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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03041116
Recruitment Status : Recruiting
First Posted : February 2, 2017
Last Update Posted : August 23, 2018
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

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

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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

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

United States, California
University of California Irvine, Clinical Trials Unit Recruiting
Irvine, California, United States, 92697
Contact: Neal Hermanowicz, MD         
United States, Florida
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Migvis Monduy, MD         
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, New York
Columbia University Medical Center Recruiting
New York, New York, United States, 10032
Principal Investigator: Nora Vanegas, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Principal Investigator: Maria Escolar, MD         
United States, Texas
The University of Texas Health Science Center at Houston Recruiting
Houston, Texas, United States, 77030
Contact: Nivedita Thakur, MD         
Canada, Ontario
Toronto Western Hospital Recruiting
Toronto, Ontario, Canada, M5T 2S8
Contact: Anthony Lang, MD         
Vseobecna fakultni nemocnice v Praze, Neurolgicka klinika 1 Recruiting
Praha 2, NAP, Czechia, 12821
Contact: Robert Jech, MUDr., Ph.D.         
Hopital Pitie-Salpetriere, Centre d'investigation clinique et centre d'evaluation des therapeutiques Recruiting
Paris, Ile-de-France, France
Contact: Emmanuel Flamand-Roze, MD         
Unite Pathologies cerebrales resistantes, Departement de Neurochiurgie Fonctionelle, CHU de Montpellier - Hopital Gui de Chauliac Recruiting
Montpellier, Languedoc-Rousillon, France
Contact: Victoria Gonzalez Martinez, MD, Ph.D.         
Klinikum der Universitat Munchen Recruiting
München, Bavaria, Germany, 80336
Contact: Thomas Klopstock, MD         
U.O Neuropsichiatria Infantile, Fondazione I.R.C.C.S., Istituto Neurologico Carlo Besta Recruiting
Milano, Italy
Contact: Giovanna S Francesca Zorzi, M.D.         
Hospital Vall d'Hebron Recruiting
Barcelona, Catalonia, Spain, 08035
Contact: Belen Perez Duenas, MD         
Hospital Clinic i Provincial de Barcelona Recruiting
Barcelona, Spain
Contact: Maria Jose Marti, M.D.         
Sponsors and Collaborators
Retrophin, Inc.
Principal Investigator: Thomas Klopstock, MD Klinikum der Universität München

Additional Information:
Responsible Party: Retrophin, Inc. Identifier: NCT03041116     History of Changes
Other Study ID Numbers: 024PKAN15004
First Posted: February 2, 2017    Key Record Dates
Last Update Posted: August 23, 2018
Last Verified: August 2018

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