CompletedPhase 1/2

IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency

7 participantsStarted 2015-12-21

Plain-language 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.

Who can participate

Age range10 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Height Velocity

Timeframe: Yearly until participant on treatment stops growing, or discontinues treatment (up to 6 years)

Trial details

NCT IDNCT02636270

SponsorChildren's Hospital Medical Center, Cincinnati

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2022-10-14

Results submitted2023-11-09

View on ClinicalTrials.gov More Short Stature trials