CALYX recruited 41 adult male cALD patients with Gd-positive lesions, and where HSCT (haematopoietic stem cell transplantation) is not recommended or refused by the patient. The key exclusion criteria were a Loes Score greater than 12, and patients that previously received HSCT or gene therapy. The trial has an adaptive duration with an initial efficacy read at 18 months and if needed subsequent efficacy assessments at 27 and 36 months respectively with the option to complete the study at any of the three time points once statistical significance is reached. After completing the randomized phase patients will continue into an open-label extension (OLE). The 18 months read out will take place in 1Q 2028.
This pivotal study was designed with input from FDA, as well as US and EU clinical experts in X-ALD and patient advocacy groups.

For further information visit:

https://beta.clinicaltrials.gov/study/NCT05819866