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STUDY NAME: A Study to Evaluate Safety and Preliminary Efficacy of EPI-321 in Adults With FSHD
ABBREVIATION: Epicrispr EPI-321 Study
STATUS: Recruiting
See on ClinicalTrials.govWho is conducting the clinical trial?
Epicrispr Biotechnologies is sponsoring this study.
What is the purpose of the clinical trial?
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.
How does the medicine work?
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.
Will some people be given a placebo instead of the medicine?
No. All participants will receive the study drug.
How will the medicine be given?
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.
How long is the clinical trial?
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.
What are the criteria to be included in the clinical trial?
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.
Do I need to have a genetic test to be included? If so, will this be provided?
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.
How will the study team determine whether I can take part in the study?
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.
What activities will I be asked to do?
How many study visits are there? Are they in-person or virtual?
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.
Will I have to stay overnight for any visits?
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.
Will the medicine be offered to patients once the clinical trial is over?
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.
Will I be reimbursed for travel costs?
Yes, travel expenses may be reimbursed. The study team will provide further details.
Who is paying for the clinical trial?
Epicrispr Biotechnologies is funding this study.
How many volunteers are needed?
The study expects to enroll 9 participants.
Where are the study sites that are conducting the clinical trial?
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 clinicaltrials.gov 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 clinicaltrials.gov 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.
