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Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

NCT03879655 · Status: TERMINATED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2023-12-26

Study results available
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Summary

This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B \[NCT02534844\] and Part C \[NCT04958642\]) with neurologic manifestations of Niemann-Pick Type C1 (NPC1) disease.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

VTS-270

Administered IT via LP infusion of VTS-270

Sponsors & Collaborators

  • Mandos LLC

    lead INDUSTRY

Principal Investigators

  • Clinical Study Lead · Mandos LLC

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
4 Years
Max Age
21 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-12-02
Primary Completion
2021-10-18
Completion
2021-10-18
FDA Drug
Yes

Countries

  • Costa Rica

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03879655 on ClinicalTrials.gov