谁在进行临床试验？
Epicrispr Biotechnologies is sponsoring this study.
临床试验的目的是什么？
This study is testing the safety and early effects of a new treatment called EPI-321 in adults with facioscapulohumeral muscular dystrophy (FSHD). It is the first study to test EPI-321 in people.
药物如何发挥作用？
EPI-321 is a gene therapy designed to stop the expression of the DUX4 gene in muscle, which scientists believe causes muscle damage in people with FSHD.
In people without FSHD, special markers called methyl groups help turn off the DUX4 gene. This keeps the DUX4 protein from building up. In people with FSHD, there aren’t enough of these markers, so too much DUX4 protein is made, which can harm muscles. EPI-321 is a treatment that helps add these markers back. This is called epigenetic editing, it helps turn the gene off without changing the DNA.
Gene therapy is a new kind of treatment. Scientists believe this could be a one-time treatment but are still studying how well it works and how long it lasts.
是否会给一些人服用安慰剂而不是药物？
No. All participants will receive the study drug.
如何给药？
The study drug will be given just one time by intravenous infusion. An intravenous infusion is when medicine is slowly given through a small tube placed in a vein, usually in your arm.
临床试验需要多长时间？
The main part of the study lasts about one year. The study team will continue tests and checkups for ~5 years after receiving EPI-321.
参加临床试验的标准是什么？
Participants must be adults (18–75 years old) with a confirmed genetic diagnosis of FSHD1. As determined by the study team, you must have a certain amount of weakness in your shoulder and arm muscles and/or a certain amount of weakness in your hip and leg muscles; however, you must still be able to walk approximately 30 feet.
What criteria would exclude me from the clinical trial?
You cannot participate if you weigh over ~200 pounds, have other health issues that could affect your safety, or if you are pregnant or breastfeeding.
我是否需要进行基因测试才能加入？如果需要，是否会提供？
Yes, you must have a genetic test. If you don’t have one, the study will be able to provide the test at no cost to you.
研究小组如何确定我能否参加研究？
You will first have a discussion with the study team to check your likely eligibility. If you qualify, you’ll be scheduled for a screening visit with physical exams, lab tests, and other assessments.
我需要参加哪些活动？
研究访问有多少次？是面对面的还是虚拟的？
Participants will be followed for approximately 5 years after being administered EPI-321, and study visits are in-person at the clinical trial site. There are 17 planned study visits after receiving the study treatment. Nine of these visits occur at least weekly in the first 6 weeks after EPI-321 dosing. Thereafter, study visits are planned at the following months after EPI-321 dosing: 3, 6, 12, 18, 24, 36, 48, and 60.
我是否需要在探访时过夜？
Yes, you will stay 48 hours at the study site after receiving your infusion and will need to remain within about an hour’s drive of the study site for the first 2 weeks after infusion.
What are the risks of taking part in the clinical trial?
As this is a first-in-human study, the risks are not fully known. The study team will discuss the potential risks with you, and you can ask as many questions as needed.
What are the benefits of taking part in the clinical trial?
You may not receive direct benefit. However, your participation helps researchers learn whether EPI-321 is safe, and potentially effective, and may lead to future treatments for FSHD.
临床试验结束后，会向患者提供这种药物吗？
EPI-321 is designed to be a one-time therapy. Thus, for eligible patients enrolled in the clinical trial, the EPI-321 study treatment is only dosed once, and no additional doses will be given after that one-time.
For patients not enrolled in the clinical trial, EPI-321 is not available at this time.
This is the first time that EPI-321 is studied in people, and the main purpose of this trial is to test safety. Future trials will determine if the drug is effective for wider use.
Will I have to pay for any medical expenses? Will my insurance be billed for any costs?
No, costs related to planned study activities are expected to be covered by the sponsor. The study team will provide further details.
我能报销差旅费吗？
Yes, travel expenses may be reimbursed. The study team will provide further details.
谁来支付临床试验的费用？
Epicrispr Biotechnologies is funding this study.
需要多少志愿者？
The study expects to enroll 12 participants.
开展临床试验的研究机构在哪里？
The study is being conducted in multiple countries and currently sites in the United States and New Zealand are open to enrolling participants. You can find study locations at the 临床试验 website. This website will be updated when new study sites open.
How do I contact a study site to learn more and sign up?
Go to the 临床试验 page and scroll down to “Contacts and Locations” to find phone numbers and emails for each study site.
What will happen after I contact a study site?
It may take a few weeks for the site to get back to you. If you meet the basic criteria, they’ll discuss scheduling a screening visit with exams and tests to see if you can enroll.
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