A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
- Conditions
- Mucopolysaccharidosis II
- Interventions
- Other: No Intervention
- 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
- Arm && Interventions
Group Intervention Description Part 3 No Intervention Participants \<8 years of age who have the neuronopathic form of mucopolysaccharidosis type II (nMPS II). Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 6 No Intervention Participants from 1 to 17 years of age with nMPS II. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 6 will also include a single collection of CSF. Part 4 No Intervention Participants 6 to 17 years of age with the non-neuronopathic form of mucopolysaccharidosis type II (nnMPS II). Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 1 No Intervention Participants from 2 through 10 years of age who have MPS II. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 5 No Intervention Participants ≤3 years of age with an undetermined MPS II phenotype. Clinical, neurocognitive, laboratory, and biomarker assessments will be conducted. Part 2 No Intervention Participants from 2 through 30 years of age who have MPS II; Part 2 will entail a single collection of cerebrospinal fluid (CSF), urine, and blood. Clinical outcome assessments are optional in Part 2 for participants aged 18 years or younger; no clinical assessments are planned for participants older than 18 years.
- 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)
UPMC | Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
UCSF Benioff Children's Hospital
🇺🇸Oakland, California, United States
Birmingham Children's Hospital
🇬🇧Birmingham, United Kingdom
Manchester Centre for Genomic Medicine
🇬🇧Manchester, United Kingdom
UNC Children's Research Institute
🇺🇸Chapel Hill, North Carolina, United States
Erasmus Medical Center
🇳🇱Rotterdam, South Holland, Netherlands