Clinical Trials Logo
  1. Home
  2. Friedreich Ataxia
  3. NCT03761511

Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia — NICOFA  

Friedreich Ataxia Clinical Trial

Official title:
A Randomized, Double-blind, Placebo-controlled, Parallel-group, Multicentre Study of the Efficacy and Safety of Nicotinamide in Patients With Friedreich Ataxia

Clinical Trial 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 Friedreich´s ataxia.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03761511
Study type Interventional
Source RWTH Aachen University
Contact
Status Withdrawn
Phase Phase 2
Start date April 2023
Completion date December 2025
View Details
See also
  Status Clinical Trial Phase
Completed NCT05573698 - Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia Phase 1
Completed NCT00229632 - Idebenone to Treat Friedreich's Ataxia Phase 2
Completed NCT05579691 - A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia Phase 2
Completed NCT04273165 - Safety and Efficacy of Etravirine in Friedreich Ataxia Patients Phase 2
Not yet recruiting NCT05874388 - Characterisation of the Cognitive Profile of Patients Suffering From Friedreich's Ataxia N/A
Completed NCT02594917 - Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension
Completed NCT01716221 - An Objective Double-blind Evaluation of Bupropion and Citalopram in an Individual With Friedreich Ataxia Phase 4
Active, not recruiting NCT05485987 - A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Phase 2
Completed NCT03214588 - Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia Phase 2
Completed NCT03418740 - Neurology Measures in FA Children
Not yet recruiting NCT06054893 - A Study of Omaveloxolone in Children With Friedreich's Ataxia Phase 1
Recruiting NCT04921930 - Evaluation of the Effect of Artesunate in Friedreich Ataxia (FA) Phase 1/Phase 2
Completed NCT03933163 - Micronised Resveratrol as a Treatment for Friedreich Ataxia Phase 2
Completed NCT02705547 - Rosuvastatin (Crestor) in Friedreich Ataxia Early Phase 1
Completed NCT02035020 - A Phase IIa Trial to Test Safety and Efficacy Interferon Gamma Treatment in Elevating Frataxin Levels in FRDA Patients Phase 2
Completed NCT00015808 - Safety Study of Idebenone to Treat Friedreich's Ataxia Phase 1
Completed NCT04817111 - NAD+ Precursor Supplementation in Friedreich's Ataxia Phase 2
Active, not recruiting NCT05168774 - FRDA Investigator Initiated Study (IIS) With Elamipretide Phase 1/Phase 2
Completed NCT03917225 - A Clinical Study to Evaluate the Effect of MIN-102 on the Progression of Friedreich's Ataxia in Male and Female Patients Phase 2
Completed NCT00224640 - Iron-Chelating Therapy and Friedreich Ataxia Phase 1/Phase 2