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

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


Lead Sponsor: Retrophin, Inc.

Source 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).

Overall Status Active, not recruiting
Start Date July 17, 2017
Completion Date July 2022
Primary Completion Date July 2020
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Evaluate change in the score of a PKAN specific activities of daily living measure (PKAN-ADL) 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. 24 weeks
Secondary Outcome
Measure Time Frame
Evaluate change in the Unified Parkinson's Disease Rating Scale (UPDRS) Part III score 24 weeks
Enrollment 82

Intervention Type: Drug

Intervention Name: fosmetpantotenate (RE-024)

Description: Daily dosing

Arm Group Label: fosmetpantotenate (RE-024)

Intervention Type: Drug

Intervention Name: Placebo

Description: Daily dosing

Arm Group Label: Placebo



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

Minimum Age: 6 Years

Maximum Age: 65 Years

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Thomas Klopstock, MD Principal Investigator Klinikum der Universität München
University of California Irvine, Clinical Trials Unit | Irvine, California, 92697, United States
Children's National Medical Center | Washington, District of Columbia, 20010, United States
Nicklaus Children's Hospital | Miami, Florida, 33155, United States
Emory University | Decatur, Georgia, 30033, United States
RUSH University Medical Center | Chicago, Illinois, 60612, United States
Massachusetts General Hospital | Boston, Massachusetts, 02114, United States
Columbia University Medical Center | New York, New York, 10032, United States
Children's Hospital of Pittsburgh | Pittsburgh, Pennsylvania, 15224, United States
The University of Texas Health Science Center at Houston | Houston, Texas, 77030, United States
The Hospital for Sick Children | Toronto, Ontario, M5G1X8, Canada
Toronto Western Hospital | Toronto, Ontario, M5T 2S8, Canada
Vseobecna fakultni nemocnice v Praze, Neurolgicka klinika 1 | Praha 2, NAP, 12821, Czechia
Hopital Pitie-Salpetriere, Centre d'investigation clinique et centre d'evaluation des therapeutiques | Paris, Ile-de-France, France
Unite Pathologies cerebrales resistantes, Departement de Neurochiurgie Fonctionelle, CHU de Montpellier - Hopital Gui de Chauliac | Montpellier, Languedoc-Rousillon, France
Klinikum der Universitat Munchen | München, Bavaria, 80336, Germany
U.O Neuropsichiatria Infantile, Fondazione I.R.C.C.S., Istituto Neurologico Carlo Besta | Milano, Italy
Section of Child Neurology Oslo University Hospital Rikshospitalet | Sognsvannsveien 20, Oslo, Norway
Klinika Neurologii, Epileptologii | Warsaw, Poland
Hospital Vall d'Hebron | Barcelona, Catalonia, 08035, Spain
Hospital Clinic i Provincial de Barcelona | Barcelona, Spain
Location Countries









United States

Verification Date

January 2019

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Label: fosmetpantotenate (RE-024)

Type: Experimental

Description: Administered as powder for reconstitution.

Label: Placebo

Type: Placebo Comparator

Description: Administered as powder for reconstitution.

Acronym PKAN
Study Design Info

Allocation: Randomized

Intervention Model: Parallel Assignment

Primary Purpose: Treatment

Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Source: ClinicalTrials.gov