As promised, I will now start trying to keep you updated on actively recruiting clinical trials in PSP. Keep in mind a few things before using my list:
- In compiling my list, I rely heavily on www.clinicaltrials.gov. Trials in the US are required to list themselves there, but some take longer than others to comply. Trials overseas may or not be required to have a listing on clinicaltrials.gov, depending on the country. My list also includes trials I’ve heard about through the grapevine that are not (yet) listed.
- In Phase 1 and sometimes in Phase 2, the trials do not typically allow patients completing the protocol to continue receiving the drug, no matter how well they were doing on it. Later Phase 2 and Phase 3 trials often do allow this, but they do not commit to it in advance. Typically, if the trial shows the drug to be ineffective (or unsafe) overall, the drug company will discontinue its program for that drug and stop producing it. That means that even the few patients who might have been doing well on it will not continue to have access to it.
- In theory, exceptions to that rule could exist for drugs that are being tested in both PSP and another disease such as Alzheimer’s. If the PSP trial shows lack of efficacy but acceptable safety, the company will continue to manufacture the drug pending the outcome of the other trial and may make it available to those who completed the PSP trial.
- For trials of neuroprotective treatment, patients may not know if they are benefitting. (Definitions: “Neuroprotective” drugs are intended to slow the progression of the abnormal brain process in the long term, as opposed to helping the symptoms experienced by the patient in the short term. For example, for infections, antibiotics are protective, while painkillers are symptomatic.) To know if an individual in a neuroprotective trial has had slowing of their rate of worsening over the 12 months on the drug, their rate of worsening over at least a few months before the trial would have to have been quantitatively assessed using the same method that the trial used. We almost never have that data, so trials work by comparing the average result from a group receiving the drug to the average from a group receiving a placebo.
- Do not expect a neuroprotective drug to improve your symptoms. At best, it could prevent worsening, and more likely would only slow the rate of worsening. The trials are typically designed to detect a slowing of the rate of worsening of 30% or 40%.
- Bottom line: Participating in a clinical trial, especially one in Phase 1 or 2, requires some level of altruism.
- For more information on these trials, go to www.clinicaltrials.gov and enter the drug’s name or ID number shown in the first column.
Here’s the current list to the best of my knowledge. The ones at the bottom labeled as “may start” are awaiting funding in most cases.
| Drug / clinicaltrials.gov ID | Sponsor | Phase | Mechanism | Location(s) | Comments |
| TPN-101 NCT04993768 | Transposon | 2a | Reduces tau production | Boca Raton, FL Farmington Hills, MI | 30 patients on drug, 10 on placebo |
| RT001 (di-deuterated linoleic acid ester) NCT04937530 | Retrotope | 2a | Reduces lipid peroxidation, enhancing mitochondrial activity | University of Munich (Germany) | Non-controlled study showed very slow PSP progression over 2 years. |
| NIO-752 NCT04539041 | Novartis | 1 | Anti-sense oligonucleotide; reduces tau production | Rochester, MN; Nashville, TN; Montreal; 3 in Germany; 1 in UK | Requires 4 injections into spinal space over 3 months. |
| AZP2006 NCT04008355 | AlzProtect | 1 | Reduces tau production | 3 sites in France | ~24 patients on drug, ~12 on placebo |
| May start recruiting in the next year: | |||||
| ASN120290 | Asceneuron | 1 | Reduces tau misfolding and aggregation by inhibiting O-GlcNAcase | ? | Press release info only |
| MP201 | Mitochon | 1 | Mitochondrial decoupler | ? | Early in planning per press release |
| Tolfenamic acid | NeuroTau | 2 | NSAID that reduces tau production | Cleveland Clinic, Las Vegas + others? | |
| Sodium selenate | Gov’t of Australia | 2 | Enhances dephosphoryl-ation of tau by protein phosphatase 2 | Multiple, in Australia |
PSP Blog 
Dear Dr. Golbe, I understand that your summary above includes only those PSP trials that are either currently recruiting or hope to begin recruiting soon. That said, would you be willing to share your thoughts about the currently ongoing trial of Fasudil (Rho Kinase Inhibitor) for PSP and CBD patients at UCSF https://clinicaltrials.gov/ct2/show/NCT04734379?term=fasudil&draw=2&rank=8 ? The estimated study completion date is July 30, 2022. Does that typically mean that we may learn more about its results within a few months of that date? I hope that it is not inappropriate for us to ask you to comment about an ongoing trial in this way. If so, we understand completely, and thank you very much one way or the other.
Dear mauraelisabeth3:
All I know is that the fasudil trial has recruited its full complement of 15 patients. No information is available on when the trial will be completed, but I have no reason to doubt the published date of July 30, 2022. Then, it will take several months for data “cleaning” and analysis, and then the drug company sponsoring the trial may wait longer to announce the result. Keep in mind that this trial is only a small, preliminary study of safety and tolerability, and of the feasibility of the outcome measures. With only 15 patients, there is not enough “statistical power” to detect benefit. Before the drug can become widely available, at least one larger trial designed to measure efficacy against PSP will be necessary. Based on my experience, that could start in late 2022 or early 2023 and end two years later. But such a trial won’t happen at all if the company decides for some reason (such as insufficient finances, competitive environment, borderline safety results) not to pursue this drug regardless of the current trial’s outcome. Fingers crossed and keep up hope!
Thank you very much, Dr. Golbe, for taking the time to share these insights into the clinical trial process. They really help.