Epicrispr EPI-321 Study

¿Quién lleva a cabo el ensayo clínico?

Epicrispr Biotechnologies is sponsoring this study.

¿Cuál es el objetivo del ensayo clínico?

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.

¿Cómo actúa el medicamento?

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.

¿Se le dará a algunas personas un placebo en lugar del medicamento?

No. All participants will receive the study drug.

¿Cómo se administrará el medicamento?

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.

¿Cuánto tiempo dura el ensayo clínico?

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.

¿Cuáles son los criterios para participar en el ensayo clínico?

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.

¿Qué criterios me excluirían del ensayo clínico?

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.

¿Necesito hacerme una prueba genética para participar? Si es así, ¿me la proporcionarán?

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.

¿Cómo determinará el equipo del estudio si puedo participar en él?

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.

¿Qué actividades se me pedirá que realice?

• 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
• Someterse a resonancias magnéticas de cuerpo completo.
• Realice una serie de pruebas para evaluar su fuerza y función muscular.
• Responda cuestionarios sobre los síntomas relacionados con la FSHD y cómo se siente.
• Perform tests to measure the health and strength of your heart and lungs

¿Cuántas visitas de estudio hay? ¿Son presenciales o virtuales?

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.

¿Tendré que pasar la noche en el hospital para alguna visita?

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.

¿Se ofrecerá el medicamento a los pacientes una vez finalizado el ensayo clínico?

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.

¿Tendré que pagar algún gasto médico? ¿Se le cobrarán los gastos a mi seguro?

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

¿Me reembolsarán los gastos de viaje?

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

¿Quién paga el ensayo clínico?

Epicrispr Biotechnologies is funding this study.

¿Cuántos voluntarios se necesitan?

The study expects to enroll 12 participants.

¿Dónde se encuentran los centros de estudio que están llevando a cabo el ensayo clínico?

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 ensayos clínicos.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 ensayos clínicos.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 se asocia con Springbok Analytics para integrar el análisis muscular con IA en el primer estudio sobre FSHD en humanos.
• Epicrispr Biotechnologies alcanza hitos en el desarrollo de una terapia innovadora para la FSHD