SPITFIRE: Phase 2/3 study of RG6206 (RO7239361) in people with Duchenne

Roche/Genentech Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy

Questions and Answers

What stage is this research?

RG6206 (or RO7239361) is an investigational molecule in a SPITFIRE Phase 2/3 clinical study (NCT03039686) in ambulatory boys with Duchenne muscular dystrophy.

What is the purpose of this research?

The main goal of the SPITFIRE Phase 2/3 study (NCT03039686) is to determine whether this investigational molecule RG6206 is effective, safe and tolerated 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 from growing too much.

Has RG6206 been studied before?

Yes. An initial clinical trial (Phase 1) with RG6206 was completed in healthy adults. Further information can be found here: NCT03100630 on ClinicalTrials.Gov.

RG6206 is currently being studied in a Phase 1/2 study to investigate its safety and tolerability in boys with Duchenne. The trial is ongoing, but is no longer open to new patients. You can find more information here: NCT02515669 on ClinicalTrials.Gov.

Where is this research being carried out?

The Phase 2/3 clinical study is global. Sites are located in the United States, Canada, Japan, Argentina, UK, Belgium, France, Spain, Sweden, Netherlands, Germany, Italy, and Australia.

In the United States there are multiple study sites. Further information on sites and their status can be found here: NCT03039686 on ClinicalTrials.Gov.

Who are the participants in this clinical study?

The participants in this trial are boys with Duchenne confirmed by medical history with genetic testing and are:

  • 6 to 11 years of age (inclusive) and weighs 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
  • Has a North Star Ambulatory Assessment (NSAA) total score ≥ 15 points at the Screening Visit

Participants in this study may have all Duchenne mutations. Full screening assessments for participation in the study will be conducted at the study sites.

How many boys will participate in the study?

Approximately 159 boys with Duchenne will participate in this study.

How long will the study be?

The study has two sequential 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 192-week open-label phase during which all boys will receive RG6206

How is RG6206 given in the study?

RG6206 or placebo is administered once weekly by an injection under the skin (subcutaneous injection) in to the tummy (abdomen), thigh or back of upper arm after initial training at the site. The three first doses must be done at the study site, while the remaining administrations are done at home.

Where can I learn more about this research?

  • Please contact your treating physician to discuss the clinical study
  • Visit ClinicalTrials.gov (search for NCT03039686)
  • Call the Roche/Genentech Clinical Trial Information Support Line
    Phone: 1-888-662-6728
    Hours: Monday-Friday, 5am-5pm Pacific Time

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