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Trial record 1 of 1 for:    re-024
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Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Patients (PKAN)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2017 by Retrophin, Inc.
Information provided by (Responsible Party):
Retrophin, Inc. Identifier:
First received: December 2, 2016
Last updated: April 25, 2017
Last verified: April 2017
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).

Condition Intervention Phase
Pantothenate Kinase-Associated Neurodegeneration
Drug: fosmetpantotenate (RE-024)
Drug: Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Participant, Care Provider, Investigator, Outcomes Assessor
Primary Purpose: Treatment
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

Resource links provided by NLM:

Further study details as provided by Retrophin, Inc.:

Primary Outcome Measures:
  • Evaluate change in the score of a PKAN specific activities of daily living measure (PKAN-ADL) [ Time Frame: 24 weeks ]
  • Evaluate safety and tolerability by occurrence of adverse events classified by MedDRA classification dictionary and safety assessments including vital signs, physical examinations, clinical laboratory tests, C-SSRS assessments, and electrocardiograms. [ Time Frame: 24 weeks ]

Secondary Outcome Measures:
  • Evaluate change in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score [ Time Frame: 24 weeks ]

Estimated Enrollment: 82
Anticipated Study Start Date: June 2017
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: fosmetpantotenate (RE-024)
Administered as powder for reconstitution.
Drug: fosmetpantotenate (RE-024)
Daily dosing
Placebo Comparator: Placebo
Administered as powder for reconstitution.
Drug: Placebo
Daily dosing


Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT03041116

Contact: Study Call Center 1-844-363-1866

United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Aleksander Videnovic, MD         
Sponsors and Collaborators
Retrophin, Inc.
  More Information

Additional Information:
Responsible Party: Retrophin, Inc. Identifier: NCT03041116     History of Changes
Other Study ID Numbers: 024PKAN15004
Study First Received: December 2, 2016
Last Updated: April 25, 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Retrophin, Inc.:

Additional relevant MeSH terms:
Nerve Degeneration
Pantothenate Kinase-Associated Neurodegeneration
Pathologic Processes
Basal Ganglia Diseases
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neuroaxonal Dystrophies
Movement Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn processed this record on April 27, 2017