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Niemann-Pick Disease, Type C1

5
Pipeline Programs
2
Companies
5
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Cyclo Therapeutics
Cyclo TherapeuticsFL - Gainesville
4 programs
2
1
1
Hydroxypropyl-beta-cyclodextrinPhase 31 trial
Hydroxypropyl-beta-cyclodextrinPhase 1/21 trial
Hydroxypropyl-beta-cyclodextrinPhase 11 trial
Hydroxypropyl-β-cyclodextrinPhase 11 trial
Active Trials
NCT02939547Completed13Est. Feb 2020
NCT03893071Unknown12Est. Mar 2022
NCT02912793Completed12Est. Mar 2021
+1 more trials
Human BioSciences
Human BioSciencesWV - Martinsburg
1 program
1
VTS-270Phase 1/21 trial
Active Trials
NCT03887533Terminated2Est. Oct 2021

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin
Human BioSciencesVTS-270
Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin
Cyclo TherapeuticsHydroxypropyl-β-cyclodextrin
Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin

Clinical Trials (5)

Total enrollment: 133 patients across 5 trials

NCT04860960Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin

Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1

Start: Jul 2021Est. completion: Jun 202694 patients
Phase 3Active Not Recruiting
NCT03887533Human BioSciencesVTS-270

Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1

Start: Jan 2020Est. completion: Oct 20212 patients
Phase 1/2Terminated
NCT02912793Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin

Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients

Start: Mar 2017Est. completion: Mar 202112 patients
Phase 1/2Completed
NCT03893071Cyclo TherapeuticsHydroxypropyl-β-cyclodextrin

Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C

Start: May 2019Est. completion: Mar 202212 patients
Phase 1Unknown
NCT02939547Cyclo TherapeuticsHydroxypropyl-beta-cyclodextrin

Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)

Start: Oct 2017Est. completion: Feb 202013 patients
Phase 1Completed

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
2 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.