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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03041116
Recruitment Status : Active, not recruiting
First Posted : February 2, 2017
Last Update Posted : January 3, 2019
Information provided by (Responsible Party):
Retrophin, Inc.

Brief Summary:
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 or disease Intervention/treatment Phase
Pantothenate Kinase-Associated Neurodegeneration Drug: fosmetpantotenate (RE-024) Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 82 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (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
Actual Study Start Date : July 17, 2017
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : July 2022

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. Evaluate change in the score of a PKAN specific activities of daily living measure (PKAN-ADL) [ Time Frame: 24 weeks ]
  2. 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 :
  1. Evaluate change in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score [ Time Frame: 24 weeks ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   6 Years to 65 Years   (Child, Adult, Older 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03041116

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Sponsors and Collaborators
Retrophin, Inc.
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Principal Investigator: Thomas Klopstock, MD Klinikum der Universität München
Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Retrophin, Inc. Identifier: NCT03041116    
Other Study ID Numbers: 024PKAN15004
First Posted: February 2, 2017    Key Record Dates
Last Update Posted: January 3, 2019
Last Verified: January 2019

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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:
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Pantothenate Kinase-Associated Neurodegeneration
Nerve Degeneration
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