A Study of DNL310 in Pediatric Participants With Hunter Syndrome
Mucopolysaccharidosis II
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis II focused on measuring MPS II, Hunter Syndrome, nMPS II
Eligibility Criteria
Key Inclusion Criteria:
- Confirmed diagnosis of MPS II
- Cohort A: Participants aged ≥5 to ≤10 years with neuronopathic MPS II
- Cohort B: Participants aged ≥1 to ≤18 years with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype
- Cohort C: Participants aged <4 years with neuronopathic MPS II (this cohort can include participants ≥4 years of age if participant is a blood relative of a participant <4 years of age)
- Cohort D: Participants aged ≤18 years with non-neuronopathic MPS II or neuronopathic MPS II with preexisting hepatomegaly who have never taken standard-of-care ERT
- Cohort E: Participants aged ≥1 to ≤18 years who have completed at least 48 weeks in Study DNLI-E-0001
- For participants receiving intravenous iduronate 2-sulfatase (IDS) ERT, tolerated a minimum of 4 months of therapy during the period immediately prior to screening.
Key Exclusion Criteria:
- Unstable or poorly controlled medical condition(s) or significant medical or psychological comorbidity or comorbidities that, in the opinion of the investigator, would interfere with safe participation in the trial or interpretation of study assessments
- Use of any CNS-targeted MPS II ERT within 3 months before study start for participants aged ≥5 years, and within 6 months before study start for participants aged <5 years
- Use of IDS gene therapy or stem cell therapy at any time (except for participants in Cohort E)
- Clinically significant thrombocytopenia, other clinically significant coagulation abnormality, or significant active bleeding, or required treatment with an anticoagulant or more than two antiplatelet agents
- Contraindication for lumbar punctures
- Have a clinically significant history of stroke, status epilepticus, head trauma with loss of consciousness, or any CNS disease that is not MPS II-related within 1 year of screening
- Have had a ventriculoperitoneal (VP) shunt placed, or any other brain surgery, or have a clinically significant VP shunt malfunction within 30 days of screening
- Have any clinically significant CNS trauma or disorder that, in the opinion of the investigator, may interfere with assessment of study endpoints or make participation in the study unsafe
Sites / Locations
- UCSF Benioff Children's HospitalRecruiting
- Ann & Robert H. Lurie Children's Hospital of ChicagoRecruiting
- UNC Children's Research InstituteRecruiting
- UPMC | Children's Hospital of PittsburghRecruiting
- McGill University Health Centre - Royal Victoria HospitalRecruiting
- Erasmus Medical CenterRecruiting
- St Mary's Hospital, Manchester Academic Health Science CentreRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Experimental
Experimental
Experimental
Experimental
Experimental
Cohort A
Cohort B
Cohort C
Cohort D
Cohort E
Dose escalation followed by a consistent dose level in participants with neuronopathic MPS II
A consistent dose level in participants with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype followed by dose escalation in some participants.
A consistent dose level in participants with neuronopathic MPS II
A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II
A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II