Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With … (NCT04860960) | Clinical Trial Compass
Active — Not RecruitingPhase 3
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
United States, Argentina, Australia94 participantsStarted 2021-07-20
Plain-language summary
A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of Niemann Pick disease type C1 (NPC1). The objective of this study is to evaluate the safety, tolerability and efficacy of 2000 mg/kg dose of Trappsol Cyclo (hydroxypropyl betacyclodextrin) administered intravenously compared to standard of care. An open-label sub-study in countries following European Medicines Agency (EMA) guidance will enroll asymptomatic or symptomatic patients from infancy up to age 3 to evaluate safety in that population.
Who can participate
Age range
3 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Confirmed diagnosis of NPC1
. Annual Severity Increment Score between 0.5 and 2.0 using the 17-domain NPC Severity Scale
. Treated or Not Treated with Miglustat (patients must be on a stable dose for at least 3 months prior to the Screening Visit, or have discontinued Miglustat for at least 3 months prior to Screening Visit).
. Body weight greater than 4.5 kg and less than or equal to 125 kg
. Presenting at least 1 neurological symptom of the disease
. Written informed consent
. Willing and capable to participate in all aspects of trial design
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change from Baseline in 4-Domain NPC Severity Score (US only)
Timeframe: Interim Analysis at Week 48
2
Change from Baseline in 4-Domain NPC Severity Score (US only)
Timeframe: End of Study at Week 96
3
Change from Baseline in 5-Domain NPC Severity Score (ex-US)
Timeframe: Interim Analysis at Week 48
4
Change from Baseline in 5-Domain NPC Severity Score (ex-US)
. Ability to travel to the trial site at scheduled times
Exclusion criteria
. Recipient of a liver transplant within \<12 months or planned liver transplantation
. Patients with active liver disease from any cause other than NPC1
. Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or international normalized ratio \> 1.8
. Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate \<60ml/min/1.73m2.
. Use of curcumin or fish oil within 12 weeks prior to enrollment
. Known or suspected allergy or intolerance to the study treatment
. In the opinion of the Investigator, the patient's clinical condition does not allow for the blood collection required as per protocol specific procedures.
. Treatment with any investigational drug during the 3 months prior to entering the study. If the investigational drug has a short half-life (\<8 hours) and would be expected to be cleared from the body within 1 month, then the wash-out period is 1 month. Treatment with any form of leucine, whether as an investigational drug or other formulation is not allowed