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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

84 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-17

Study Completion Date

2019-12-30

Brief Summary

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

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Detailed Description

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Conditions

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Pantothenate Kinase-Associated Neurodegeneration

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Fosmetpantotenate

Administered as powder for reconstitution.

Group Type EXPERIMENTAL

Fosmetpantotenate

Intervention Type DRUG

Daily dosing

Placebo

Administered as powder for reconstitution.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Daily dosing

Interventions

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Fosmetpantotenate

Daily dosing

Intervention Type DRUG

Placebo

Daily dosing

Intervention Type DRUG

Other Intervention Names

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RE-024

Eligibility Criteria

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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.

Minimum Eligible Age

6 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No