The SPITFIRE phase 2/3 clinical trial investigating RG6206 in boys with Duchenne is currently active.
The trial will find out if RG6206 is effective, safe and well tolerated in boys with Duchenne.
Around 159 boys with Duchenne will be enrolled.
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.
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:
This is called a 2:1 randomisation, it means that twice as many people receive RG6206 as placebo.
All participants will receive corticosteroids and RG6206.
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: