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

Phase 3TerminatedNCT03041116

Travere Therapeutics, Inc.84 enrolled

Overview

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

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Pantothenate Kinase-Associated Neurodegeneration

Interventions

FosmetpantotenateDRUG

Daily dosing

PlaceboDRUG

Daily dosing

Eligibility

Sex: ALLMin age: 6 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (20)

Travere Investigational Site

Irvine, California, United States

Travere Investigational Site

Washington D.C., District of Columbia, United States

Outcomes

Primary Outcomes

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

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.

Time frame: 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

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.

Time frame: From Screening until end of Week 24

Secondary Outcomes

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

Data from ClinicalTrials.gov

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