Open‐label pilot study of interferon gamma‐1b in Friedreich ataxia

  title={Open‐label pilot study of interferon gamma‐1b in Friedreich ataxia},
  author={Lauren A. Seyer and Nathaniel R Greeley and Dominik Foerster and Cassandra J Strawser and Sarah Gelbard and Yi Na Dong and Kimberly A. Schadt and Maria Grazia Cotticelli and Alicia F D Brocht and Jennifer M. Farmer and R B Wilson and David R. Lynch},
  journal={Acta Neurologica Scandinavica},
  pages={15 - 7}
This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. 

Randomized, double‐blind, placebo‐controlled study of interferon‐γ 1b in Friedreich Ataxia

Evaluated the efficacy and safety of IFN‐γ 1b in the treatment of Friedreich Ataxia through a double‐blind, multicenter, placebo‐controlled trial.

IFN-γ for Friedreich ataxia: present evidence.

Ongoing Phase II and III trials in both adults and children with FRDA showed that IFN-γ-1b was reasonably well-tolerated and improved overall neurological function as measured by the Friedreich Ataxia Rating Scale after 12 weeks of treatment, though the primary outcome measure of frataxin level showed no improvement.

Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia

The dose‐ranging portion of this Phase 2 study assessed the safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia patients.

Safety and Efficacy Of Interferon γ in Friedreich's Ataxia

The treatment was safe, with only 2 serious adverse effects, both spontaneously resolved, and 1 termination as a result of a refusal by the patient to continue the treatment despite resolution of the event, otherwise the treatment was reasonably well tolerated with the occurrence of common adverse events known to be associated with IFNγ treatment.

GIFT-1, a phase IIa clinical trial to test the safety and efficacy of IFNγ administration in FRDA patients

A phase IIa clinical trial, the first in Italy, aimed at assessing both safety and tolerability of IFNγ in Friedreich’s patients and ability to increase frataxin levels in peripheral blood mononuclear cells, found IFnγ was generally well tolerated and the main adverse event was hyperthermia/fever.

Efficacy and Tolerability of Interferon Gamma in Treatment of Friedreich's Ataxia: Retrospective Study.

The clinical features, tolerability, and the prognosis of individuals with FRDA to whom IFN-γ was administered in a university hospital were evaluated retrospectively and the results were discussed.

An open-label pilot study of recombinant granulocyte-colony stimulating factor in Friedreich’s ataxia

An open‐label, pilot study of recombinant human granulocyte-colony stimulating factor administration in seven people with Friedreich’s ataxia shows potential for G-CSF therapy to have a clinical impact in people with FA.

Progress in the treatment of Friedreich ataxia.

Emerging therapies in Friedreich's ataxia.

Emerging therapies and future perspectives are discussed, including the need for more precise measures for detecting changes in neurologic symptoms as well as a disease-modifying agent.

Etravirine in Friedreich's ataxia: Lessons from HIV?

The reconstitution of aconitase shows that the etravirine-induced increase in frataxin leads to functional enzymatic improvements that may be disease related, and there are several necessary steps before this medication could be tried widely in patients.



Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia

This multicenter, double‐blind, placebo‐controlled, phase II clinical trial aimed to evaluate the safety and tolerability of Lu AA24493 (carbamylated EPO; CEPO).

Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia – open‐label trial

The objective of the study was to test the efficacy, safety and tolerability of triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia (FRDA) patients in a clinical pilot

Erythropoietin in Friedreich ataxia: No effect on frataxin in a randomized controlled trial

Friedreich ataxia is a rare disease caused by GAA‐trinucleotide‐repeat expansions in the frataxin gene, leading to marked reduction of qualitatively normal frataxin protein. Recently, human

A0001 in Friedreich ataxia: Biochemical characterization and effects in a clinical trial

  • D. LynchS. Willi T. Sciascia
  • Medicine, Psychology
    Movement disorders : official journal of the Movement Disorder Society
  • 2012
Although A0001 did not alter the Disposition Index, it caused a dose‐dependent improvement in neurologic function, as measured by the Friedreich Ataxia Rating Scale, and longer studies will assess the reproducibility and persistence of neurologic benefit.

Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model

It is shown that frataxin levels can be upregulated by interferon gamma (IFNγ) in a variety of cell types, including primary cells derived from FRDA patients, and in vivo treatment with IFNγ increases fr ataxin expression in DRG neurons, prevents their pathological changes and ameliorates the sensorimotor performance in FRDA mice.

A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia.

Iebenone did not significantly alter neurological function in Friedreich ataxia during the 6-month study, and larger studies of longer duration may be needed to assess the therapeutic potential of drug candidates on neurologicalfunction in Fried reaxia.

Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy.

The discovery of the genetic cause of Friedreich ataxia has significantly affected understanding of the disorder at both the clinical and basic science levels, and antioxidants are a potential method for therapeutic intervention.

A short 2 week dose titration regimen reduces the severity of flu-like symptoms with initial interferon gamma-1b treatment

A short 2 week, dose-titration regimen reduces FLS severity following interferon gamma-1b treatment initiation in normal subjects and results in near baseline severity scores throughout the treatment period.

A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia.