Phase 2 study of ATL1102 (CD49d Antisense Oligonucleotide) in people with Duchenne

A Phase 2 open label study to determine the safety, efficacy and pharmacokinetic profile of weekly dosing of ATL1102 in patients with non-ambulatory Duchenne Muscular Dystrophy.

Questions and Answers

What stage is this research?

This is a Phase 2 trial. The study is fully enrolled and ongoing.

What is the goal or purpose of this study?

The study is being conducted to assess the safety, tolerability, pharmacokinetics and efficacy of ATL1102 when administered as a 25 mg, once weekly by an injection under the skin (subcutaneous injection) for 24 weeks in non-ambulatory participants with DMD.

Who is funding this study?

This study is funded by Antisense Therapeutics Limited

Who is eligible to participate in this study?

To participate in this study you must:

  • Be male aged 10 to 18 years inclusive, at the time of providing informed consent.
  • Have documented diagnosis with Duchenne Muscular Dystrophy and have been non-ambulatory for at least 3 months
  • If currently receiving glucocorticoid therapy, have been on a stable dose of glucocorticoid therapy for at least 3 months prior to Day 1
  • Be able, and have a parent/guardian who are, willing and able to comply with scheduled visits, study drug administration plan, and study procedures

Additional criteria apply and will be reviewed with participants during the screening process. The study doctor determines whether a participant meets the study inclusion criteria and therefore, whether the participant is eligible to take part in the study.

What do I have to do if I decide to participate in this study?

It is planned 9 participants with DMD will be enrolled into the study and will all receive ATL1102. The study does not have a placebo arm.

Written informed consent will be obtained from the parent/guardian of the participant before any tests or investigations outlined in the study protocol are carried out.

The study doctor will review study requirements with all participants during the screening process and determine the eligibility of the participant to take part in the study. If eligible, the participant will receive weekly injections of ATL1102 over 24 weeks. Participants will be administered ATL1102 either at the study site (on 10 occasions) or in the home environment (on 14 occasions). A study nurse is appointed to perform the ATL1102 administration at the participant’s home (if the participant and their parent/guardian agree). Participants return to the study site about every 2 weeks for assessments. Safety and tolerability are assessed throughout the study via collection of side effect information, injection site reactions, laboratory assessments (blood and urine samples), electrocardiograms (ECG), vital signs and physical examinations. Efficacy assessments including assessment of lymphocyte modulation and upper limb functional measurements are also performed during the study visits.

Blood samples will be taken periodically throughout the study to assess levels of ATL1102 in the bloodstream (pharmacokinetics).

How long will this study last, and will I have access to the drug/treatment once the study has ended?

The anticipated length of the study is about 9 months (36 weeks), including:

  • up to 4 weeks screening period,
  • 24-week treatment period
  • 8-week follow up period

Long term access to ATL1102 after study closure is not available at this stage of the study.

Where does this study take place?

This study takes place in Australia.

How many visits to the study site are necessary?

The study involves 16 study site visits over a period of up to 36 weeks.

Can any visits be done locally?

No, visits are to be done at the study site.

Is there any funding to help pay for travel?

Yes, study visit related travel expenses will be reimbursed.

Will I get paid for participating in this study?

No, there is no compensation for participating in the study, but reasonable travel, parking and meal expenses incurred due to study requirements will be reimbursed. Additional information regarding reimbursement can be provided by the study doctor.

Why should I consider participating in this study?

While no personal benefit can ever be guaranteed by participation in a clinical trial, there are other benefits, including allowing you to play an active role in your own health care (or that of your child), gaining access to new research treatments before they are widely available and having access to medical specialists that are normally not available to you or your child, and helping others by contributing to the better understanding of DMD.

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