Study Overview

Gender
Male
Age
4 - 7
years
Status
Recruiting
Phase
Phase III

The study will evaluate the efficacy of systemic gene delivery in approximately 120 boys aged 4-7 with DMD. This is a randomized, double-blind, placebo-controlled study of SRP 9001, of which the total duration of participation is expected to be approximately 2 years. 

Purpose of Study

embark boy EMBARK is a phase III clinical study to evaluate the safety and effectiveness of an investigational gene delivery drug in boys aged 4-7 with Duchenne muscular dystrophy. 

Duchenne muscular dystrophy (also referred to as Duchenne or DMD) is a genetic disorder that primarily affects males and is caused by a specific genetic mutation (error) in the gene that codes for dystrophin. 

Dystrophin is a protein that plays a key role in the function of muscle cells and protects them from damage as muscles contract and relax. These mutations in the dystrophin gene lead to a lack of dystrophin protein in muscles. Without enough dystrophin, muscles gradually grow weaker.

There is currently no cure for Duchenne, but there are medicines and other therapies that can ease symptoms and protect muscles. Researchers are currently investigating ways to slow the disease progression. One approach is gene therapy. This type of therapy adds a new gene to the body, with the hope of treating the disease.
 

About SRP-9001 gene therapy

The EMBARK Study is testing an investigational drug, called SRP-9001 (scientific name delandistrogene moxeparvovec), to see whether it is safe and effective in individuals with Duchenne. SRP-9001 is a gene therapy treatment being developed to treat the cause of Duchenne. The aim of gene therapy for Duchenne is to add a shorter form of dystrophin to the muscle cells. This is referred to as micro-dystrophin. Once the micro-dystrophin gene is inside the muscle, researchers believe it may teach the cells to make micro-dystrophin protein in order to promote muscle function. 

How SRP-9001 gene therapy work:

SRP-9001 gene therapy comprises three main building blocks: a vector, promoter, and transgene. It is given as a one-time administration by intravenous (IV) infusion – a small tube inserted by a needle will deliver a slow ‘drip’ of study medication into a vein in the arm.

To better understand the building blocks of SRP-9001, picture a ship out at sea heading toward land. Like the ship, when SRP-9001 is introduced to the body it is heading toward certain cells.

embark cargo boat

  1. The vector is the ship itself, it carries the micro-dystrophin through the body and delivers it into the muscle cell like a ship reaching land.
  2. The vector used in this study is known as rAAVrh74
  3. The micro-dystrophin transgene is like cargo inside a ship. It is a gene made of DNA that teaches the muscle cells how to make micro-dystrophin protein. The transgene is being carried inside the vector to the muscle cell.
  4. The promoter functions like the captain of a ship. Like a captain commanding orders, the promoter instructs the muscle cells to produce micro-dystrophin protein.
vector-promoter-transgene

 

About the Study

Study Name: EMBARK

Study Official Title: A Phase 3 Multinational, Randomized, Double-Blind, Placebo‑Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP‑9001 in Subjects with Duchenne Muscular Dystrophy (EMBARK)

Study Number: SRP-9001-301

ClinicalTrials.Gov: https://clinicaltrials.gov/ct2/show/NCT05096221

Status: Enrolling

Location: United States (active), United Kingdom, Belgium, Spain (active), France, Germany, Italy, Hong Kong, Taiwan, Japan

embark global locations journey us locations

For a full list of trial locations please go to ClinicalTrials.gov and search NCT05096221 (https://clinicaltrials.gov/ct2/show/NCT05096221?term=9001-301&draw=2&rank=1) or you can email SareptAlly@Sarepta.com to find a trial location nearest to you.

*Important Note: Please note that travel restrictions may apply. This study does not allow for cross-border participation, except under certain circumstances. Exceptions may be made on a case-by-case basis. Please discuss this with your doctor and reach out to SareptAlly for additional questions.

 

Participate

boy with doctorApproximately 120 participants in 40 study centers worldwide are expected to take part.

Study Eligibility:

EMBARK is enrolling boys age 4 to under 8 years of age who:

  • Are ambulatory (The definition of ambulatory will vary from trial to trial. The criteria for ambulatory for this study will be discussed during the screening process.)
  • Have been diagnosed with Duchenne based on clinical findings and prior genetic testing.
  • Have been on a stable dose of oral corticosteroids for at least 12 weeks prior to the Screening visit.
  • Do not have antibodies to rAAVrh74.
  • Have a parent, legal guardian or caregiver who can accompany him at each visit and offer support throughout the study.

There are additional requirements for participation that will be reviewed with you and your child during the screening process.

SareptAlly

SareptAlly is a clinical trial patient matching service for Sarepta studies.

If you are interested in considering participation click here and schedule a call with a SareptAlly Patient Navigator who will help you identify the best clinical trial option and study site location for you and your child, now or in the future.

Schedule a call with a SareptAlly Patient Navigator »

EMBARK Procedures

EMBARK Screening Visit:

Patients who are selected to screen for the EMBARK Study will meet with the study physician and their research team to discuss the study in greater detail and answer questions about participation.

If agreeable, potential patients will be consented and enter the screening period, which could consist of several visits over 31 days.  During the screening period, the study physician will collect medical information on the patient and perform several tests to assess eligibility.   

Assessments include:

blood work

 

Blood work
 

review medical history

 

Review of medical history
and medications

performing functional assessments

 

Performing some functional
assessments

If the results of the screening visit show that the patient is eligible, he will be invited back to the study center for study participation.

EMBARK Study Visits:

infusion

 

In total, participants will receive two infusions (SRP-9001 or placebo), one at the beginning of the study (Year 1), and one at the beginning of the second year of the study (Year 2).

site visits

 

There are approximately 50 visits over the course of the two years. About 37 of these visits will be performed in-person with the study doctor and staff. Other visits will be performed over the telephone.

blood vials

 

Participants will visit a study center for infusions, MRI scans, 2 muscle biopsies, and periodic blood draws. Please note, only select sites will be conducting MRI scans and biopsies.

 

study center

 

Functional movement assessments (standing, walking, jumping tests), vital signs, and physical examinations will also be performed at the study center.

questionaires

 

Completion of Duchenne related questionnaires, and usage of wearable devices for tracking movement, will also be required for this study.

calendar icon

 

Additional visits may be scheduled if required – this will be decided by the study doctor. More information on the schedule of study visit activities will be provided by the study center.

 

What is randomized placebo-controlled study?

Randomized, placebo-controlled means that each study participant will be picked randomly, by chance (like tossing a coin) to receive either active SRP-9001 study drug or “Placebo.” Placebo is made to look just like the study drug, but it will not contain any active drug. Neither participant nor the study doctor will know if they have been assigned the active study drug or placebo. During the first year of the study, participants will have a 50% chance of receiving active SRP-9001 study drug. All eligible participants will have received active SRP-9001 study drug by the start of the second year of the study.

Researchers use a placebo to compare how safe and how well the study drug works in individuals treated with the drug, versus those receiving placebo and standard of care. This study, like others in Duchenne, utilize a placebo group because it is considered a gold standard from regulatory agencies who may eventually decide whether the drugs are safe and effective.

How will the study drug be given?

Participants will receive an infusion at Day 1 of the first year of the study, and at Day 1 of the second year of the study.  The infusion will either contain active SRP-9001 study drug or placebo based on the randomization assignment at the start of the study.  Both SRP-9001 and placebo will be given as a single intravenous infusion – a small tube inserted by a needle will deliver a slow ‘drip’ of study medication into a vein in the arm. The infusion will last approximately 1–2 hours.  After the infusion, the study team will monitor the participant for at least 6 hours.  

 

Year 1; Day 1

Year 2; Day 1

boy icon  

Randomized to active SRP-9001

SRP-9001

Placebo

boy icon 2 

Randomized to Placebo

Placebo

SRP-9001

During the first year of the study participants will have a 50% chance of receiving active SRP-9001 study drug. All eligible participants will have received active SRP-9001 study drug by the start of year 2.

Frequently Asked Questions

  • What is a placebo and why are they used?

    Placebo is made to look just like the active study drug, but it will not contain any active substance. Researchers use a placebo to compare how safe and how well the study drug works in individuals treated with the drug, versus those receiving placebo and standard of care. This study, like others in Duchenne, utilize a placebo group because it is considered a gold standard from regulatory agencies who may eventually decide whether the drugs are safe and effective.

  • Why is it important to study gene therapies over a long period?

    Gene therapies are designed to be long-lasting. Because we do not know how long a single SRP-9001 infusion will remain active, it is difficult to predict how people will respond. Study participants are therefore assessed over several years, to see whether they develop any complications after the infusion. Evaluating study participants over a long period can also protect the community. For example, finding any safety issues early could prevent other people from experiencing unwanted side effects from SRP-9001. Even if SRP-9001 is approved for general use, it will still need to be studied over years (or even decades) for researchers to truly understand its effects.

  • Are clinical research studies safe?

    Clinical research studies are performed according to strict government and ethical guidelines. These guidelines help to ensure that participants’ rights are protected while information about the investigational drug is collected. All study participants are supported by a dedicated team of healthcare professionals, each of whom is committed to the safety and well-being of those involved.

  • What is an antibody and why would my child have antibodies to rAArh74?

    Antibodies are proteins in the blood that the body develops to help fight off an infection. Antibodies are an important part of building immunity. Once they develop, antibodies often remain (to some extent) in a person’s body and could help fight off the same or similar infection in the future.

    A participant might not be eligible for EMBARK due to having antibodies to rAAVrh74. Because AAVs are found in nature, it’s possible that a person has been exposed to the AAV virus and formed antibodies. This is known as seroprevalence. If that’s the case, antibodies will be present prior to delivering SRP-9001 gene therapy. These antibodies can hinder the effectiveness of SRP-9001 and pose a safety concern if the immune system recognizes the vector as a virus and attacks it, similar to how the immune system works against naturally-occurring viruses.

    Participants will be tested for antibodies to rAAVrh74 via blood work during the screening process. To learn more about antibodies and seroprevalence click here

  • Will we be compensated for taking part in this study?

    Participants and family members will not be paid for taking part in this study. However, transportation related to participation, accommodations, and all reasonable expenses will be reimbursed. Further details regarding travel arrangements and reimbursement will be provided at the study site.

  • Can we travel to another country to participate?

    This study does not allow for cross-border participation, except under certain circumstances. Exceptions may be made on a case-by-case basis. Please discuss this with your doctor and reach out to SareptAlly at SareptAlly@Sarepta.com, or schedule an appointment with a SareptAlly Patient Navigator for additional questions and assistance.

  • Do I need to ask my child’s regular doctor before participating in this study?

    We encourage you to inform your child’s regular doctor that you are taking part in this study. There are some medications that are prohibited to take while on the study and the doctor may wish to contact the study team (with your permission) to request additional information.

  • Will we have to switch doctors?

    No. The EMBARK Study provides study-related care only.

    Clinical research studies do not provide extended or comprehensive healthcare. At the end of the study, study participants will return, in full, to their regular doctor for care.

  • Are there side effects from SRP-9001?

    Because SRP-9001 is still in clinical development, there may be side effects that are not known at this time. Participants may also experience side effects from the medical tests and procedures. During the informed consent process, the study team will discuss potential side effects and discomfort that may occur while on the study.

    If a study participant does experience side effects, contact the study doctor at any time to discuss the best course of action.

  • What happens at the end of the EMBARK Study?

    At the end of the two years of the study, participants can enter a long-term extension, which will assess the safety and effectiveness of SRP-9001 over a 3-year period. Further details will be given to you by the study team.

  • Can we leave the study early?

    It is voluntary to join the study. Participants can leave the study at any time without giving a reason. Your decision will not affect any future medical care that he will receive. However, once the participant has received the infusion, it is in his best interests to allow the study doctor to continue to monitor his health for the remainder of the study.

  • Who will have access to the information collected during the study?

    Personal identifiable information, such as names and addresses, will not be given to anyone who is not directly associated with this study, except with your permission or as required by law. Any information gained from this study may be used for publishing results. However, this information will be combined with other data and will not be used to identify participants.

  • How do we take part?

    If you think your child may be suitable for the EMBARK Study, you can schedule a meeting with a SareptAlly Patient Navigator to learn more and see if your child is eligible. If you have general questions about the trial, trial locations, or participation, you may also contact the Patient Navigator at SareptAlly@Sarepta.com.

  • What should we consider before taking part?

    Taking part in clinical research requires careful thought and commitment. We encourage you to review all the documents provided, which give important information about the different components of SRP-9001 gene therapy; the reasons why SRP-9001 is being studied; and the risks and benefits of study-related procedures. Also, make sure that you understand the advantages and disadvantages of gene therapy. For example, SRP-9001 is given as a one-time administration compared with standard drugs for Duchenne which are usually taken regularly. After you have read these documents, we strongly recommend that you discuss your questions and concerns with the study doctor, who will also be happy to discuss results from previous studies of SRP-9001 to help you make your decision. You may also wish to discuss the study with your regular doctor.

  • Why should we take part?

    A clinical research study is an important step toward finding potential future treatments for individuals with Duchenne. If you and your child decide to take part, you’ll be helping researchers understand this disease better and playing an important role in ongoing efforts to improve healthcare.