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: 0
• Participant gender: All

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: Accepts Healthy Volunteers

Details

• Lead Sponsor: Children's Hospital Medical Center, Cincinnati
• Treatments: Mecasermin

Criteria

PAPP-A2 deficient

Inclusion Criteria:

• Defect in PAPP-A2 (heterozygous or homozygous mutation)

Exclusion Criteria:

• None

Healthy Volunteers

Inclusion Criteria:

• Between the ages of 18 and 30

• In general good health

Exclusion Criteria:

• Any medications (with the exception of contraceptives)

• Pregnancy