Thank you for all the great answers to my question. A friend of mine was in a clinical trial (for PV) and when it ended he said he didn't get the test drug,. I am not sure if it was phase 2 or 3. I need to check if he received HU or just placebo.

I've read many trial protocols. MPN patients are not left without treatment. In a phase 3 trial those not receiving the trial medication/treatment are given the best available treatment. I was in a phase 2 trial, where all participants are given the trial medication. If the 'patient' is benefiting from the trial medication, treatment continues indefinitely as additional trial periods are started at the end of a cycle. ie 28 week trial period. I was on the medicine for about four years.

The answer is no, since it is not considered ethical for some participants to only get a placebo.

@A myMPNteam Member Now I'm wondering if there are requirements set by the FDA or other organization with regards to trial participants continuing on a trial medication if they are benefitting. And are the rules different for phase 2 vs phase 3. I went on the trial because I had exhausted all, the limited?, treatment options. My body was saying no to them. I spoke with another trial participant about our ability to continue on the medication. She told me that we definitely would because if something happened to our bodies, to us, as a result of discontinuing the treatment, the 'sponsor', aka pharma company, could be held liable. When I started the trial, the lead investigator was Dr. Mesa. He told me I would be able to continue on the medication. Even so, I still had some worry. 🙃

I would not have been comfortable if the trial was phase 3 and I had a 50/50 chance of being placed on the best available treatment BAT. My body was not tolerating the BAT. I needed a new treatment.

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A myMPNte...

I asked GEMINI this question, and this was the answer. Before a drug reaches the public, it typically goes through four distinct phases. Each phase has a higher number of participants. Phase I is the Safety phase and has 20-100 participants. Phase II is the Efficacy phase with 100-300 participants. Phase III is the Comparison phase with 300-3,000. Phase IV is the Monitoring phase with thousands of participants. The “Add-on” Design: Sometimes everyone gets the standard treatment, but one group gets the new drug added to it, while the other gets a placebo added to it. This way, no one is left without care.

Key Protections for Participants:

If you join a trial, you aren’t just a “guinea pig.” You are protected by:
1. Informed Consent: You must be told exactly what the trial involves, including the risks and whether or not a placebo might be used.

2. IRB Oversight: An Institutional Review Board (a group of experts and citizens) must approve the study to ensure it is ethical.

3. Randomization: To prevent bias, a computer often randomly assigns you to a group. In “double-blind” studies, neither you nor your doctor knows which group you are in until the study ends.