Growth Hormone Treatment in Patients With Aggrecan (ACAN) Deficiency
Status:
Active, not recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
This is an open-label, single-arm prospective pilot study to study the effects of a single
dose regimen of daily growth hormone medication (Norditropin) on pre-pubertal children with
Aggrecan deficiency. The growth response will be tracked over a 12 month period. A protocol
extension has been approved to continue subjects on treatment for an additional 2 years.