Phase 3 study of RG6206 Anti-Myostatin Adnectin in people with Duchenne

Questions and Answers

What stage is this research?

RG6206 is an investigational molecule in a Phase 3 clinical study (NCT03039686) in ambulatory boys with Duchenne muscular dystrophy that is currently actively recruiting.

What is the purpose of this research?

The main goal of the Phase 3 study (NCT03039686) is to determine whether this investigational molecule RG6206 may be a safe and effective treatment for ambulatory boys with Duchenne.

What is RG6206?

RG6206 is an investigational molecule that is designed to bind to a protein called myostatin and limit its function.

Myostatin is a naturally occurring protein that is made mostly in skeletal muscle cells. These are muscles like the ones found in the arms and legs. Everyone has some myostatin and its natural function usually is to stop muscles growing too much.

Limiting myostatin has been shown in some studies in people to increase muscle size.

Where is this research being carried out?

This is a global study. Sites are already open in the United States and Japan. Additional sites are open or planned to be opened in Canada, Argentina, UK, Belgium, France, Spain, Sweden, Netherlands, Germany, Italy, and Australia in 2018.

In the United States there are multiple study sites open and/or planned in the following states: CA, FL, GA, IL, KS, MD, MO, NY, OH, PA, Washington DC, NV, AZ, and Canada.

Who would be eligible to participate in this clinical study?

Your son may be eligible to participate if he has Duchenne confirmed by medical history with genetic testing and is:

  • 6 to 11 years of age (inclusive) and weight at least 15kg (33 lbs)
  • Able to walk without assistance and climb stairs on his own, 4 stairs in 8 seconds or less
  • Receiving corticosteroids

Note that all Duchenne mutations are eligible for this study and full eligibility requirements will be assessed at a participating study site.

How many boys will be enrolled in the study?

Approximately 159 boys with Duchenne will be enrolled in this study.

How long will the study be?

The study has two parts:

PART 1: consists of an initial 48 week randomized placebo-controlled phase during which all boys will receive either RG6206 or placebo (2:1 ratio, meaning twice as many participants will receive RG6206 versus placebo)

PART 2: consists of a 48-week open-label phase during which all boys will receive RG6206

How is the study medication given?

RG6206 or placebo is administered once weekly by an injection under the skin (subcutaneous injection) in to the tummy (abdomen). The three first doses must be done at the study site, while the remaining administrations may be done at home.

Will I have access to the study drug once the study has ended?

After completion of the 48 week open-label phase, depending on the results of the study, your child may be given the option to enter a separate extension study to continue to receive RG6206.

Is there any funding to help pay for travel?

Eligible participants may receive, at no cost, transportation services with a travel concierge service to cover the costs of study-required travel, according to local guideline.

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