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Trial record 1 of 12 for:    pkan
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Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants (PKAN)

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ClinicalTrials.gov Identifier: NCT03041116
Recruitment Status : Terminated (The study was terminated since fosmetpantotenate did not show statistically significant effects or clinical benefits during the double-blind period.)
First Posted : February 2, 2017
Results First Posted : December 29, 2020
Last Update Posted : January 26, 2021
Information provided by (Responsible Party):
Travere Therapeutics, Inc.

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

Condition or disease Intervention/treatment Phase
Pantothenate Kinase-Associated Neurodegeneration Drug: Fosmetpantotenate Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 84 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : July 17, 2017
Actual Primary Completion Date : December 30, 2019
Actual Study Completion Date : December 30, 2019

Arm Intervention/treatment
Experimental: Fosmetpantotenate
Administered as powder for reconstitution.
Drug: Fosmetpantotenate
Daily dosing
Other Name: RE-024

Placebo Comparator: Placebo
Administered as powder for reconstitution.
Drug: Placebo
Daily dosing

Primary Outcome Measures :
  1. 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.

  2. 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.

Secondary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03041116

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United States, California
Travere Investigational Site
Irvine, California, United States, 92697
United States, District of Columbia
Travere Investigational Site
Washington, District of Columbia, United States, 20010
United States, Florida
Travere Investigational Site
Miami, Florida, United States, 33155
United States, Georgia
Travere Investigational Site
Decatur, Georgia, United States, 30033
United States, Illinois
Travere Investigational Site
Chicago, Illinois, United States, 60612
United States, Massachusetts
Travere Investigational Site
Boston, Massachusetts, United States, 02114
United States, New York
Travere Investigational Site
New York, New York, United States, 10032
United States, Pennsylvania
Travere Investigational Site
Pittsburgh, Pennsylvania, United States, 15224
United States, Texas
Travere Investigational Site
Houston, Texas, United States, 77030
Canada, Ontario
Travere Investigational Site
Toronto, Ontario, Canada, M5G1X8
Travere Investigational Site
Toronto, Ontario, Canada, M5T 2S8
Travere Investigational Site
Praha 2, NAP, Czechia, 12821
Travere Investigational Site
Paris, Ile-de-France, France
Travere Investigational Site
Montpellier, Languedoc-Rousillon, France
Travere Investigational Site
München, Bavaria, Germany, 80336
Travere Investigational Site
Milano, Italy
Travere Investigational Site
Oslo, Norway
Travere Investigational Site
Warsaw, Poland
Travere Investigational Site
Barcelona, Catalonia, Spain, 08035
Travere Investigational Site
Barcelona, Spain
Sponsors and Collaborators
Travere Therapeutics, Inc.
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Principal Investigator: Thomas Klopstock, MD Klinikum der Universität München
  Study Documents (Full-Text)

Documents provided by Travere Therapeutics, Inc.:
Study Protocol  [PDF] May 23, 2019
Statistical Analysis Plan  [PDF] April 22, 2019

Publications of Results:
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Responsible Party: Travere Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03041116    
Other Study ID Numbers: 024PKAN15004
First Posted: February 2, 2017    Key Record Dates
Results First Posted: December 29, 2020
Last Update Posted: January 26, 2021
Last Verified: January 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Travere Therapeutics, Inc.:
Additional relevant MeSH terms:
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Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
Pathologic Processes
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn