DELIVER FAQ

1. Who is sponsoring the DELIVER study?

This research study, also referred to as a clinical trial, is being sponsored by Dyne Therapeutics, a clinical stage company based in Waltham, Massachusetts.

2. What is the intent of the DELIVER study?

This research study, also referred to as a clinical trial, is being sponsored by Dyne Therapeutics, to learn more about an investigational therapeutic called DYNE-251. DYNE-251 is designed to target muscle tissue and promote exon skipping, which allows muscle cells to create a shortened, functional dystrophin protein. “Investigational” means DYNE-251 is currently being tested to understand if it can help people with Duchenne. It has not yet been approved for use in treating Duchenne by any governmental regulatory agency.

3. What is purpose of this study?

The DELIVER study is testing DYNE-251 in boys and adolescents with Duchenne who have a mutation that is amenable to exon 51 skipping. This study has been designed to help answer the following questions:

Is DYNE-251 safe?
What is the best dose of DYNE-251 for participants?
Can DYNE-251 increase muscle dystrophin and improve muscle function?

4. Who is eligible to participate in the DELIVER study?

A participant in the DELIVER study must be:

4 to 16 years old
Male with Duchenne and a mutation amenable to exon 51 skipping
Able to raise his arms over his head
Currently receiving a steroid medicine

There are additional criteria for getting into the study, which will be explained during the informed consent and assent process. Information on the DELIVER study is also available on the DELIVER study page at clinicaltrials.gov.

5. How long is this study?

Participation in the study could last approximately 3 years. However, being in this study is completely voluntary. Participants can leave at any time. Leaving this study will have no impact on care received outside the study.

6. What treatment will be given?

An eligible study participant who chooses to take part in this study, with consent from his parent or caregiver, will receive treatment with:

Either DYNE-251 or placebo for 24 weeks followed by
DYNE-251 for 120 weeks

A computer will assign the participant to receive either DYNE-251 or placebo in a 2:1 or 3:1 ratio, via a random assignment process, for the first 24 weeks.

7. Are there costs for participating?

Study treatment and all study tests and procedures are provided at no cost to study participants and their parents or caregivers. Payment is not allowed for participation in this study, but participants will be reimbursed for approved study-related travel expenses. Concierge services will be available to participants who choose to have study-related travel arranged for them at no cost.

8. What will happen during the study?

screening period

Study participants and their parents or caregivers will be given an Informed Consent or Assent Form that explains the DELIVER study. The study doctor will review this with them and answer any questions they may have. If a study participant agrees to join the study, and his parent or caregiver consents, the study doctor will check to see if he meets all criteria for getting into the study. This will include some medical tests and procedures.

placebo-controlled period, open-label period, and long-term study period

A study participant who meets all criteria for getting into the study will:

Receive DYNE-251 OR placebo by intravenous (IV) infusion every 4 weeks for the first 24 weeks
Receive DYNE-251 by IV infusion every 4 weeks throughout the Open-Label and Long-Term Study Periods
Have his Duchenne and overall health assessed regularly throughout the study

Taking part in this study is completely voluntary. Contacting a study doctor does not commit parents or caregivers to anything.

For more information:

If parents or caregivers are interested in the DELIVER study and want to know more, they can:

Talk to their Duchenne treating physician about this study
Contact a doctor working on this study; for a list of participating physicians and sites, visit the DELIVER study page at clinicaltrials.gov.

As of May 2023

Frequently Asked Questions