Active not recruitingPHASE3INTERVENTIONAL

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

This research study is looking at a new medicine called Trappsol Cyclo for people aged 3 and over with Niemann-Pick Disease Type C1 (NPC1). The main goal is to find out if this medicine, given directly into the bloodstream every two weeks, is safe, well-tolerated, and helps reduce the effects of NPC1 more than the usual care. Some people will receive the new medicine, while others will receive a dummy treatment (placebo) alongside their usual care. Another part of the study is open to very young children (from birth up to 3 years old) to specifically check the medicine's safety in this age group. The study will last about 96 weeks, with an option to continue treatment after that.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Cyclo Therapeutics, Inc.

Enrolment target

Start

20 Jul 2021

Estimated completion

01 Jun 2026

Niemann-Pick Disease, Type C1

What is this study about?

This study is about a condition called Niemann-Pick Disease Type C1, often shortened to NPC1. It's a rare and serious genetic condition that affects how the body processes fats, leading to problems in many parts of the body, especially the brain. Currently, there isn't a cure, so researchers are always looking for new ways to help people living with NPC1.

This particular study is testing a medicine called Trappsol Cyclo. Doctors want to see if this medicine can help improve the symptoms of NPC1 or slow down its progression. They are especially interested in whether it's safe to use and if people can tolerate it well. For the main study, participants must be at least 3 years old. There's also a smaller, separate part of the study for very young children, right from birth up to 3 years old, to gather important safety information for this age group.

In the main study, some participants will receive Trappsol Cyclo, while others will receive a 'placebo'. A placebo looks just like the real medicine but contains no active ingredients. This helps researchers fairly compare the new medicine's effects. Everyone in the study will also continue to receive their standard medical care. By doing this carefully, researchers hope to learn if Trappsol Cyclo could be a helpful new treatment option for people with NPC1.

Key takeaways

This study is testing a new medicine, Trappsol Cyclo, for Niemann-Pick Disease Type C1 (NPC1).
It aims to find out if the medicine is safe, well-tolerated, and effective in improving NPC1 symptoms.
Participants aged 3 and over will either receive the medicine or a placebo, alongside their usual care.
There's a separate safety study for very young children (birth to 3 years).
The main treatment phase lasts about 96 weeks, with an optional extension.
Your safety and well-being are the top priorities throughout the study.

Who may be eligible?

To join the main part of this study, you usually need to be aged 3 or older and have a confirmed diagnosis of Niemann-Pick Disease Type C1 (NPC1). Your doctor will need to check how severe your condition is using a special score. You also need to weigh more than 4.5kg (about 10 pounds) and less than 125kg (about 275 pounds). You should have at least one symptom affecting your brain or nerves, and be able to travel to the study site for appointments.

If you are currently taking another medicine for NPC1 called Miglustat, you can still participate, but your dose must have been stable for at least 3 months. If you stopped taking Miglustat, it needs to have been at least 3 months since your last dose. Both you (if old enough) and your caregiver will need to agree to take part and follow all study instructions. There's also a separate part of the study for very young children (from birth up to 3 years old) with NPC1, even if they don't have symptoms yet, if they meet certain criteria.

You cannot join the study if you've had a liver transplant recently (within the last year) or are planning to have one. You also can't participate if you have active liver disease from another cause or severe kidney problems. Your study doctor will go through all the detailed requirements to make sure the study is a safe and suitable option for you.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Do I have a confirmed diagnosis of Niemann-Pick Disease Type C1?
Am I aged 3 years or older (or a very young child aged 0-3 for the sub-study)?
Do I weigh more than 4.5kg (about 10 pounds)?
Do I have at least one symptom affecting my brain or nerves (for the main study)?
Am I able to travel to the study site for regular visits?
Do I have any significant kidney or liver conditions not related to NPC1?

Answer every question to see your result.

What does participation involve?

If you join this study, you will receive either the investigational medicine Trappsol Cyclo or a placebo (a dummy treatment). This will be given through a drip into your vein (intravenously) every two weeks. These treatments are given in addition to your usual care for NPC1. The main treatment part of the study will last for 96 weeks, which is about 1 year and 10 months. However, the study involves regular visits to the trial site, where doctors will check your health, ask about your symptoms, and carry out various tests.

There will be checks at 48 weeks and 96 weeks to see how the treatment is working. After the initial 96 weeks, there's an option to continue receiving the active medicine in an 'open-label extension' for up to another 96 weeks, provided you meet certain criteria. This means everyone in the extension part would know they are receiving the actual medicine. If your condition worsens significantly during the study, you might have the option to switch to receiving the active medicine earlier.

Potential risks and benefits

Taking part in any clinical study has potential benefits and risks. The potential benefit is that Trappsol Cyclo might help improve your condition or slow down its progression, beyond what your usual care provides. However, this is not guaranteed, and the medicine might not work for everyone. There may also be side effects or risks associated with the medicine or the procedures involved, such as discomfort from intravenous infusions or regular blood tests. These will be explained in detail by the study team. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your future medical care. Your safety and well-being are the top priority throughout the study.

Locations (35)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UCSF Benioff Children's Hospital Oakland
Verified postcode
Oakland, United States
University of Florida
Verified postcode
Jacksonville, United States
Emory
Verified postcode
Atlanta, United States
Cincinnati Children's Hospital Medical Center
Verified postcode
Cincinnati, United States
UPMC Children's Hospital
Verified postcode
Pittsburgh, United States
University Utah
Verified postcode
Salt Lake City, United States
Lysosomal and Rare Disorders Research & Treatment Center, Inc.
Verified postcode
Fairfax, United States
Hospital de Alta Complejidad en Red "El Cruce"
Verified postcode
Buenos Aires, Argentina
Hospital de Niños de la Santísima Trinidad
Verified postcode
Córdoba, Argentina
Melbourne Children's Trials Centre Murdoch Children's Research Institute
Verified postcode
Parkville, Australia
Royal Melbourne Hospital
Verified postcode
Parkville, Australia
Metabolic Clinical Trials Unit
Verified postcode
Adelaide, Australia

Common questions

What is Niemann-Pick Disease Type C1 (NPC1)?

NPC1 is a rare genetic condition where the body struggles to process fats, leading to different health problems, mainly affecting the brain and nervous system.

What is Trappsol Cyclo?

Trappsol Cyclo is a new medicine being tested in this study. It's given through a drip into your vein to see if it can help people with NPC1.

Will I get the actual medicine or a placebo?

In the main study, participants are randomly assigned to receive either Trappsol Cyclo or a placebo (a dummy treatment). Neither you nor your doctors will know which you are receiving until the study treatment phase is over.

How long will I be in the study?

The main treatment part of the study lasts for about 96 weeks (just under two years). There's also an option to continue treatment in an 'open-label' extension for another 96 weeks.

Are very young children able to join?

Yes, there's a separate part of the study for children aged from birth up to 3 years old, specifically to look at the safety of Trappsol Cyclo in this age group.