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A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL310 in Pediatric Participants with Hunter Syndrome
EU CTIS ID: 2023-508619-22-00
What this study is testing
To characterize the safety and tolerability of DNL310 in pediatric participants with MPS II (all cohorts)
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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, neuronopathic MPSII or unknown phenotype
- Cohort C:Participants aged < 4 years with neuronopathic MPS II (this cohort can include participants ≥4 to ≤18 years of age if the participant is a blood relative with the same genetic mutation as a participant aged < 4 years who will be enrolled in the study)
- Cohort D: Participants aged ≤ 18 years, with nMPS II and nnMPS II and preexisting hepatomegaly, who have never taken standard-of-care ERT
- Cohort E: neuronopathic MPS II participants aged ≥6 years at screening, non-neuronopathic MPS II participants <6 or ≥17 years at screening, and neuronopathic MPS II participants ≥1 to ≤18 years at screening with a history of prior haematopoietic stem cell transplantation or gene therapy who have completed at least 48 weeks in Study DNLI-E-0001
You likely can't join if
- 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
See the full eligibility criteria
Who can join
- 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, neuronopathic MPSII or unknown phenotype
- Cohort C:Participants aged < 4 years with neuronopathic MPS II (this cohort can include participants ≥4 to ≤18 years of age if the participant is a blood relative with the same genetic mutation as a participant aged < 4 years who will be enrolled in the study)
- Cohort D: Participants aged ≤ 18 years, with nMPS II and nnMPS II and preexisting hepatomegaly, who have never taken standard-of-care ERT
- Cohort E: neuronopathic MPS II participants aged ≥6 years at screening, non-neuronopathic MPS II participants <6 or ≥17 years at screening, and neuronopathic MPS II participants ≥1 to ≤18 years at screening with a history of prior haematopoietic stem cell transplantation or gene therapy 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.
What rules you out
- 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
- Have clinically significant anemia
The study team makes the final eligibility decision.
Where it's taking place
- United States
- United Kingdom
- Canada
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States; United Kingdom; Canada. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.