A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
- Conditions
- Mucopolysaccharidosis II
- Registration Number
- NCT04007536
- Lead Sponsor
- Denali Therapeutics Inc.
- Brief Summary
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.
- Detailed Description
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.).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 18
Not provided
Not provided
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Changes in levels of total urine glycosaminoglycans (GAGs), levels of heparan sulfate (HS) and dermatan sulfate (DS) in cerebrospinal fluid (CSF), urine and/or blood up to 96 weeks Changes in adaptive behavior over time as measured by Vineland Adaptive Behavior Scales, Second Edition (VABS II) and/or Vineland Adaptive Behavior Scales, Third Edition (Vineland-3) Up to 96 weeks Changes in neurocognition over time as measured by Bayley Scales of Infant and Toddler Development, 3rd Edition; Kaufman Assessment Battery for Children, 2nd Edition; or Wechsler Intelligence Scale for Children, Fifth Edition Up to 96 weeks
- Secondary Outcome Measures
Name Time Method
Trial Locations
- Locations (6)
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
UNC Children's Research Institute
🇺🇸Chapel Hill, North Carolina, United States
UPMC | Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Erasmus Medical Center
🇳🇱Rotterdam, South Holland, Netherlands
Birmingham Children's Hospital
🇬🇧Birmingham, United Kingdom
Manchester Centre for Genomic Medicine
🇬🇧Manchester, United Kingdom
UCSF Benioff Children's Hospital🇺🇸Oakland, California, United States