IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
Status:
Active, not recruiting
Trial end date:
2022-05-01
Target enrollment:
Participant gender:
Summary
With this study we want to investigate the pharmacokinetic (PK) effect of a single injection
of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy
controls. This will be followed by treatment of PAPP-A2 deficient patients with IGF-1 for a
period of one year to assess growth velocity. Additionally we want to further describe the
phenotypic characteristics of patients with PAPP-A2 deficiency.