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Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

NCT03041116 · Status: TERMINATED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 84

Last updated 2021-01-26

Study results available
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Summary

This study investigated whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, was safe and effective in treating participants with PKAN.

Conditions

  • Pantothenate Kinase-Associated Neurodegeneration

Interventions

DRUG

Fosmetpantotenate

Daily dosing

DRUG

Placebo

Daily dosing

Sponsors & Collaborators

  • Travere Therapeutics, Inc.

    lead INDUSTRY

Principal Investigators

  • Thomas Klopstock, MD · Klinikum der Universität München

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
6 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-07-17
Primary Completion
2019-12-30
Completion
2019-12-30
FDA Drug
Yes

Countries

  • United States
  • Canada
  • Czechia
  • France
  • Germany
  • Italy
  • Norway
  • Poland
  • Spain

Study Locations

More Related Trials

Entities

Drugs

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 NCT03041116 on ClinicalTrials.gov