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

Travere Therapeutics

Status and phase: Terminated; Phase 3

Conditions: Pantothenate Kinase-Associated Neurodegeneration

Treatments: Drug: Fosmetpantotenate; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03041116

024PKAN15004

Details and patient eligibility

About

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

Enrollment: 84 patients

Sex: All

Ages: 6 to 65 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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84 participants in 2 patient groups, including a placebo group

Fosmetpantotenate

Experimental group

Description:

Administered as powder for reconstitution.

Treatment:

Drug: Fosmetpantotenate

Placebo

Placebo Comparator group

Description:

Administered as powder for reconstitution.

Treatment:

Drug: Placebo