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Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System

NCT03643562 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 27

Last updated 2024-01-17

Study results available
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Summary

This study was amended from expanded access to a clinical trial. Information will be collected about long-term safety and effectiveness of adrabetadex shots in the spine every 2 weeks.

Participants who were already taking adrabetadex will receive their stable dose. Participants who have not ever taken it will start by receiving 400 mg.

Participants will receive treatment every 2 weeks until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason. Participants will not receive additional study treatment after their participation in this protocol.

Conditions

  • Niemann-Pick Type C Disease

Interventions

DRUG

Adrabetadex

Administered via lumbar puncture (LP) and IT infusion

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
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-06-18
Primary Completion
2021-11-02
Completion
2021-11-02
FDA Drug
Yes

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 NCT03643562 on ClinicalTrials.gov