Clinical Trials

Interventional clinical trials ("drug trials") test the safety and efficacy of new drugs and treatments that could potentially help manage symptoms better or even slow or halt disease progression.

Ataxia Centre researchers lead a wide range of clinical trials carried out across UCL-affiliated hospitals, including University College London Hospital (UCLH), National Hospital for Neurology and Neurosurgery at Queen Square

MOXIe trial

In partnership with the pharmaceutical company Reata, this study is recruiting patients with Friedreich’s ataxia (FRDA) in the United Kingdom for a Phase 2 clinical trial studying the safety and effects of RTA408 (a semi-synthetic triterpenoid, an Nrf2 activator with antioxidant properties) in FRDA.
To be eligible for this trial you must:

  • Have genetic confirmation of your FRDA.
  • Be between the ages of 16 and 40.
  • Be willing to maintain a consistent exercise routine and stable medication dosesthroughout the study.
  • Be willing to discontinue taking all antioxidant supplements and vitamins, or any other medication intended to treat Friedreich’s ataxia, before beginning this study drug and throughout your participation in the study.
  • Use an acceptable form of contraception throughout the study.

In addition, you must NOT:

  • Have any clinically relevant medical or surgical condition that could interfere with the administration of study drug, or compromise your safety or well-being.
  • Be pregnant, planning a pregnancy, or breastfeeding.

We are no longer recuiting any new participants for this study. The conclusive results of this trial has led to the very first treatment approved by the FDA for patients living with Friedreich's ataxia. You can read more about this unprecedent achievement announced on the International Rare Disease Day 2023 here. 

You can also read the publications linked to this study:   and . 

NICOFA trial                                                                                                                                                                    

Organized in the Center for Translational and Clinical Research of Aachen (Germany), this is a phase 2 clinical tria to study the effects of nicotinamide (vitamin B3) in Friedreich's ataxia. This substance has shown to increase the production of the frataxin protein by binding to the frataxin gene and reactivating it. 

To be eligible for this trial you must:                                                                                                                            

  • Have genetic confirmation of your Friedreich's ataxia.
  • Be between 18 and 50 years of age and weight at least 50kg.
  • If female and with childbearing potential, use highly effective birth control methods throughout the study.

In addition, you must NOT:

  • Have any clinically relevant medical or surgical condition, including hypersensitity to nicotinamide and severe allergies, that could interfere with the administration of study drug, or compromise your safety or well-being.
  • Have a history of subtance abuse in the 6 months prior to enrolment.
  • Be taking sodium valproate, tranylcypromine (monoamine oxidase inhibitor (MAOI)), any other known histone deacetylase inhibitor, or vitamin B1 (thiamine).
  • Be taking nicotinamide at least 3 months before the start of the trial.
  • Be taking drugs from other clinical trials.
  • Modify the dose of idebenone or coenzyme Q10 during the study, if they were being taken at the beginning of the study
  • Be pregnant, planning a pregnancy, or breastfeeding.

Recruitment for this study will start in the near future. In the meantime,  you can read the publications linked to this study:   and .

For any general questions about participation in our clinical research studies please contact our research nurses Dr Shamsher Khan shamsher.khan2@nhs.net or Mrs Katarina Manso katarina.manso@nhs.net