Actively Recruiting
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Led by IntraBio Inc · Updated on 2026-04-03
53
Participants Needed
14
Research Sites
439 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent signed by the patient or legal representative/parent/witness
- Male or female aged 4 years or older with confirmed genetic diagnosis of NPC at consent
- Females of childbearing potential must be sexually inactive or use highly effective contraception before and during the study
- Females of non-childbearing potential must have undergone sterilization or be postmenopausal with appropriate hormone levels
- Non-vasectomized males agree to use condom with spermicide or abstain from sex during study and 90 days after last dose; vasectomized males must use condom if vasectomy was under 6 months ago
- Males agree not to donate sperm from first dose until 90 days after last dose
- SARA score between 7 and 34 points; must meet gait subtest or 9-Hole Peg Test time criteria
- Weight at least 15 kg at screening
- Willingness to disclose existing medications/therapies for NPC symptoms and maintain stable dose/type for at least 42 days before baseline and during study
- Understanding and willingness to comply with study participation and visits
You will not qualify if you...
- Known hypersensitivity to Acetyl-Leucine or excipients in study or placebo sachets
- Participation in another clinical study with investigational drug within 42 days prior to baseline
- Physical, cognitive, or psychiatric condition interfering with study participation or assessment
- Known or persistent misuse or dependency on medication, drugs, or alcohol
- Current or planned pregnancy or breastfeeding
- Severe vision or hearing impairment not corrected by aids affecting ability to perform assessments
- Diagnosed arthritis or musculoskeletal disorders affecting mobility and assessment
- Use of prohibited medications (N-Acetyl-DL-Leucine, N-Acetyl-L-Leucine outside study, Sulfasalazine, Rosuvastatin) within 42 days before baseline and during study
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 14 locations
1
Mayo Clinic
Rochester, Minnesota, United States, 55905
Completed
2
The Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Active, Not Recruiting
3
First Faculty of Medicine, Charles University Hospital Prague
Prague, Czechia, 128 08
Active, Not Recruiting
4
University of Giessen
Giessen, Germany, 35389
Active, Not Recruiting
5
University of Hamburg
Hamburg, Germany, 20246
Completed
6
SphinCS - Institute of Clinical Science in Lysosomal Storage Disorders
Höchheim, Germany, 65239
Active, Not Recruiting
7
Ludwig Maximilian University of Munich
München, Germany, 80539
Not Yet Recruiting
8
University Hospital Münster
Münster, Germany, 48149
Active, Not Recruiting
9
Amsterdam UMC
Amsterdam, Netherlands, 1105
Actively Recruiting
10
Comenius University in Bratislva
Bratislava, Slovakia, 833 40
Active, Not Recruiting
11
University Hospital Bern Inselspital
Bern, Switzerland, 3010
Active, Not Recruiting
12
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Active, Not Recruiting
13
Royal Free London NHS Foundation Trust
London, United Kingdom
Active, Not Recruiting
14
Royal Manchester Children's Hospital
Manchester, United Kingdom, M13 9WL
Active, Not Recruiting
Research Team
M
Michael Strupp, MD
CONTACT
T
Taylor Fields
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
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