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Enzyme Replacement Therapy
Tividenofusp Alfa for Hunter Syndrome
Phase 1 & 2
Recruiting
Research Sponsored by Denali Therapeutics Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Confirmed diagnosis of MPS II
Cohort B: Participants aged ≥1 to ≤18 years with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 weeks, 104 weeks, and 261 weeks
Awards & highlights
Study Summary
This trial is for a new treatment for MPS II, a disease that affects both the peripheral and central nervous system. The goal is to see if it is safe and effective. People who benefit from the treatment will have the opportunity to continue in the trial for further evaluation.
Who is the study for?
This trial is for children with Hunter Syndrome (MPS II), including various subgroups based on age, whether they have neuronopathic or non-neuronopathic forms, and their treatment history. It's not for those who've had certain brain surgeries or conditions, significant bleeding disorders, recent CNS-targeted treatments outside specified windows, or contraindications to lumbar punctures.Check my eligibility
What is being tested?
The study tests Tividenofusp Alfa (DNL310), a new enzyme replacement therapy that could treat both body and brain symptoms of MPS II. The trial will monitor safety and how the drug works in the body over time. Participants benefiting may continue in extended phases for ongoing evaluation.See study design
What are the potential side effects?
While specific side effects are not listed here, common ones from similar therapies include allergic reactions at infusion sites, headaches, joint pain, fever and chills. As this is an investigational drug assessing safety is a primary goal of this study.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with MPS II.
Select...
I am between 1 and 18 years old with a type of MPS II.
Select...
I have been on IDS ERT treatment for at least 4 months without issues.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 24 weeks, 104 weeks, and 261 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 weeks, 104 weeks, and 261 weeks
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from baseline in concomitant medications
Change from baseline in urine total glycosaminoglycan (GAG) concentrations
Incidence and severity of infusion-related reactions (IRRs)
+1 moreSecondary outcome measures
Characterization of immunogenicity of DNL310 in serum, as measured by the incidence of anti-drug antibodies (ADAs) relative to baseline
PK parameter: Apparent terminal elimination half-life (t½) of DNL310 in serum
PK parameter: Area under the concentration-time curve from time zero to infinity (AUC∞) of DNL310 in serum
+11 moreTrial Design
5Treatment groups
Experimental Treatment
Group I: Cohort EExperimental Treatment1 Intervention
A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II
Group II: Cohort DExperimental Treatment1 Intervention
A consistent dose level in participants with non-neuronopathic MPS II or neuronopathic MPS II
Group III: Cohort CExperimental Treatment1 Intervention
A consistent dose level in participants with neuronopathic MPS II
Group IV: Cohort BExperimental Treatment1 Intervention
A consistent dose level in participants with non-neuronopathic MPS II, neuronopathic MPS II, or unknown phenotype followed by dose escalation in some participants.
Group V: Cohort AExperimental Treatment1 Intervention
Dose escalation followed by a consistent dose level in participants with neuronopathic MPS II
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Who is running the clinical trial?
Denali Therapeutics Inc.Lead Sponsor
22 Previous Clinical Trials
1,822 Total Patients Enrolled
Katia Meirelles, MDStudy DirectorDenali Therapeutics
Anna Bakardjiev, MDStudy DirectorDenali Therapeutics
1 Previous Clinical Trials
18 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I don't have serious blood clotting issues or ongoing major bleeding.I haven't used CNS-targeted MPS II ERT recently.I have not used IDS gene or stem cell therapy before.I haven't had a stroke, severe head injury, or serious brain condition unrelated to MPS II in the last year.I am over 6 with neuronopathic MPS II, under 6 or over 17 with non-neuronopathic MPS II, or had specific treatments for MPS II.I have been diagnosed with MPS II.I do not have any brain disorders that could affect my study participation.I have had brain surgery or a VP shunt placed/malfunctioned recently.I am between 1 and 18 years old with a type of MPS II.I am between 5 and 10 years old with neuronopathic MPS II.I have been on IDS ERT treatment for at least 4 months without issues.I am under 4 with neuronopathic MPS II or a relative aged 4-18 of someone who is.I am 18 or younger with MPS II and have not started standard ERT.I cannot have lumbar punctures due to health risks.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort E
- Group 2: Cohort D
- Group 3: Cohort C
- Group 4: Cohort B
- Group 5: Cohort A
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
How many individuals are being included in this clinical experiment?
"To complete this trial, Denali Therapeutics Inc. will need to recruit 45 participants that meet the criteria for inclusion in the study from UNC Children's Research Institute and UPMC | Children's Hospital of Pittsburgh."
Answered by AI
Are participants of this clinical investigation limited to those 18 or older?
"The specified age range for this trial is 2 to 18 years old, based on the given inclusion criteria."
Answered by AI
Who is enabled to participate in this research endeavor?
"Patients aged between two and eighteen, with a confirmed diagnosis of mucopolysaccharidosis ii, can sign up for this clinical trial. The overall recruitment target is 45 participants."
Answered by AI
Are there currently any opportunities for patients to join this experiment?
"The clinical trial is still seeking participants, as evidenced by its listing on clinicaltrials.gov; it was listed initially on 16th July 2020 and modified most recently on 14th November 2022."
Answered by AI
How extensive is the scope of this research trial across medical centers?
"This medical research is currently taking place at 5 centres situated in Pittsburgh, Chicago, Oakland and other locales. To ease burden on patients during the trial period, it is advised to select a clinic that can be reached with minimal effort."
Answered by AI
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