N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC) (NCT03759639) | Clinical Trial Compass
CompletedPhase 2
N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
United States, Germany, Slovakia33 participantsStarted 2019-09-04
Plain-language summary
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Niemann-Pick type C disease (NPC).
There are two phases to this study: the Parent Study, and the Extension Phase.
The Parent Study evaluates the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the symptomatic treatment of NPC.
The Extension Phase evaluates the long-term safety and efficacy of IB1001 for the neuroprotective, disease-modifying treatment of NPC.
Who can participate
Age range6 Years
SexALL
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Inclusion criteria
✓. Written informed consent signed by the patient and/or their legal representative/ parent
✓. Male or female aged ≥6 years in Europe OR ≥18 years in the United States with a confirmed diagnosis of NPC at the time of signing informed consent. Patients must have clinical features of NPC and a positive genetic test for mutations in both copies of NPC1 or in both copies of NPC2.
✓. 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 continuing 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);
✓. 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
✕. Asymptomatic patients
✕. Patient has clinical features of NPC and a positive biomarker screen and/or filipin test, but a negative result on a previous genetic test for NPC
What they're measuring
1
Clinical Impression of Change in Severity (CI-CS) [Fields et al 2021]
Timeframe: CI-CS comparing Baseline (Day 1) with IB1001 versus the end of 6-weeks treatment with IB1001 (Approximately Day 42) MINUS the CI-CS comparing the end of 6-weeks treatment with IB1001 (Approximately Day 42) versus the end of 6-weeks post-treatment washout