General knowledge about clinical trials

       1. As the old cliché goes, oral hormone therapy has been the cornerstone of treatment for patients with DMD and is currently the most effective treatment method. Patients participating in clinical trials need to receive standard oral hormone therapy for at least 6 months in order to meet the basic requirements for enrollment. In other words, patients who are not receiving oral hormone therapy are no longer typical patients representative of this patient population. It has been proven that combination therapy with oral hormones at its core has taken the survival of DMD patients to another level. From an ethical point of view, a new trial drug can only add to the existing treatment. If oral hormone therapy is stopped in order to conduct a clinical trial of a new drug, it will have a negative impact on the patient’s condition and is not in line with ethical principles.  2, accept the clinical trial of the experimental drug, no matter how good the efficacy of this drug is in the preliminary trial and how bright the prospect of the future drug launch. The use of drugs during the clinical trial is completely free of charge. Not only is it completely free, but the cost of some related tests will also be completely free, and the patient will be subsidized to a certain extent for the cost of transportation and other aspects of participation in the clinical trial. This is the real clinical trial. A lot of domestic projects under the banner of clinical trials, but charging a lot of money, it can be said for sure, are “hanging sheep’s head, selling dog meat”.  3, clinical trials are not only benefit, not pay. In order to obtain accurate data on the effects of drug treatment, participation in clinical trials requires more tests than general treatment, including invasive tests. If you participate in this clinical trial, you will have a muscle biopsy under general anesthesia before and after the drug treatment, so there is a price to pay for the free treatment. In addition, in most clinical trials there is a control group, which is given no therapeutic drug but only a placebo (a non-therapeutic agent that is not apparently distinguishable from the therapeutic drug). For the sake of objectivity in clinical trial results, receipt of drug or placebo treatment is screened completely randomly, with a 50 percent to 50 percent chance. Not only do patients themselves not know whether they are receiving a drug or a placebo, but neither do the physicians who administer the treatment. Only at the end of the clinical trial, when the blind is removed, will we know. Therefore, it is entirely possible to go into a clinical trial with great enthusiasm and not receive a drug. Of course, for ethical reasons, after the clinical trial is unblinded, patients in the placebo control group will generally be given the relevant drug treatment to try to ensure their interests.  4. As mentioned above, at least two general anesthesia procedures are required to participate in this clinical trial. Some patients will need to have an “infusion port” placed and will receive additional general anesthesia. Again, genetically diagnosed DMD patients are not at risk for special anesthesia and are perfectly capable of receiving general anesthesia. If general anesthesia was a significant risk, such a trial design would never have passed ethical review. From overseas and our own experience, DMD patients undergoing major scoliosis surgery, cardiac surgery and various surgeries requiring general anesthesia do not have special risks higher than those of the general population.  5. During participation in clinical trials, subjects and families have the right to opt out at any time due to subjective and objective reasons. This is a basic right of the subject and is protected (of course, the trial drug treatment will be stopped after withdrawal). There are times when a subject has an adverse event and the physician decides to withdraw from a clinical trial, and these are explicitly stated in the trial design.  With the rapid development of precision medicine and gene therapy, we will see more new drugs and clinical trials, and we should treat clinical trials rationally.