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

This study is currently recruiting participants.
Verified October 2017 by Retrophin, Inc.
ClinicalTrials.gov Identifier:
First Posted: February 2, 2017
Last Update Posted: October 25, 2017
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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Retrophin, Inc.
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: 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

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
Actual Study Start Date: July 17, 2017
Estimated Study Completion Date: July 2022
Estimated Primary Completion Date: July 2020 (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


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.

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
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 ClinicalTrials.gov identifier (NCT number): NCT03041116

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

United States, Georgia
Emory University Recruiting
Decatur, Georgia, United States, 30033
Principal Investigator: Hyder Jinnah, MD,PhD         
United States, Illinois
RUSH University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Principal Investigator: Cynthia Comella, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Principal Investigator: Aleksander Videnovic, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Principal Investigator: Maria Escolar, MD         
Sponsors and Collaborators
Retrophin, Inc.
Principal Investigator: Thomas Klopstock, MD Klinikum der Universität München
  More Information

Additional Information:
Responsible Party: Retrophin, Inc.
ClinicalTrials.gov Identifier: NCT03041116     History of Changes
Other Study ID Numbers: 024PKAN15004
First Submitted: December 2, 2016
First Posted: February 2, 2017
Last Update Posted: October 25, 2017
Last Verified: October 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