Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)

• Register: EUCTR
• Last refreshed on: 16 December 2019
• Main ID: EUCTR2016-001955-29-ES
• Date of registration: 30/03/2017
• Prospective Registration: Yes
• Primary sponsor: Retrophin, Inc.
• Public title: Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)
• Scientific title: Efficacy, Safety, and Tolerability of Fosmetpantotenate (RE-024), a Phosphopantothenate replacement therapy, in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN): A Randomized, Double-blind, Placebo-Controlled Study with an Open-Label Extension
• Date of first enrolment: 16/10/2017
• Target sample size: 82
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-001955-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Canada, Czech Republic, France, Germany, Italy, Norway, Poland, Spain, United Kingdom, United States

Contacts

• Name: Clinical Trial Information Desk
• Affiliation: Retrophin Inc.

• Name: Clinical Trial Information Desk
• Affiliation: Retrophin Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
- The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene.
- The patient has a score of >= 6 on the Pantothenate Kinase-associated Neurodegeneration Activities of Daily Living (PKAN-ADL) scale.
Are the trial subjects under 18? yes
Number of subjects for this age range: 41
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 41
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- The patient has required regular or intermittent invasive ventilatory support to maintain vital signs within 24 weeks prior to randomization.
- The patient has had a deep brain stimulation (DBS) device implanted within 6 months prior to screening.
- The patient is unable or unwilling to remain on their pre-study dose(s) of allowed concomitant PKAN maintenance medications and therapies for the double-blind period of the study.
- The patient has taken deferiprone within 30 days prior to screening.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Pantothenate kinase associated neurodegeneration (PKAN), an autosomal recessive genetic disorder, the most common form of Neurodegeneration with Brain Iron Accumulation (NBIA). It is a progressive, often fatal, neurodegenerative disease.
MedDRA version: 19.1 Level: PT Classification code 10053643 Term: Neurodegenerative disorder System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Product Name: Fosmetpantotenate
Product Code: RE-024
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: Fosmetpantotenate
CAS Number: 1858268-66-2
Current Sponsor code: RE-024
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 75-
Pharmaceutical form of the placebo: Powder for oral suspension
Route of administration of the placebo: Oral use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: For each patient, the change from Baseline in PKAN-ADL scores at Weeks 3, 6, 12, 18, and 24 of the double-blind period will be used for analysis.

Primary end point(s): Primary Efficacy Endpoint
- Change in the score from the PKAN-ADL

Safety Endpoint
- Safety and tolerability

Secondary Objective: To determine the PK following multiple doses of fosmetpantotenate in patients with PKAN.
To explore potential biomarkers of disease, along with their potential response to treatment in patients with PKAN.

Main Objective: The efficacy objective of this study is to evaluate the efficacy of fosmetpantotenate over 24 weeks in patients with PKAN.
The safety objective of the study is to assess the safety and tolerability of fosmetpantotenate in patients with PKAN.

Secondary Outcome(s)

Secondary end point(s): Secondary Efficacy Endpoint
- Change in the score from Part III of the UPDRS

Timepoint(s) of evaluation of this end point: Continuous measures recorded at each study visit from Baseline to the end of the 24-week double-blind period.

Secondary ID(s)

024PKAN15004

2016-001955-29-DE

Source(s) of Monetary Support

Retrophin, Inc.

Ethics review

Status: Approved
Approval date:
Contact: