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

January 7, 2021 updated by: Travere Therapeutics, Inc.

Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate Replacement Therapy, in Pantothenate Kinase-Associated Neurodegeneration (PKAN) Patients: A Randomized, Double-Blind, Placebo-Controlled Study With an Open-Label Extension

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

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

84

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Canada
    • Ontario
      • Toronto, Ontario, Canada, M5G1X8
        • Travere Investigational Site
      • Toronto, Ontario, Canada, M5T 2S8
        • Travere Investigational Site
  • Czechia
    • NAP
      • Praha 2, NAP, Czechia, 12821
        • Travere Investigational Site
  • France
    • Ile-de-France
      • Paris, Ile-de-France, France
        • Travere Investigational Site
    • Languedoc-Rousillon
      • Montpellier, Languedoc-Rousillon, France
        • Travere Investigational Site
  • Germany
    • Bavaria
      • München, Bavaria, Germany, 80336
        • Travere Investigational Site
  • Italy
      • Milano, Italy
        • Travere Investigational Site
  • Norway
      • Oslo, Norway
        • Travere Investigational Site
  • Poland
      • Warsaw, Poland
        • Travere Investigational Site
  • Spain
      • Barcelona, Spain
        • Travere Investigational Site
    • Catalonia
      • Barcelona, Catalonia, Spain, 08035
        • Travere Investigational Site
  • United States
    • California
      • Irvine, California, United States, 92697
        • Travere Investigational Site
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Travere Investigational Site
    • Florida
      • Miami, Florida, United States, 33155
        • Travere Investigational Site
    • Georgia
      • Decatur, Georgia, United States, 30033
        • Travere Investigational Site
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Travere Investigational Site
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Travere Investigational Site
    • New York
      • New York, New York, United States, 10032
        • Travere Investigational Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15224
        • Travere Investigational Site
    • Texas
      • Houston, Texas, United States, 77030
        • Travere Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years to 63 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. The participant had a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 gene.
  2. The participant was male or female aged 6 to 65 years, inclusive.
  3. The participant had a score of ≥ 6 on the PKAN-specific activities of daily living measure.

Exclusion Criteria:

  1. The participant had required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
  2. The participant had a deep brain stimulation device implanted within 6 months prior to screening.
  3. The participant had taken deferiprone within 30 days prior to screening.
  4. The participant was unable to maintain stable doses of allowed concomitant medications for the first 24 weeks of the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Fosmetpantotenate
Administered as powder for reconstitution.
Drug: Fosmetpantotenate
Daily dosing
Other Names:
  • RE-024
Placebo Comparator: Placebo
Administered as powder for reconstitution.
Drug: Placebo
Daily dosing

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change From Baseline In The Pantothenate Kinase-Associated Neurodegeneration-Activities of Daily Living (PKAN-ADL) Total Score To The End Of The 24-week Double-blind Period
Time Frame: Baseline (Day -1), Week 24
Change from baseline to Week 24 activities of daily living was assessed on the PKAN-ADL scale based on the Unified Parkinson's Disease Rating Scale (UPDRS) Part II. The PKAN-ADL is a validated measure of the participant's ability to complete ADL that are impacted by the diffuse motor manifestations of PKAN. It consists of 12 items related to activities of daily living, including eating, dressing, and walking. Each item has responses ranging from 0-4, with a higher value indicating greater disability in the given activity. To compute the total score, responses are summed across the 12 items. The available range of total scores on the PKAN-ADL scale was from 0 (no ADL affected) to 48 (ADL highly affected). The reported least square mean (LSM) was adjusted for baseline score and age group from the Type III analysis. A decrease in score indicates improvement in symptoms.
Baseline (Day -1), Week 24
Number Of Participants With At Least 1 (≥1) Treatment-emergent Adverse Event (TEAE) And Treatment-emergent Serious Adverse Event (TESAE) During The 24-week Double-blind Period
Time Frame: From Screening until end of Week 24
An adverse event (AE) is any untoward medical occurrence associated with the use of the investigational product (IP; active or placebo) in a participant, without regard to possibility of causal relationship with IP. A serious adverse event (SAE) is an AE resulting in any of the following outcomes: death; initial or prolonged inpatient hospitalization; life threatening experience (immediate risk of death from AE); persistent or significant disability/incapacity; congenital anomaly/birth defect; other medically important serious medical events. The TEAEs in the double-blind period are defined as AEs that are new or are a worsening of an existing condition that begins from day of first dose of IP until day after last dose for double-blind treatment period.
From Screening until end of Week 24

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period
Time Frame: Baseline (Day -1), Week 24
The UPDRS is a comprehensive assessment of the burden and severity of signs and symptoms of Parkinsonism captured via systematic interview and neurological examination. The UPDRS-III is a standardized neurological examination that evaluates the performance of movements commonly affected in Parkinson's disease, PKAN, and other movement disorders. Part III of the UPDRS consists of 27 items, which correspond to 14 domains related to motor abilities such as tremor, stability, and bradykinesia. Each item has responses ranging from 0-4. To compute the UPDRS-III total score, responses are summed across the 27 items, and accordingly, range from 0-108. For domain totals, responses are summed across all of the items in a given domain (when domain corresponds to multiple items). An increase in score indicates greater disability. The reported LSM was adjusted for baseline score and age group from the Type III analysis.
Baseline (Day -1), Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Travere Therapeutics, Inc.

Investigators

  • Principal Investigator: Thomas Klopstock, MD, Klinikum der Universität München

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 17, 2017

Primary Completion (Actual)

December 30, 2019

Study Completion (Actual)

December 30, 2019

Study Registration Dates

First Submitted

December 2, 2016

First Submitted That Met QC Criteria

January 31, 2017

First Posted (Estimate)

February 2, 2017

Study Record Updates

Last Update Posted (Actual)

January 26, 2021

Last Update Submitted That Met QC Criteria

January 7, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 024PKAN15004

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Pantothenate Kinase-Associated Neurodegeneration

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Ukraine

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe