Table 1 Patient demographics and medical history
Distribution (N = 7 total caregiver participants) | |
|---|---|
Patient age at the start of trial | |
Mean (SD) | 9 (± 3.51) |
Range | 5–15 Years |
Patient age at VoC interviews | |
Mean (SD) | 12.1 (± 3.38) |
Range | 8–18 Years |
Patient Sex | |
Male | 7 (100.0%) |
Caregiver relationship to patient | |
Parent | 7 (100.0%) |
Patient medical history | |
Phenotype: neuronopathic vs. non-neuronopathic* | Neuronopathic (n = 5, 71.4%), Non-neuronopathic (n = 2, 28.6%) |
Date of enrollment in phase 3 study | 22 August 2018–22 November 2018 |
Pabinafusp alfa treatment start date | 2 October 2018–27 December 2018 |