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VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

NCT02534844 · Status: COMPLETED · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 56

Last updated 2023-02-22

Study results available
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Summary

Due to different study designs, the sponsor separated Part C into a separate registration (NCT04958642), leaving Parts A/B here in NCT02534844.

This study is to find out how safe and effective VTS-270 is for patients with Niemann-Pick Type C1 (NPC1) disease who have neurologic symptoms (listed under Keywords).

In Parts A/B, two out of every three patients will receive the study drug. The third patient will receive 1 to 2 small needle pricks at the location where the LP and IT injection is normally made (sham control).

In Part C, all participants will receive study drug, as described in the Part C registration record.

Start date for this record is the first day a participant was enrolled in Parts A/B. The trial is actually continuing until the last primary outcome measure of safety data are collected from Part C participants. The last primary outcome measure of safety, along with final adverse events results will be posted in the separate Part C registration record.

Conditions

  • Niemann-Pick Disease, Type C

Interventions

DRUG

Parts A/B: Adrabetadex

900 - 1800 milligram (mg) of adrabetadex administered every 2 weeks via lumbar intrathecal infusion

OTHER

Parts A/B: Sham Control

No experimental drug is administered to participants - intrathecal administrations are simulated by skin prick

Sponsors & Collaborators

  • Mandos LLC

    lead INDUSTRY

Principal Investigators

  • Clinical Study Lead · Mandos LLC

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
PARALLEL

Eligibility

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

Timeline & Regulatory

Start
2015-10-31
Primary Completion
2018-03-28
Completion
2018-03-28
FDA Drug
Yes

Countries

  • United States
  • Australia
  • France
  • Germany
  • New Zealand
  • Singapore
  • Spain
  • Turkey (Türkiye)
  • United Kingdom

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 NCT02534844 on ClinicalTrials.gov