Efficacy, Safety, and Tolerability of Fosmetpantotenate in patients with Pantothenate Kinase-associated Neurodegeneration (PKAN)
- Conditions
- 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 disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2016-001955-29-ES
- Lead Sponsor
- Retrophin, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 82
- 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
- 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.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method