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Enzyme Replacement Therapy

Tividenofusp Alfa for Hunter Syndrome

Q: Does this research endeavor presently have the capacity to accept participants?

<p>Affirmative. Clinicaltrials.gov indicates that this trial is in the process of recruiting participants, having been first posted on September 20th 2023 and last updated on October 3rd 2023. It requires 99 enrollees from a single centre to be completed.</p>

Q: How many individuals have been approved for participation in this clinical experiment?

<p>Affirmative. As stated on clinicaltrials.gov, this research is actively seeking out participants who can take part in the study. It was first posted on September 20th 2023 and the most recent edit was made October 3rd of that year. The trial requires 99 people to be recruited from one location.</p>

Phase 2 & 3

Waitlist Available

Research Sponsored by Denali Therapeutics Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 5 years

Awards & highlights

Study Summary

This trial will study the safety, tolerability, and efficacy of an investigational therapy for Hunter Syndrome (MPS II) for up to 5 years. Participants, site staff, & the Sponsor remain blinded to the treatment assignment.

Who is the study for?

This trial is for people with Hunter syndrome (MPS II) who have already been part of earlier studies DNLI-E-0002 or DNLI-E-0007. They must have completed the required weeks in those studies without early discontinuation and should not have any unstable medical conditions that could affect their safety or the study results.Check my eligibility

What is being tested?

The trial is testing Tividenofusp Alfa, a new IV therapy designed to treat Hunter syndrome by reaching the central nervous system. Participants will receive this treatment for up to five years to evaluate its long-term safety and effectiveness.See study design

What are the potential side effects?

As an investigational drug, specific side effects of Tividenofusp Alfa are not detailed here but may include reactions related to enzyme replacement therapies such as allergic reactions, infusion-related responses, and potential immune system impacts.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Clinically significant changes in urine total glycosaminoglycan (GAG) concentrations throughout the treatment period

Incidence and intensity of infusion-related reactions (IRRs)

Incidence and intensity of treatment-emergent adverse events (TEAEs)

Secondary outcome measures

Change from baseline in distance walked (meters) in the Six-Minute Walk Test (6MWT)

Change from baseline in the Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) cognitive raw score

Change from baseline in the Vineland-3 Adaptive Behavior Scale

+5 more

Trial Design

7Treatment groups

Experimental Treatment

Group I: Cohort E2Experimental Treatment1 Intervention

Participants with nMPS II, aged ≥6 years; participants with nnMPS II, aged <6 or ≥17 years; or participants with nMPS II, aged ≥1 to ≤18 years, with a history of prior HSCT or gene therapy and have completed at least 48 weeks in Study DNLI-E-0001

Group II: Cohort D2Experimental Treatment1 Intervention

Participants with nMPS II or nnMPS II, aged ≤18 years with preexisting hepatomegaly who have never taken standard-of-care ERT

Group III: Cohort C2Experimental Treatment1 Intervention

Participants with nMPS II, aged <4 years

Group IV: Cohort B7Experimental Treatment1 Intervention

Participants with nnMPS II, aged ≥6 to <17 years

Group V: Cohort B2Experimental Treatment1 Intervention

Participants with nMPS II or nnMPS II, aged ≥1 to ≤18 years

Group VI: Cohort A7Experimental Treatment1 Intervention

Participants with nMPS II, aged ≥2 to <6 years

Group VII: Cohort A2Experimental Treatment1 Intervention

Participants with nMPS II, aged ≥5 to ≤10 years

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Denali Therapeutics Inc.Lead Sponsor

22 Previous Clinical Trials

1,768 Total Patients Enrolled

Jose Alcantara Rodriguez, PharmDStudy DirectorDenali Therapeutics

1 Previous Clinical Trials

54 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Does this research endeavor presently have the capacity to accept participants?

"Affirmative. Clinicaltrials.gov indicates that this trial is in the process of recruiting participants, having been first posted on September 20th 2023 and last updated on October 3rd 2023. It requires 99 enrollees from a single centre to be completed."

Answered by AI

How many individuals have been approved for participation in this clinical experiment?

"Affirmative. As stated on clinicaltrials.gov, this research is actively seeking out participants who can take part in the study. It was first posted on September 20th 2023 and the most recent edit was made October 3rd of that year. The trial requires 99 people to be recruited from one location."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

2023. "An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06075537.