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Niemann-Pick Disease, Type C

5
Pipeline Programs
3
Companies
6
Clinical Trials
3 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
3
1
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

IntraBio
IntraBioTX - Austin
2 programs
1
1
N-Acetyl-L-LeucinePhase 31 trial
IB1001Phase 21 trial
Active Trials
NCT03759639Completed33Est. Nov 2022
NCT05163288Recruiting53Est. Dec 2030
Human BioSciences
Human BioSciencesWV - Martinsburg
4 programs
2
N-Acetyl CysteinePhase 1/2
VTS-270Phase 1/21 trial
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type CN/A1 trial
Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type CN/A1 trial
Active Trials
NCT05588167Recruiting100Est. Sep 2026
NCT00344331Recruiting900
NCT03471143Completed4Est. Oct 2024
Genome & Company
Genome & CompanyKorea - Suwon
1 program
1
N-Acetyl CysteinePhase 1/21 trial
Active Trials
NCT00975689Completed35Est. Nov 2010

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
IntraBioN-Acetyl-L-Leucine
IntraBioIB1001
Human BioSciencesVTS-270
Genome & CompanyN-Acetyl Cysteine
Human BioSciencesEstablishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Human BioSciencesEvaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Clinical Trials (6)

Total enrollment: 1,125 patients across 6 trials

NCT05163288IntraBioN-Acetyl-L-Leucine

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

Start: Jun 2022Est. completion: Dec 203053 patients
Phase 3Recruiting
NCT03759639IntraBioIB1001

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)

Start: Sep 2019Est. completion: Nov 202233 patients
Phase 2Completed
NCT03471143Human BioSciencesVTS-270

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Start: Feb 2019Est. completion: Oct 20244 patients
Phase 1/2Completed
NCT00975689Genome & CompanyN-Acetyl Cysteine

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Start: Aug 2009Est. completion: Nov 201035 patients
Phase 1/2Completed
NCT05588167Human BioSciencesEstablishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C

Start: Nov 2022Est. completion: Sep 2026100 patients
N/ARecruiting
NCT00344331Human BioSciencesEvaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Evaluation of Biochemical Markers and Clinical Investigation of Niemann-Pick Disease, Type C

Start: Aug 2006900 patients
N/ARecruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 late-stage (Phase 3) programs, potential near-term approvals
3 actively recruiting trials targeting 1,125 patients
3 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.