IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
This trial is active, not recruiting.
|Phase||phase 1/phase 2|
|Sponsor||Children's Hospital Medical Center, Cincinnati|
|Start date||December 2015|
|End date||December 2016|
|Trial size||7 participants|
|Trial identifier||NCT02636270, 2015-6218|
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.
|Endpoint classification||efficacy study|
|Intervention model||single group assignment|
Patients deficient in PAPP-A2 with short stature will be treated with Increlex (rhIGF-1)
Improve growth velocity
time frame: One year
Pharmacokinetic/Pharmacodynamic (PK/PD) relationship
time frame: One year
Male or female participants at least 10 years old.
PAPP-A2 deficient Inclusion Criteria: - Defect in PAPP-A2 (heterozygous or homozygous mutation) Exclusion Criteria: 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
|Official title||Treatment With Recombinant Human Insulin-like Growth Factor 1 (rhIGF-1) in Patients With Pappalysin-2 (PAPP-A2) Gene Mutation.|
|Principal investigator||Andrew Dauber, MD|
|Description||The 24-hour pharmacokinetic response of free and total IGF-1 and IGF binding protein-3 (IGFBP-3) to a single dose of rhIGF-1 (120 mcg/kg) in three patients with PAPP-A2 mutation compared to up to four unaffected heterozygous relatives and 2 healthy adult controls. One-year trial of rhIGF-1 at standard dose given to the two youngest males with PAPP-A2 mutation. The primary end point of this trial will be first year height velocity. Secondary outcomes will include change in height SDS, change in height velocity SDS, and change in whole body and lumbar spine bone mineral density. Description of additional phenotypic characteristics of patients with PAPP-A2 mutation by studying glucose and insulin metabolism, body composition, bone geometry and bone density before and after treatment with rhIGF-1.|
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