Information for families and the NPC community
We know that people living with Niemann-Pick disease type C (NPC), and their families, want to be kept informed about research and potential new treatment opportunities.
We would therefore like to share information about NAVIGATE, a clinical trial that is currently recruiting children and adults with NPC.
Please note: This information is intended to help families understand the NAVIGATE study and decide whether they would like to explore it further. It is not a recommendation to take part in the trial. Eligibility can only be assessed by the clinical trial team.
What is the NAVIGATE study?
NAVIGATE is a clinical research study investigating a new medicine called nizubaglustat.
The study aims to understand whether nizubaglustat is safe and whether it may help people living with NPC.
The study plans to include around 72 people with NPC from different countries.
How does the study work?
People taking part in NAVIGATE will be randomly assigned to receive either:
- Nizubaglustat, the study medicine; or
- A placebo, a treatment that does not contain the active medicine.
For every three people who take part:
- Two people will receive nizubaglustat
- One person will receive the placebo
During the main part of the study, neither participants and their families nor the study team will know which treatment an individual is receiving. This is known as a blinded clinical trial.
At the end of the study, participants may be offered the opportunity to continue into an extension phase, where everyone would receive nizubaglustat.
What would taking part involve?
The study lasts for around 78 weeks, or approximately 18 months.
During this time, participants will attend approximately eight clinic visits. At these visits, the study team will carry out a range of tests and assessments to monitor participants and assess how they are responding to the study treatment.
Before anyone decides whether to take part, the clinical trial team will explain what participation involves. This will include information about:
- The study visits
- Tests and assessments
- Possible risks and side effects
- Potential benefits
- What taking part would mean for the participant and their family
The clinical trial team will also answer any questions you may have so that you have the information you need before making a decision.
Who may be able to take part?
To be considered for the study, participants must:
- Be 4 years of age or older
- Have a genetically confirmed diagnosis of NPC
- Have a late-infantile or juvenile form of NPC, meaning their first neurological symptoms appeared between 2 and 15 years of age
For children and young people under 18, a parent or legal guardian will need to provide consent for them to take part.
Children and young people may also be asked to provide their own agreement to participate (known as assent), depending on their age and understanding.
There are also other criteria that the clinical trial team will consider. For example, a clinician will assess how the condition is progressing before deciding whether someone is eligible to take part.
Meeting the criteria listed above does not necessarily mean that someone will be eligible for the study. The clinical trial team will assess each potential participant individually.
Important information about current treatments
Nizubaglustat is taken once a day as a tablet that dissolves in water.
Nizubaglustat and miglustat
Nizubaglustat cannot be taken at the same time as miglustat.
If you or your family member currently takes miglustat, or any other medication, and you are interested in exploring the NAVIGATE study, please speak to your specialist clinical team.
Do not stop, change or adjust any current medication without first discussing this with your clinical team.
Your specialist team can advise you about your current treatment and whether it may be appropriate to explore participation in the NAVIGATE study.
What happens if I am interested in taking part?
If you are interested in finding out whether NAVIGATE may be suitable for you or your family member, the first step is to speak to your specialist NPC clinical team.
They can:
- Discuss the study with you
- Answer questions about your individual circumstances
- Advise whether it may be appropriate to explore the study further
- Explain the next steps
- Discuss referral to a participating study centre where appropriate
The clinical trial team will then be able to provide detailed information about the study and assess whether the participant meets the full eligibility criteria.
Deciding whether to take part
Taking part in a clinical trial is a very personal decision.
Some people and families may feel that NAVIGATE is an opportunity they would like to explore, while others may decide that participating in a clinical trial is not right for them.
There is no obligation to take part.
NPUK’s role is to ensure that our community is aware of opportunities to participate in research and has access to clear information to help people make an informed decision.
We encourage anyone considering the study to discuss it with their specialist clinical team and the clinical trial team, who can provide information specific to their circumstances.
Where can I find more information?
Further information about the NAVIGATE study is available online:
ClinicalTrials.gov
Study information and details about the clinical trial can be found on ClinicalTrials.gov.
NAVIGATE Study Website
You can also find further information about the study on the official NAVIGATE study website.
Questions or support
If you have questions about NAVIGATE, please do not hesitate to contact the NPUK team.
We understand that clinical trials can raise lots of questions, and we are always here to support our community and help you find the information you need.
For questions about whether you or your family member may be eligible to participate, please speak directly with your specialist NPC clinical team, who can discuss your individual circumstances and advise on the appropriate next steps.
With best wishes,
The NPUK Team