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Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease

NCT01747135 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 14

Last updated 2021-07-23

No results posted yet for this study

Summary

Background:

\- Hydroxypropyl beta cyclodextrin (HPBCD) is being tested for a disease called Niemann-Pick disease type C1 (NPC1). NPC1 is a genetic disorder that results in gradual loss of nervous system function. Cholesterol and other fats have trouble moving out of the brain cells, which makes the cells work poorly and leads to symptoms. There is no treatment currently approved in the US for NPC1. Researchers want to test if it is safe to use HPBCD for NPC1. They want to see if it can help brain cells process cholesterol better.

Objectives:

\- To test the safety and effectiveness of HPBCD for NPC1.

Eligibility:

\- Individuals between 2 and 25 years of age who have been diagnosed with NPC1 and who have not already received HPBCD in an attempt to treat NPC1.

Design:

* Participants will be screened with a physical exam and medical history. They will provide blood and urine samples for screening. They will also have neurological tests, including tests of hearing, speech and movement.
* Participants will have a lumbar puncture (also called a spinal tap) every month to deliver the drug to the spinal fluid that surrounds the brain. The length of the trial will be determined by the safety and efficacy information that is obtained.
* Treatment will be monitored with frequent blood and urine tests, cerebral spinal fluid tests, hearing and neurological exams.

Conditions

  • Niemann-Pick Disease, Type C1

Interventions

DRUG

VTS-270

Sponsors & Collaborators

  • Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company

    lead INDUSTRY

Principal Investigators

  • Global Clinical Leader · Mallinckrodt

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
2 Years
Max Age
25 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-01-31
Primary Completion
2017-03-31
Completion
2017-03-31

Countries

  • United States

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 NCT01747135 on ClinicalTrials.gov