The first, an open-label add-on trial, was conducted in 20 subjects aged 13 35 years, with one dropping out as a result of adverse effects.
A previous add-on trial with the antioxidant N-acetylcysteine (NAC) led to negative symptom reductions in chronic patients.
Ganaxolone was safe and effective in treating this group of refractory infantile spasms patients in an open-label, add-on trial.
Wynn, D. et al. Daclizumab in active relapsing multiple sclerosis (CHOICE study): a phase 2, randomised, double-blind, placebo-controlled, add-on trial with interferon beta.
This is a multicenter, open-label, add-on trial, investigating the safety and efficacy of ganaxolone (GNX) in a population of children with refractory infantile spasms, or with continuing seizures after a prior history of infantile spasms.
In this article, we examine both the risk benefit profile and the potential for exploitation with an add-on trial design in the context of the locally available standard of care in South Africa.
Diazgranados N, Ibrahim L, Brutsche NE, Newberg A, Kronstein P, Khalife S et al. A randomized add-on trial of an N-methyl-D-aspartate antagonist in treatment-resistant bipolar depression.
Zarate CA Jr., Mathews D, Ibrahim L, Chaves JF, Marquardt C, Ukoh I et al. Replication of ketamine's antidepressant efficacy in bipolar depression: a randomized controlled add-on trial.
Zarate CA Jr, Brutsche NE, Ibrahim L, Jranco-Chaves J, Diazgranados N, Cravchik A et al. Replication of ketamine's antidepressant efficacy in bipolar depression: a randomized controlled add-on trial.
There are compelling reasons to conduct such a trial in low-middle income countries (LMICs) with a high burden of disease, such as South Africa, and to adopt an add-on trial design in which participants are randomized to receive the GBS vaccine or placebo in addition to the locally available standard of care.
The first and only trial for lithium in FXS was a 15 subject, open-label, add-on trial.
