A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
This research study is looking at a medication called vatiquinone for people living with Friedreich Ataxia. It's specifically for individuals who have previously taken part in another vatiquinone study. The main goal is to check the long-term safety of the medicine and see if it continues to be helpful over a longer period. This study allows participants to continue receiving vatiquinone, addressing a need for ongoing treatment. Researchers will be monitoring the effects of the medication over about three years. If you've been in a vatiquinone study before, this might be a way to keep receiving the treatment and contribute to understanding its long-term benefits.
At a glance
What is this study about?
This study is about a medicine called vatiquinone, which is being investigated for people who have a condition called Friedreich Ataxia. Friedreich Ataxia is a rare inherited disease that affects the nervous system, causing problems with movement and coordination.
The main aim of this study is to look at the long-term safety of vatiquinone. Since you would have been in a previous study with this medicine, the researchers want to continue watching how it affects you over a longer period. They also want to see if it continues to help with the symptoms of Friedreich Ataxia.
This study is designed to offer continued access to vatiquinone for people who have already used it in a clinical trial. This means that if you found the medicine helpful, you might be able to carry on taking it for about three years, while doctors continue to gather important information about its effects. This helps doctors understand if the medicine is a good long-term option for people with Friedreich Ataxia.
Key takeaways
- This study is for people with Friedreich Ataxia who have already used vatiquinone in a past study.
- It aims to check the long-term safety and effectiveness of vatiquinone over about three years.
- Participation means continued access to the study medicine and regular health check-ups.
- Contraception is required for men and women who could have children.
- You cannot be taking part in another treatment study or be pregnant/breastfeeding.
Who may be eligible?
To join this study, you must have Friedreich Ataxia and have already completed a previous research study involving the medicine vatiquinone. It's important that you move directly from that previous study into this one.
If you are a man or woman who could have children, you must agree to use effective contraception (birth control) throughout the study and for 30 days after your last dose of the medicine. This is to prevent pregnancy during the study.
You cannot join this study if you are already taking part in another clinical trial where you are receiving a treatment. Also, you cannot be pregnant or breastfeeding.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Do I have Friedreich Ataxia?
- Have I already completed a previous study with vatiquinone?
- Am I willing to use effective birth control if I could have children?
- Am I currently pregnant or breastfeeding?
- Am I participating in any other treatment study right now?
What does participation involve?
If you decide to take part, you will be given the study medicine, vatiquinone, which you would have taken in a previous study. You will continue to have regular check-ups with the study team over approximately three years. These visits will involve various tests and assessments to monitor your health, how the medicine is working, and to check for any side effects. The study team will explain exactly what each visit involves, but it's important to remember that this is a long-term commitment, likely involving regular trips to the clinic or hospital.
Potential risks and benefits
Locations (14)
- UCLAVerified postcodeLos Angeles, United States
- University of FloridaVerified postcodeGainesville, United States
- University of South FloridaVerified postcodeTampa, United States
- University of IowaVerified postcodeIowa City, United States
- The Children's Hospital of PhiladelphiaVerified postcodePhiladelphia, United States
- Murdoch Children's Research InstituteVerified postcodeParkville, Australia
- University of Campinas (UNICAMP) - School of Medical Sciences, Dept of NeurologyVerified postcodeSão Paulo, Brazil
- Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)Verified postcodeMontreal, Canada
- CHU Sainte-JustineVerified postcodeMontreal, Canada
- Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)Verified postcodeParis, France
- Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)Verified postcodeTübingen, Germany
- Ospedale Pediatrico Bambino Gesu' IRCCSVerified postcodeRoma, Italy
Common questions
What is Friedreich Ataxia?
Friedreich Ataxia is a condition that affects the nervous system, causing problems with balance, coordination, and movement.
What is vatiquinone?
Vatiquinone is a medicine being studied to see if it can help people with Friedreich Ataxia.
Will I get the actual medicine or a dummy pill?
Because you've been in a previous study and this is continuing access, you will receive the actual medicine, vatiquinone.
How long will the study last?
This study is planned to last for about three years.
Can I leave the study if I change my mind?
Yes, you can leave the study at any time, and you don't have to give a reason. Your medical care won't be affected.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
Community discussion
Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.