Clinical trial NCT03041116: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Pantothenate Kinase-Associated Neurodegeneration
Keywords: PKAN
Lead sponsor: Travere Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2017-02-02
Enrollment: 84
Sex: ALL
Minimum age (years): 6
Maximum age (years): 65
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: 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
timeFrame: Baseline (Day -1), Week 24
measure: 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
timeFrame: From Screening until end of Week 24
Secondary outcomes:
measure: Absolute Change From Baseline In The UPDRS Part 3 (UPDRS-III) Total Score To The End Of The 24-week Double-blind Period
timeFrame: Baseline (Day -1), Week 24
Other outcomes:
Locations:
city: Irvine
country: United States
facility: Travere Investigational Site
state: California
city: Washington
country: United States
facility: Travere Investigational Site
state: District of Columbia
city: Miami
country: United States
facility: Travere Investigational Site
state: Florida
city: Decatur
country: United States
facility: Travere Investigational Site
state: Georgia
city: Chicago
country: United States
facility: Travere Investigational Site
state: Illinois
city: Boston
country: United States
facility: Travere Investigational Site
state: Massachusetts
city: New York
country: United States
facility: Travere Investigational Site
state: New York
city: Pittsburgh
country: United States
facility: Travere Investigational Site
state: Pennsylvania
city: Houston
country: United States
facility: Travere Investigational Site
state: Texas
city: Toronto
country: Canada
facility: Travere Investigational Site
state: Ontario
city: Toronto
country: Canada
facility: Travere Investigational Site
state: Ontario
city: Praha 2
country: Czechia
facility: Travere Investigational Site
state: NAP
city: Paris
country: France
facility: Travere Investigational Site
state: Ile-de-France
city: Montpellier
country: France
facility: Travere Investigational Site
state: Languedoc-Rousillon
city: München
country: Germany
facility: Travere Investigational Site
state: Bavaria
city: Milano
country: Italy
facility: Travere Investigational Site
city: Oslo
country: Norway
facility: Travere Investigational Site
city: Warsaw
country: Poland
facility: Travere Investigational Site
city: Barcelona
country: Spain
facility: Travere Investigational Site
state: Catalonia
city: Barcelona
country: Spain
facility: Travere Investigational Site
Study officials:
affiliation: Klinikum der Universität München
name: Thomas Klopstock, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Klopstock T, Escolar ML, Marshall RD, Perez-Duenas B, Tuller S, Videnovic A, Greblikas F. The FOsmetpantotenate Replacement Therapy (FORT) randomized, double-blind, Placebo-controlled pivotal trial: Study design and development methodology of a novel primary efficacy outcome in patients with pantothenate kinase-associated neurodegeneration. Clin Trials. 2019 Aug;16(4):410-418. doi: 10.1177/1740774519845673. Epub 2019 May 6.