Epicrispr EPI-321 Study

Qui mène l'essai clinique ?

Epicrispr Biotechnologies is sponsoring this study.

Quel est l'objectif de l'essai clinique ?

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.

Comment le médicament agit-il ?

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.

Certaines personnes recevront-elles un placebo au lieu du médicament ?

No. All participants will receive the study drug.

Comment le médicament sera-t-il administré ?

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.

Quelle est la durée de l'essai clinique ?

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.

Quels sont les critères d'inclusion dans l'essai clinique ?

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.

Quels sont les critères qui m'excluraient de l'essai clinique ?

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.

Dois-je subir un test génétique pour être inclus ? Dans l'affirmative, ce test sera-t-il fourni ?

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.

Comment l'équipe de l'étude déterminera-t-elle si je peux participer à l'étude ?

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.

Quelles sont les activités qui me seront demandées ?

• Provide blood and other samples for laboratory testing
• Undergo a muscle biopsy at 3 different times in the study. This involves using a needle to draw a small piece of muscle, about the size of several pieces of long-grain rice. The procedure is done with a local anesthetic
• Passer des examens IRM du corps entier
• Effectuer une série de tests pour évaluer la force et la fonction musculaires.
• Répondre à des questionnaires sur les symptômes liés à la FSHD et sur la façon dont vous vous sentez.
• Perform tests to measure the health and strength of your heart and lungs

Combien de visites sont prévues dans le cadre de l'étude ? Sont-elles en personne ou virtuelles ?

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.

Devrai-je passer la nuit sur place lors des visites ?

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.

Le médicament sera-t-il proposé aux patients une fois l'essai clinique terminé ?

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.

Devrai-je payer les frais médicaux ? Les frais seront-ils facturés à mon assurance ?

No, costs related to planned study activities are expected to be covered by the sponsor. The study team will provide further details.

Les frais de voyage me seront-ils remboursés ?

Yes, travel expenses may be reimbursed. The study team will provide further details.

Qui finance l'essai clinique ?

Epicrispr Biotechnologies is funding this study.

Combien de volontaires sont nécessaires ?

The study expects to enroll 12 participants.

Où se trouvent les sites d'étude qui mènent l'essai clinique ?

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. Enrollment for this study is now complete.

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.

• Epicrispr Biotechnologies Partners with Springbok Analytics to Integrate AI Muscle Analysis in First-in-Human FSHD Study
• Epicrispr Biotechnologies franchit des étapes importantes dans le développement d'une thérapie révolutionnaire pour la FSHD