If eligible, your child will either receive study treatment (GTX-102) for 2 years or be in a Sham Control group for the first year, then receive GTX-102 in the second year. This is designed to show that GTX-102 is safe and works better than routine medical care. All children will have the opportunity to receive GTX-102 during the study. GTX-102 is given as a low starting dose every 4 weeks to start. Over time, the dose increases, and the dosing interval increases to 8 weeks and then 12 weeks.
1. Double-blind Period
(11 months)
GTX-102 or standard AS care‡
Transition
(2 months)
2. Open-label Period
(11 months)
GTX-102
‡ Double-blind study treatment is assigned at random, with an equal chance of each option. The Double-blind Period will include a Sham Control group.
Sham Control group
Sham Control groups are included in clinical studies so researchers can compare the effects of an investigational treatment to the absence of treatment. Participants in the Sham Control group will go through all the same procedures as the GTX-102 group, except they will not receive GTX-102 in the Double-blind Period. This keeps you and the study team from knowing if a participant is receiving active treatment or not. This helps researchers understand more about the investigational treatment.
Study visits
You and your child will have monthly visits to monitor your child’s health. About half will be in-clinic visits. Where allowed, support is provided for travel costs.
In-clinic visits
Assessments, testing, and study treatment (GTX-102) or Sham Control procedure (study procedure with no drug administration)
Phone/virtual visits
Discussion about changes in your child’s health, other medications/therapies