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Study on the Safety and Effectiveness of Hydroxypropylbetadex and Miglustat for Patients with Niemann-Pick Disease Type C1

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What is this study about?

This clinical trial is focused on studying a rare genetic disorder called Niemann-Pick Disease Type C1. This disease affects the body’s ability to transport cholesterol and other fatty substances inside cells, which can lead to a variety of symptoms, including problems with movement, speech, and swallowing. The study will evaluate the safety and effectiveness of a treatment called Trappsol Cyclo, which contains the active substance hydroxypropylbetadex. This treatment is given through an intravenous infusion, which means it is administered directly into the bloodstream. The study will compare the effects of Trappsol Cyclo combined with standard care to a placebo combined with standard care.

Participants in the study will receive either Trappsol Cyclo or a placebo, along with the usual care for Niemann-Pick Disease Type C1. The study will last for a period of up to 96 weeks, during which participants will be monitored for any changes in their symptoms. The goal is to see if Trappsol Cyclo can improve the symptoms of the disease, such as movement, speech, and swallowing abilities. The study will also look at how well participants tolerate the treatment and any side effects they may experience.

In addition to Trappsol Cyclo, the study will also involve the use of Zavesca, a medication containing miglustat, which is taken orally in the form of hard capsules. Zavesca is already used in the treatment of Niemann-Pick Disease Type C1, and the study will consider its effects when used alongside Trappsol Cyclo. Participants who are already taking Zavesca will continue their treatment, while those who are not will be monitored for any changes in their condition. The study aims to provide valuable information on the potential benefits of Trappsol Cyclo in managing Niemann-Pick Disease Type C1.

1 initial visit

Upon joining the study, the patient will attend an initial visit. During this visit, the patient will undergo various assessments to establish a baseline for their health status. These assessments may include physical examinations, blood tests, and questionnaires to evaluate symptoms related to Niemann-Pick Disease Type C1.

2 randomization

After the initial assessments, the patient will be randomly assigned to one of two groups. One group will receive the study medication Trappsol Cyclo along with standard care, while the other group will receive a placebo with standard care. This process is called randomization and ensures that the study results are unbiased.

3 treatment phase

The patient will begin the treatment phase, which involves receiving the assigned medication. If assigned to the Trappsol Cyclo group, the patient will receive 2000 mg/kg of the medication through an intravenous infusion. The frequency and duration of the treatment will be determined by the study protocol and communicated to the patient by the study team.

In addition to the study medication, the patient may continue to receive standard care treatments, which could include medications like miglustat in the form of 100 mg hard capsules taken orally, if applicable.

4 follow-up visits

Throughout the study, the patient will attend regular follow-up visits. These visits are scheduled to monitor the patient’s health and the effects of the treatment. During these visits, the patient will undergo various assessments similar to those conducted during the initial visit.

The study will assess changes in the patient’s condition using specific scales, such as the 4-domain and 5-domain Niemann-Pick Disease Type C Severity Scale, to evaluate improvements in areas like ambulation, fine motor skills, speech, and swallowing.

5 final assessment

At the end of the study period, which may last up to 96 weeks, the patient will undergo a final assessment. This assessment will include a comprehensive evaluation of the patient’s health and any changes in their condition since the start of the study.

The results of this assessment will contribute to the overall analysis of the study’s effectiveness in treating Niemann-Pick Disease Type C1.

Who Can Join the Study?

  • Patients must be 3 years of age or older at the time of screening.
  • For those who are sexually active and can have children, effective birth control must be used during the study and for 3 months after the last dose of the study treatment. This includes methods like birth control pills, implants, IUDs, or having a partner who has had a vasectomy. Abstaining from sex is also an option if it is a consistent lifestyle choice.
  • The patient or their legally authorized representative must read and sign a consent form before any study-related procedures begin.
  • If applicable, the legally authorized representative must agree for the patient to participate in all parts of the study.
  • The patient’s caregiver, if applicable, must agree to participate in all assessments, questionnaires, and interviews required by the study.
  • The patient must have a confirmed diagnosis of Niemann Pick Disease Type C1 through genetic testing or specific medical tests.
  • Patients must have a specific score between 0.5 to 2.0 on a scale that measures the severity of Niemann-Pick Type C symptoms. Adjustments can be made for certain conditions like incontinence.
  • Patients can be either treated or not treated with a medication called miglustat. If treated, the dose must be stable for at least 3 months before screening. If not treated, they must have stopped taking it at least 3 months before screening.
  • Patients must have a body weight between 4.5 kg and 125 kg.
  • Patients must show at least one neurological symptom of the disease, such as hearing loss, difficulty with eye movement, balance issues, memory problems, muscle stiffness, seizures, sudden muscle weakness, speech difficulties, or trouble swallowing.
  • Patients must be willing and able to participate in all parts of the study, including giving blood samples and completing all required tests and assessments.
  • Patients who have previously been treated with a substance called hydroxypropyl-β-cyclodextrin (HPβCD) can join the study if their last treatment was at least 3 months ago for spinal injections or 6 months ago for IV treatments. Only a small percentage of participants can have had this treatment before.
  • Patients must be able to travel to the study site for scheduled visits and follow-up evaluations.

Who Cannot Join the Study?

  • Patients who have a different condition than Niemann Pick Disease Type C1.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial group.
  • Patients who are not able to follow the study procedures or assessments.
  • Patients who are not able to provide informed consent or do not have a legal representative to do so.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Bellvitge University Hospital L'hospitalet De Llobregat Spain

Other Sites

Site Name City Country Status
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania Catania Italy
Hospital Universitario 12 De Octubre Madrid Spain
SphinCS GmbH Hochheim Am Main Germany
Azienda Ospedaliera di Padova Padua Italy
Instytut Pomnik Centrum Zdrowia Dziecka Warsaw Poland
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Fondazione I.R.C.C.S. Istituto Neurologico Besta Milan Italy
Unkrgoqjiwowydwihgbyv Msrhdxif Axj Munster Germany

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Germany Germany
Not recruiting
06.07.2022
Italy Italy
Not recruiting
06.07.2022
Poland Poland
Not recruiting
06.07.2022
Spain Spain
Not recruiting
06.07.2022

Trial locations

Investigated drugs:

Trappsol Cyclo is a medication being studied for its potential to help people with Niemann-Pick Disease Type C1, a rare genetic disorder. This medication is designed to work by helping the body process and remove certain substances that can build up and cause damage in this disease. The goal of using Trappsol Cyclo in this study is to see if it can improve symptoms and overall health in patients when used alongside the standard treatments they are already receiving.

Investigated diseases:

Niemann-Pick Disease Type C1 – Niemann-Pick Disease Type C1 is a genetic disorder that affects the body’s ability to transport cholesterol and other lipids inside cells. This leads to an accumulation of these substances in various tissues, including the brain, liver, and spleen. The disease often begins in childhood and can cause a range of symptoms, such as difficulty with movement, speech, and swallowing. As the disease progresses, individuals may experience cognitive decline and problems with coordination. The severity and progression of symptoms can vary widely among affected individuals. The condition is considered a rare disease.

Trial ID:
2024-518266-27-00
Protocol code:
CTD-TCNPC-301
NCT ID:
NCT04860960
Trial Phase:
Therapeutic confirmatory (Phase III)

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