What is the current status of the trial?

The SPITFIRE phase 2/3 clinical trial investigating RG6206 in boys with Duchenne is currently active.

What is the purpose of the clinical trial?

The trial will find out if RG6206 is effective, safe and well tolerated in boys with Duchenne.

How many people will take part?

Around 159 boys with Duchenne will be enrolled.

How long will the trial take?

The trial has two parts:
PART 1: 48-week double-blind treatment phase of the trial. Followed by;
PART 2: 48-week open-label phase

After Parts 1 and 2 of the trial are complete, there is also a 24 week follow-up period, during which study staff will continue to monitor participants.

What treatment will be given? Will a placebo be used?

Double Blind versus Open Label periods

During PART 1 of the trial:

All participants will receive corticosteroids.
Some patients will also receive RG6206, and others will receive a harmless substance that looks identical but does not contain the ingredient that makes it ‘active’ – this is called a placebo.

As this is a blinded trial, patients will be randomly allocated to RG6206 or placebo. Neither the patient, nor the investigator will know if they are receiving RG6206 versus placebo.
Out of every three participants:

  • 2 people will receive RG6206
  • 1 will receive placebo

This is called a 2:1 randomisation, it means that twice as many people receive RG6206 as placebo.

During PART 2 of the trial:

All participants will receive corticosteroids and RG6206.

Can my child with Duchenne take part?

Currently, doctors at participating sites are enrolling boys with any Duchenne mutation.
The trial staff will also look at a number of factors including whether your son is:

  • Genetically diagnosed as having Duchenne Muscular Dystrophy
  • 6 to 11 years of age, inclusive
  • Able to walk without assistance and climb stairs on his own (4 stairs in 8 seconds or less)
  • Receiving corticosteroids for 6 months (and has been on a stable dose for at least 3 months) prior to trial participation
  • Not diagnosed with kidney disease or heart failure

Frequently Asked Questions

Please contact your treating physician to discuss the clinical trial.

Your local patient group may also be able to provide you with additional information. You can find more information about patient groups in your country on our Resources page.

You can locate hospitals that are participating in the trial using our Find My Trial tool.

The countries taking part in the trial are also listed on, the
identifying code for this trial is: NCT03039686

Talk to your physician about your interest in taking part, they can help to connect with sites that may consider international participation.

Participating in a trial in another country will be at the discretion of the investigator and is decided on an individual basis.

You can also regularly check back at and Find My Trial for newly opened sites.

When enrolled into the trial, you will be asked to visit the study site every 6 weeks. As much as possible, the study team will work with you to schedule each visit at a time that is convenient for you.

Roche will cover the cost of study medication, visits, assessments and reasonable costs of travel.

Depending on the results from the trial, a separate extension of the study might be available. Once the entire trial is completed and results are available, these will be shared with all participants.

Please talk to your physician or local patient group for more information. A number of patient groups for Duchenne can be found on our Resources
page. is a website that lists all registered clinical trials. If you want to find out more, the ClinicalTrials.Gov identifying code for this trial is: NCT03039686

The trial is also registered on (EudraCT Number: 2016-001654-18)

If you are interested in supporting the Duchenne community there are a number of patient advocacy groups that you can offer your support to. See our Resources page for more information.