Ignite Creation Date:
2024-05-06 @ 12:27 PM
Last Modification Date:
2024-10-26 @ 12:59 PM
Study NCT ID:
NCT03761511
Status:
WITHDRAWN
Last Update Posted:
2024-01-24
First Post:
2018-11-27
Brief Title:
Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia
Sponsor:
RWTH Aachen University
Organization:
RWTH Aachen University
Study Overview
Official Title:
A Randomized Double-blind Placebo-controlled Parallel-group Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia
Status:
WITHDRAWN
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Due to a temporary halt the recruitment start was delayed A re-evaluation of the sponsor revealed that the study is no longer feasible due to increased administrative and financial requirements
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
NICOFA
Brief Summary:
Friedreich ataxia is the most frequent early-onset autosomal recessive hereditary ataxia It is caused by a pathological expansion of a GAA repeat in the first intron of the frataxin gene FXN and results in decreased levels of FXN protein FXN deficiency results in a relentlessly progressive neurodegenerative condition which frequently presents around puberty Patients gradually lose coordination become dysarthric and are frequently wheel-chair bound as adolescents There is no disease modifying therapy and many patients die prematurely of cardiomyopathy It was subsequently found that the FXN gene is silenced at the chromatin level by the formation of heterochromatin and that this heterochromatin formation can be antagonized by histone deacetylase inhibitors HDACi Chan et al 2013 A recent proof-of-concept clinical study on ten patients with Friedreich ataxia demonstrated that FXN levels can be restored to those seen in asymptomatic carriers using the class III HDACi nicotinamide at a dose that is well tolerated by patients Libri et al 2014 Since carriers are asymptomatic this degree of restoration of FXN expression might be expected to halt disease progression Nicotinamide readily crosses the blood brain barrier and has previously been given at high doses for long periods to normal individuals without serious adverse effects Gale et al 2004 Knip et al 2000 This study will be the first to provide clinical evidence for the efficacy and safety of nicotinamide in patients with Friedreichs ataxia
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None