A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C

Part of paid clinical trials in Rochester, Minnesota.

Sponsor
IntraBio Inc
Study ID
NCT05163288
Phase
PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Niemann-Pick Disease, Type C

Eligibility Criteria

Sex
ALL
Age
4 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • N-Acetyl-L-Leucine — DRUG
    N-Acetyl-L-Leucine is a modified amino-acid ester that is orally administered (granules for suspension in a sachet)
  • Placebo — OTHER
    Matching Placebo Sachet

Study Details

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.

Key Dates

Start date
Jun 30, 2022
Status verified
Mar 2026
Primary completion
Jun 12, 2030
Completion
Dec 31, 2030

Study Design

Enrollment
53 participants (estimated)
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT

Arms

  • Experimental: N-acetyl-L-leucine (IB1001)
    Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day); patients \<13 will receive weight-tiered doses.
  • Placebo Comparator: Placebo comparator
    Oral administration (granule in a sachet for suspension in water, orange juice, or almond milk). Patients ≥13 years old will receive a total daily dose of 4 g/day (administered as 3 doses per day); patients \<13 will receive weight-tiered doses.

Primary Outcome Measure

Scale for the Assessment and Rating of Ataxia (all jurisdictions except US) [ Time Frame: End of Period I (week 12) vs. End of Period 2 (week 24) ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Mayo ClinicRochesterMinnesota55905-

Find similar trials in Rochester, MN

By research site
Mayo Clinic· Rochester, MN

Related Studies