibility | Inclusion Criteria: - Male or female =5 years of age; - Diagnosis of MPS I, II or VI; - Treatment with ERT for =1 year or no treatment with ERT for =1 year; - Weight =15 kg; - Significant bodily pain reported by the CHQ-PF50 or SF-36 (> 1 SD more severe [below] than the general population mean); - = 3 joints with limitations in motion; and Patient or parent/legal guardian is able and willing to provide informed consent. For patients 7 to 17 years of age, assent must also be provided. Exclusion Criteria: - History of HCT less than 2 years prior to enrollment; - Immune suppression therapy less than 1 year prior to enrollment; - Active graft versus host disease; - Current diagnosis or history of lymphoma or other malignancy; - Current active infection; - History of serious opportunistic infection (e.g., bacterial [Legionella and Listeria]; tuberculosis [TB]; invasive fungal infections; or viral, parasitic, and other opportunistic infections); - Positive TB skin test, positive Quantiferon-TB Gold TB test, positive chest X-ray, or a recent exposure to TB - Congestive heart failure defined by an ejection fracture <50% measured by ECHO; - Demyelinating disorders (e.g., central nervous system [CNS] disorders including multiple sclerosis and optic neuritis and peripheral nervous system disorders including Guillain-Barre syndrome); - Hematologic abnormalities (e.g., pancytopenia, aplastic anemia); - Hepatitis B infection (active or chronic carrier); - Latex sensitivity; - Pregnancy or breastfeeding; - Known or suspected allergy to adalimumab or related products; - Participation in simultaneous therapeutic study that involves an investigational study drug or agent within 4 weeks of study enrollment; - Requirement for live vaccine exposure that would be expected to occur during the time frame of the study; or - Any other social or medical condition that the Investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated or be detrimental to the study. |
Country | Name | City | State |
---|---|---|---|
United States | The Lundquist Institute at Harbor-UCLA Medical Center | Torrance | California |
Lead Sponsor | Collaborator |
---|---|
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Pain - 16 weeks | Mean difference in bodily pain measured by the Children's Health Questionnaire - Parent Form 50 (CHQ-PF50) or the Medical Outcomes Study - Short Form 36 (SF-36) in treatment versus placebo at 16 weeks | 16 weeks | |
Primary | Adalimumab trough | Percentage of subjects who achieve a goal trough concentration of adalimumab with every other week dosing | 32 weeks | |
Secondary | Joint range-of-motion - 16 weeks | Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion in treatment versus placebo at 16 weeks. | 16 weeks | |
Secondary | Pain - 52 weeks | Mean difference in bodily pain measured by the CHQ-PF50 or the SF-36 at 52 weeks compared to baseline. | 52 weeks | |
Secondary | Joint range-of-motion - 52 weeks | Percentage of subjects who achieve a 5 degree or more improvement in joint range-ot-motion at 52 weeks compared to baseline. | 52 weeks | |
Secondary | Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability | Percentage of subjects who develop an AE and/or SAE | 52 weeks |
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
|