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Enrolling by invitationPHASE2, PHASE3INTERVENTIONAL

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

This study is an extension of previous research, focusing on the long-term safety, how well it's tolerated, and the effectiveness of a medicine called tividenofusp alfa. This medicine is for children with a rare condition called Hunter syndrome, also known as Mucopolysaccharidosis Type II (MPS II). It's given through an IV (into a vein) and aims to help the brain and spinal cord. If you've already been part of related studies (DNLI-E-0002 or DNLI-E-0007) and meet certain conditions, you might be able to join this study. Participants will receive the medicine for up to 5 years, and for some, the original treatment they received in a previous study will remain unknown to everyone involved, ensuring fair evaluation.

At a glance

Status
Enrolling by invitation
Phase
PHASE2, PHASE3
Sponsor
Denali Therapeutics Inc.
Enrolment target
99
Start
20 Sep 2023
Estimated completion
01 Jun 2027
Mucopolysaccharidosis II

What is this study about?

This study is an important next step for children living with Hunter syndrome, also known as Mucopolysaccharidosis Type II (MPS II). MPS II is a rare genetic condition that can affect many parts of the body, including the brain and spinal cord. Currently, treatments are available, but doctors are always looking for ways to improve care and find new options.

This particular study is testing a new investigational medicine called tividenofusp alfa (also known as DNL310). It's given through a drip into a vein (IV) and is designed to reach the brain and spinal cord. The main goal is to see how safe this medicine is over a longer period and how well children tolerate it. Researchers also want to understand how effective it is in managing the symptoms of Hunter syndrome long-term.

Because this is an 'extension study,' it's specifically for children who have already taken part in similar research studies (DNLI-E-0002 or DNLI-E-0007). By continuing to monitor these participants, researchers can gather valuable information about the medicine's long-term effects, which is crucial for helping to develop new treatments.

Key takeaways

  • This study is for children with Hunter syndrome (MPS II).
  • It's testing a new medicine called tividenofusp alfa given by IV.
  • The main goal is to check its long-term safety and how well it works.
  • Only children who completed previous studies (DNLI-E-0002 or DNLI-E-0007) can join.
  • Participation could last up to 5 years, with regular clinic visits.
  • You can stop participating at any time.

Who may be eligible?

This study is for children who have already taken part in one of two previous research studies for Hunter syndrome (MPS II): Study DNLI-E-0002 or Study DNLI-E-0007.

If you were in Study DNLI-E-0002, you would need to have completed at least 49 weeks of that study and not have stopped the treatment early. If you were in Study DNLI-E-0007, you would need to have finished the entire treatment period in that study.

You would not be able to join this study if you have any serious or uncontrolled medical issues, or other health problems that the study doctors believe would make it unsafe for you to participate or would make it difficult to properly understand the study results.

Quick self-check
  • Have you or your child been diagnosed with Hunter syndrome (MPS II)?
  • Did you or your child participate in study DNLI-E-0002 and complete at least 49 weeks without stopping early?
  • OR, did you or your child participate in study DNLI-E-0007 and complete the entire treatment period?
  • Do you or your child have any uncontrolled medical conditions or other health issues that a doctor might consider too significant for study participation?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you join this study, you will receive the study medicine, tividenofusp alfa, for up to 5 years. The medicine is given through an IV (intravenously), meaning it will be delivered directly into your bloodstream, likely at a hospital or clinic. Throughout this period, you will have regular visits to the study site. During these visits, the study team will carefully check your health, how you are feeling, and any effects the medicine might be having. They will perform various assessments to monitor the safety of the treatment and see how well it's working. These assessments might include physical exams, blood tests, and other procedures relevant to Hunter syndrome. The exact schedule of visits and tests will be explained in detail by the study team.

Potential risks and benefits

Taking part in any study has potential benefits and risks. The main potential benefit of this study is continued access to the investigational medicine tividenofusp alfa, which aims to help with Hunter syndrome. It contributes to understanding long-term safety and effectiveness, possibly leading to a new approved treatment. Potential risks could include side effects from the medicine, discomfort from IV infusions, or the time commitment for study visits. It's important to remember that you can stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (27)

  • UCSF Benioff Children's Hospital Oakland
    Oakland, United States
  • UNC Children's Research Institute
    Chapel Hill, United States
  • Cincinnati Children's Hospital Medical Center
    Cincinnati, United States
  • Children's Hospital of Philadelphia
    Philadelphia, United States
  • The University of Texas Medical School at Houston
    Houston, United States
  • Huntsman Cancer Hospital
    Salt Lake City, United States
  • Sanatorio Mater Dei
    Buenos Aires, Argentina
  • Universitair Ziekenhuis Antwerpen
    Edegem, Belgium
  • UZ Brussel
    Jette, Belgium
  • University of Alberta - Faculty of Medicine & Dentistry
    Edmonton, Canada
  • Hospital for Sick Children
    Toronto, Canada
  • McGill University Health Center
    Montreal, Canada

+15 more sites — see the official record for the full list.

Common questions

What is Hunter syndrome (MPS II)?

Hunter syndrome is a rare genetic condition where the body can't break down certain complex sugars, leading to their build-up and causing various health problems, including developmental challenges.

What is tividenofusp alfa?

Tividenofusp alfa is an investigational medicine being tested in this study. It's designed to help the body process the substances that build up in Hunter syndrome, especially in the brain and spinal cord.

How is the medicine given?

The medicine is given through a drip into a vein, which is called an intravenous (IV) infusion.

How long will I be in this study?

If you join, you could receive the study medicine and be monitored for up to 5 years.

Can I join if I haven't been in a previous study?

No, this study is an extension and is only for children who have already participated in specific earlier studies (DNLI-E-0002 or DNLI-E-0007).

How to find out more

Official trial record

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "An Extension Study of the Long-Term Safety, Tolerability, an…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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