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

Phase 1

• 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: 21.1Level: PTClassification code 10053643Term: Neurodegenerative disorderSystem Organ Class: 10029205 - Nervous system disorders; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2016-001955-29-IT
• Lead Sponsor: RETROPHIN, INC

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Completion: 2019-12-30
• Study Start: 2020-11-05
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 84

Inclusion Criteria

- The patient has a diagnosis of PKAN as indicated by confirmed mutations in the pantothenate kinase 2 (PANK2) gene (if available, the specific mutation will be recorded).
- 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:
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 (including DBS settings) 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