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

Description

This study will investigate whether fosmetpantotenate (RE-024), a phosphopantothenate replacement therapy, is safe and effective in treating patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN).

Study Start Date

July, 17 2017

Estimated Completion Date

July 2022

Interventions

  • Drug: Placebo
  • Drug: fosmetpantotenate (RE-024)

Study ID

Retrophin, Inc. -- 024PKAN15004

Status

Recruiting

Trial ID

NCT03041116

Study Type

Interventional

Trial Phase

Phase 3

Enrollment Quota

82

Sponsor

Retrophin, Inc.

Inclusion Criteria

    1. The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene. 2. The patient is male or female aged 6 to 65 years, inclusive. 3. The patient has a score of >6 on the PKAN-specific activities of daily living measure (PKAN-ADL)

Exclusion Criteria

    1. The patient has required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization. 2. The patient has had a deep brain stimulation (DBS) device implanted within 6 months prior to screening. 3. The patient has taken deferiprone within 30 days prior to screening. 4. The patient is unable to maintain stable doses of allowed concomitant medications for the first 24 weeks of the study.

Gender

All

Ages

6 Years to 65 Years

Accepts Healthy Volunteers

No

Study Locations and Contact Information (11)

Study Location Distance Name Phone Email
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles None None None
Massachusetts General Hospital - Boston, Massachusetts 2.8 miles None None None
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles None None None
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles None None None
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles None None None
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles None None None
Childrens Hospital of Pittsburgh - Pittsburgh, Pennsylvania 477.6 miles None None None
RUSH University Medical Center - Chicago, Illinois 851.3 miles None None None
RUSH University Medical Center - Chicago, Illinois 851.3 miles None None None
Emory University - Decatur, Georgia 929.2 miles None None None
Emory University - Decatur, Georgia 929.2 miles None None None

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