A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C (NCT05163288) | Clinical Trial Compass
RecruitingPhase 3
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
United States, Australia, Czechia53 participantsStarted 2022-06-30
Plain-language summary
A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care.
Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.
Who can participate
Age range
4 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
. Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness
. Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of signing informed consent.
. Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose and confirm to continue through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in \<1% failure rate when used consistently and correctly) 14 days prior to the first dose continuing through 28 days after the last dose:
. intrauterine device (IUD);
. surgical sterilization of the partner (vasectomy for 6 months minimum);
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
Scale for the Assessment and Rating of Ataxia (all jurisdictions except US)
Timeframe: End of Period I (week 12) vs. End of Period 2 (week 24)
2
Modified Scale for the Assessment and Rating of Ataxia (US only)
Timeframe: End of Period I (week 12) vs. End of Period 2 (week 24)
. combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);
. progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);
. intrauterine hormone releasing system (IUS);
Exclusion criteria
. Patients who are unable to consistently Patients who have any known hypersensitivity or history of hypersensitivity to:
. Acetyl-Leucine (DL-, L-, D-) or derivatives.
. Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry flavour).
. Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product (IMP; 'study drug') for at least 42 days prior to Visit 1. At the discretion of the investigator, Medical Monitor, and Sponsor, the washout period for specific IMPs may be longer based on the pharmacological activity and pharmacokinetics of the drug.
. Patients with a physical, cognitive, or psychiatric condition which, at the investigator's discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient's participation in the clinical study, i.e. reliably perform study assessments.
. Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.
. Current or planned pregnancy or women who are breastfeeding.