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

Brief Title

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

Official Title

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

Brief Summary

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

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

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

Secondary Outcome

 Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period

Condition

Pantothenate Kinase-Associated Neurodegeneration

Intervention

Fosmetpantotenate

Study Arms / Comparison Groups

 Fosmetpantotenate

Description:  Administered as powder for reconstitution.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

84

Start Date

July 17, 2017

Completion Date

December 30, 2019

Primary Completion Date

December 30, 2019

Eligibility Criteria

        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.
      

Gender

All

Ages

6 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Thomas Klopstock, MD, , 

Location Countries

Canada

Location Countries

Canada

NCT ID

NCT03041116

Organization ID

024PKAN15004

Responsible Party

Sponsor

Study Sponsor

Travere Therapeutics, Inc.

Study Sponsor

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

Verification Date

January 2021