About this PKAN study

What is the FORT Study?
The FORT (FOsmetpantotenate Replacement Therapy) Study will assess how well an investigational study drug, called fosmetpantotenate, works in children and adults with pantothenate kinase-associated neurodegeneration (PKAN). ‘Investigational’ means that the study drug has not been approved for the treatment of PKAN, and is still being evaluated in clinical studies such as this one. The investigational study drug will be compared with a placebo – a substance that looks like fosmetpantotenate but contains no real treatment. The FORT Study will also assess whether there are any side effects from taking the investigational study drug.

Why is the FORT Study taking place?

There are currently no approved medicines to treat the underlying causes of PKAN. Treatment options for people with PKAN include medicines that relieve symptoms. While these help people feel better, they do not slow disease progression. The FORT Study will investigate whether fosmetpantotenate can treat the cause, not just the symptoms, of PKAN. By targeting what we think is a cause, it is hoped that the investigational study drug could help improve symptoms and slow the progression of PKAN.

What will the FORT Study help us learn?

The study aims to discover whether there are any changes in your/your child’s signs and symptoms of PKAN after taking the investigational study drug. Daily activities, such as speaking, walking, bathing, dressing, eating and swallowing, will be assessed.


Who can take part?
Approximately 82 people in study clinics across North America and Europe will take part in the FORT Study. Not everyone will be able to join the study as several inclusion criteria must be met.

Which study drug will I receive?
If you/your child are eligible to take part in the FORT Study, you/your child will be placed into one of two study treatment groups. This is decided at random, like the toss of a coin. About 50% of participants will be assigned to the fosmetpantotenate group, and about 50% will be assigned to the placebo group. This is decided at random, like the toss of a coin. Neither you, your child, nor the study doctor will know to which study treatment group you/your child have been assigned. This ensures that the results from the different groups will be handled in the same way.


How often will I take the study drug?
Following an initial dose escalation, you/your child will receive three doses, approximately 8 hours apart. You/your child will take three doses per day with meals for the rest of the study. Both the investigational study drug and placebo are supplied as a dry powder, which should be added to water and swallowed. Details of how and when to take the investigational study drug or placebo will be given by the study team.

How long will the study last?
Excluding the screening process, participation in the study could last about 120 weeks. During this time, you/your child will need to visit the study clinic approximately 20 times. Below outlines each step of the study:

  • Pre-screening – participants taking deferiprone for PKAN will need to discontinue treatment for at least 30 days prior to Screening. This is to ensure that the effects of deferiprone do not interfere with the investigational study drug.
  • Screening – to assess whether you/your child are suitable to take part, you/your child will be asked to attend a Screening visit at the study clinic to undergo tests and assessment. This visit takes place up to 4 weeks before Part 1
  • Part 1 – if eligible to take part, you/your child will be asked to return to the study clinic to undergo further study-related tests and receive either fosmetpantotenate or placebo. You/your child will be required to travel to the study clinic 6 times over 24 weeks. During the 1st week of the study, you will visit the clinic each day (e.g. V2-V6). The other visits are spaced weeks apart. There are 10 study office visits in total.
  • Part 2 – volunteers who choose to participate in Part 2 of the study will undergo a variety of tests and receive fosmetpantotenate only (no placebo). Participants taking placebo in Part 1 of the study will have the opportunity to receive the investigational study drug. You/your child will be required to visit the study clinic 10 times for up to 96 weeks. Thirty days after the last dose of the investigational study drug, the study team will contact you to check on your health and well-being.


If you/your child do not continue to Part 2 of the study, or if you/your child leave the study before the end of Part 1, you/your child will be asked to return to the study clinic for final assessment.

What happens during visits to the study clinic?
During visits to the study clinic, the study team will ask you/your child several questions and perform a number of study-related medical tests. The purpose of these tests is to assess your/your child’s health and determine how your body is responding to the investigational study drug.


Some of the tests will be recorded on video to ensure that they are performed correctly. Prior to any procedures, you/your child must agree to and sign an Informed Consent Form. This document provides details about the study, what participation entails, and how the clinical information will be used.

The assessment schedules for Part 1 and Part 2 provide a more detailed overview of what happens at each visit.


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