The ENTR-601-44-201 study (also called ELEVATE-44) is a global, two-part, randomized, double-blind placebo-controlled*, Phase 1/2b study evaluating the safety, tolerability and effectiveness of ENTR-601-44 in people living with Duchenne who are amenable to exon 44 skipping.
*A placebo looks like a study drug but does not have any medicine in it. Researchers use placebos to help make sure any changes in participants’ health are actually caused by the study drug.
ELEVATE-44 is a two-part study. Currently, the study is enrolling participants for Part A.
Part A of the study is designed to:
Evaluate the safety of ENTR-601-44 (also called the study drug)
Study its effects on the body (called pharmacodynamics), and its circulation within the body (called pharmacokinetics)
Evaluate how effective it is, by measuring how much dystrophin is produced after treatment
Determine the optimal dose for further study in Part B. Additional details on Part B will become available as we approach its expected start.
Part B of the study is designed to:
.png)
Evaluate how effective ENTR-601-44 is by measuring dystrophin production
Measure functional changes in movement and quality of life
Further assess safety and tolerability
An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-44 over a longer period of time. All participants in the open label extension will receive ENTR-601-44.
ENTR-601-44 is a study drug, which has not been approved for use outside of clinical studies in any country. To date it has been evaluated in both animal models and healthy volunteers. To learn more about these studies, visit Entrada's website.
Participants in Part A need to be*:
4-20 years old
Ambulatory
Assigned male at birth
Diagnosed with Duchenne muscular dystrophy amenable to exon 44 skipping (if you're unsure, the study staff will help you to determine this.)
*Additional inclusion and exclusion criteria apply. These will be discussed and assessed during the study screening period.
The study is currently approved in the United Kingdom, Belgium and Spain with additional sites being considered in Italy.
Reasonable study-related expenses, such as travel, accommodation and meals will be covered for study participants in accordance with local regulations. This includes booking assistance for travel and lodging. The study doctor can provide more information on additional covered expenses.
There will be approximately 24 participants in Part A. Participation in Part A of the study will take approximately six months (25 weeks). During this time, study-related care will be provided by a study doctor with deep experience in Duchenne.
The study is evaluating three separate dose levels of ENTR-601-44 (starting at a dose level of 6mg/kg with a planned dose escalation of up to 18mg/kg) compared to placebo. A placebo looks like a study drug but does not have any medicine in it. Researchers use placebos to help make sure any changes in participants' health are actually caused by the study drug.
Each participant will be randomized to receive one of the dose levels of ENTR-601-44 or the placebo. There is a 75% chance that a participant is assigned to receive the study drug and a 25% chance they are assigned to receive placebo during Part A of the study.
The study doctor will administer the study drug or the placebo via intravenous (IV) infusion approximately once every six weeks. Additional visits will occur throughout the study to learn more about the safety and potential impact of ENTR-601-44 on the body's functions. These visits may be done at the study site or at the participant's home by a qualified home healthcare professional, in accordance with local regulations.
Participants will have a muscle biopsy at the beginning of their participation and after their last dose. Muscle biopsies are important because they allow researchers to compare whether there have been changes in the muscle as a result of the study drug.
Participants in Part A may be eligible to enter an open label extension study (OLE). An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-44 over a longer period of time. All participants in the open label extension will receive ENTR-601-44. Additional details will become available as we approach the expected start of the OLE.
Clinical studies come with risks. It is important to make an informed decision before deciding to join a study. Making an informed decision means asking questions until those considering participation know what to expect.
If you or a loved one are interested in participating in a clinical study, consult your doctor to determine eligibility and collect the information needed to make an informed decision.
Entrada believes that study participation should not be a financial burden. For the inconvenience related to participation, participants may be compensated for the study visits completed, according to their country’s local regulations. In addition, reasonable study-related expenses, such as travel, accommodation and meals will be covered for study participants in accordance with local regulations. This includes booking assistance for travel and lodging. The study doctor can provide more information on additional covered expenses.
No additional payments or compensation will be provided for participating in the study.
No, participants in the ELEVATE-44 study will be randomly assigned to receive either one of the three different doses of ENTR-601-44 (starting at a dose level of 6mg/kg with a planned dose escalation of up to 18mg/kg) or the placebo. There is a 75% chance that a participant receives the study drug and a 25% chance they receive placebo during Part A of the study.
Participants in Part A may be eligible to enter an open label extension study (OLE). An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-44 over a longer period of time. All participants in the open label extension will receive ENTR-601-44. Additional details will become available as we approach the expected start of the OLE.
Participation in Part A of the study will take approximately six months (25 weeks). During this time, study-related care will be provided by a study doctor with deep experience in Duchenne.
The study doctor will administer the study drug or the placebo via intravenous (IV) infusion approximately once every six weeks. Additional visits will occur throughout the study to learn more about the safety and potential impact of ENTR-601-44 on the body's functions. These visits may be done at the study site or at the participant's home by a qualified home healthcare professional, in accordance with local regulations.
Participants in Part A may be eligible to enter an open label extension study (OLE). An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-44 over a longer period of time. All participants in the open label extension will receive ENTR-601-44. Additional details will become available as we approach the expected start of the OLE.
No, participants in Part A will not be eligible to participate in Part B. However, Entrada is committed to having an open label extension study (OLE) for which participants from both Part A and Part B may be eligible. An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-44 over a longer period of time. All participants in the open label extension will receive ENTR-601-44. Additional details will become available as we approach the expected start of the OLE.
Yes. Being in the study is completely voluntary and participants can stop at any time.
Yes, it is important for participants to maintain regular visits with their primary doctor and care team to ensure that existing care plans remain on track.
For the most part, yes. However, this will depend on the type of medicine the participant is currently taking. Some medicines are not allowed during the study for safety reasons. The study doctor will inform potential participants if any changes to current medicines are necessary.
Entrada adapted the below information from materials developed by Center for Information and Study on Clinical Research Participation, an independent non-profit organization dedicated to engaging the public and patients as partners in the clinical research process.
Clinical studies are the best way to evaluate a study drug and learn if it works and how safe it is. If you have ever taken medicine or gotten a vaccine, then you have benefited from clinical studies.
Studies try to answer specific health questions, like if the study drug has any effects on the disease, if it could work better than an existing treatment and if it’s safe for people to use. A “study drug” means that it has not been approved by a government agency for use outside of clinical studies. If clinical studies show that the study drug works and is safe, then it can be submitted for approval to government agencies. If approved, the study drug can then become available to the people who need it.
People participate in clinical studies for a variety of reasons. Some participate to help others and to increase the understanding of a condition or the science behind a study drug. Those with a disease might participate to receive access to a study drug before it is approved and accessible to the general public.
Clinical studies come with risks. Comparing the potential risks and benefits is an important part of deciding whether to participate. If you or a loved one are interested in participating in a clinical study, consult your doctor to determine eligibility and collect the information needed to make an informed decision.
A placebo looks like a study drug but does not have any medicine in it. Researchers use placebos to help make sure any changes in participants’ health are actually caused by the study drug. In a placebo-controlled study, participants are often randomly assigned (also known as randomization) to receive either the study drug or the placebo. Both researchers and participants may not know who received the placebo until after the study.
Information on active studies can be found by searching the clinical study databases below.
Patient advocacy organizations, such as the World Duchenne Organization, also maintain a list of ongoing Duchenne studies.
Each study needs participants who meet certain requirements, like having a certain disease or medical condition. These requirements are called inclusion criteria. Other inclusion criteria could be characteristics like age, sex and current medicine(s) that the participant may be taking.
Clinical studies have specific requirements to ensure safety. These requirements might exclude participation by people whose conditions could worsen with the study drug. This helps protect participants from potential harm. These requirements are called exclusion criteria.
Inclusion and exclusion criteria are in place to make sure the researchers get the most accurate results and make studies as safe as possible for participants.
It's best to ask your/your child’s doctor that you see for Duchenne care or speak with representatives from a Duchenne patient advocacy group.
.png)
Personal data may be collected and processed through inquiries. To learn more about how your personal data is processed, please see Entrada's Privacy Policy located at Entrada Therapeutics | Privacy Policy
ENTR-601-44 is a study drug, which has not been approved for use outside of clinical studies in any country.
Stock photos posed by models.