Share your perspective on PML clinical trials

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1. 

a. Awareness. When my father was diagnosed with PML he was left to die. He was told there were no treatments available, nothing they could do. He was not informed of any potential clinical trials. So that would be the first aspect - awareness of an opportunity to participate. Neither the neurologist nor the ID doctor mentioned that a clinical trial could be available to him. 

b. Clinical visits are minimal or virtual. Visits should be arranged by the trial coordinating center or they send a nurse to him to collect blood or do other tests. My father is paralyzed and bedbound so any transport would require ambulance travel. We would need this to be arranged by the trial coordinating center with his nursing home. We can't just go pick him up in our car and take him to a doctor/hospital/lab. Even moving him from the bed into a wheelchair is a long, strenuous process. The family/caretakers already have too many other things to manage and arrange as it is, we don't have the capacity to also try to add in visits for a clinical trial. This would be the same for medical records. If the PI requests them through his facility that is much easier than the family having to go through the process and sending them. 

c. There was a delay in diagnosis for my father's PML. So the damage had been done. So trials that we would have him participate in would have to be for compounds/therapies to treat the damage done to his nerves. I can only imagine this would be stem cell treatment or other cellular therapies. It is too late for him to receive any treatment for the JC virus, as his immune system has now been stabilized from his HIV medication. Other trials he might consider are for the muscle spams or pain. He might consider trials that test certain assistive devices or methods of physical therapy. But at this point in his disease experience, an antiviral treatment would not be needed/effective. 

d. My father's qualityof life is so poor now that I don't think concerns about adverse events would be a high priority. I realize many people might say they would want to participate in a trial only if it is safe. But he is already severely ill and has many health problems. I think if there was potential for a treatment to help then he would be willing to try it. I believe he would be willing to participate even if he could potentially be in a placebo arm, since being a trial would give him access to more testing/evaluation of his condition and potentially more face time or contact with PML experts, which he has not been able to get so far. 

 

2. Generally, the only thing that would prohibit participation would be if the trial required him to make frequent in-person visits to a clinic. He is bedbound and cannot leave his facility without great trouble to him, his family, and the facility staff. There is a cost for ambulance transport that is not covered by Medicare. He also is paralyzed and unable to write, fill out any forms (by hand or on a tablet). So any PROM questionnaires would need to be administered by study staff.

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Idea No. 113