1 What causes PKAN?
Pantothenate kinase-associated neurodegeneration (PKAN) affects only 1–3 people in every million (a total of approximately 5000 people worldwide). Because of a defective gene, the bodies of people with PKAN cannot make enough of a substance called pantothenate kinase 2 (an enzyme). This enzyme is needed to turn vitamin B5 (found in some vegetables, eggs, and meat) into another substance called Coenzyme A in the body. A lack of Coenzyme A is believed to cause neurodegeneration and all of the symptoms of PKAN, such as movement disorders.
2 How does the investigational study drug work?
The investigational study drug, fosmetpantotenate, is a replacement therapy – a treatment that aims to restore the lack of a substance normally present in the body. People with PKAN lack sufficient quantities of the pantothenate kinase 2 (PANK2) enzyme. The investigational study drug is designed to deliver a product of PANK2, called phosphopantothenate, to cells. This could help restore the PANK2 enzyme, which in turn can increase levels of Coenzyme A – proteins that activate important chemical reactions within the body.
3 Will there be side effects from the investigational study drug?
All medications may cause side effects. The potential and known side effects of fosmetpantotenate are listed in the Informed Consent Form and will be discussed further with your study doctor. If you/your child experience any discomfort during the study, contact the study doctor at any time to discuss the best course of action. Throughout the study, your/your child’s health will be monitored continuously. The study treatment will be stopped if you or the study doctor have any concerns.
4 Can I keep taking my current medication(s)?
With the exception of deferiprone, you/your child will be allowed to continue taking most medicines for PKAN throughout the study. If you /your child are currently prescribed deferiprone but are willing to stop taking it, please speak to your doctor about the most suitable withdrawal plan.
The study team will confirm which medicines are allowed and prohibited. Do not start taking any new medicines*, modify Deep Brain Stimulator settings if using one, or change the dose of current medicines without first discussing this with your/your child’s study doctor. If you/your child need emergency treatment during the study, tell the doctors or nurse that you/your child are taking part in this study and contact the study team as soon as possible.
*Including prescription and over-the counter treatments, pain medication, vitamins, herbal supplements, acupuncture, and physical therapies.
5 How will I know if I am eligible for this study?
To find out whether you/your child can take part in the FORT Study, visit the Can I join? page. If you/your child meet the criteria, take the pre-qualification screener or call our study team on 1-844-363-1866. If you/your child pre-qualify for the FORT Study, you will be referred to your nearest study clinic. At this visit, the study doctor will ask a number of questions and perform medical tests to see whether you/your child are right for the study and the study is right for you/your child.
6 Where are the study clinics?
The study clinics are located across North America and Europe. Find a participating study clinic near you.
7 What if I want to leave the study early?
It is voluntary to join the study. You/your child can leave at any time for any reason without having to explain why. You/your child will retain the right to the same standard of care previously received. If you/your child decide to leave before the end of the study, you will be invited to attend the study clinic for final tests and assessment.
8 Do I need to ask my regular doctor before participating in this study?
No. However, you should tell your/your child’s regular doctor that you are thinking of participating in this clinical research study. Your/your child’s doctor may wish to contact the study doctor (with your permission) to request additional information.
9 Will I have to switch doctors?
No. The FORT Study will provide short-term study-related care only. Clinical research studies do not provide extended or comprehensive primary health care. Your/your child’s family doctor may wish to work with the study team to best determine which treatments or medications are most appropriate for you/your child. At the end of the study, your/your child’s care will return, in full, to your regular doctor.
10 Will I be compensated for my participation in this study?
You will not receive payment for taking part in this study. However, the investigational study drug and all study-related care will be provided without charge. Expenses associated with taking part in this study, for instance travel to/from the study clinic, may be reimbursed. Please refer to the Informed Consent Form for further details.
11 Who will have access to the information collected during the study?
Your/your child’s personally identifiable information (PII: eg name and address) will not be accessible to anyone who is not directly associated with this study, except with your permission or as required by law. The Sponsor (the company carrying out the study) will not have access to any PII submitted through this website. Any information gained from this study may be used for publishing results. However, this information will be combined with other participant data and will not be used to identify participants.