Pappalysin 2 (PAPP-A2) Enzyme Replacement

Completed · Phase 1

Conditions studied: Short Stature, Idiopathic

In brief

This study evaluates the transfusion of fresh frozen plasma containing the enzyme PAPP-A2 into the a female adult. This female adult has a mutated version of PAPP-A2 that prevents the unbinding of IGF-1 from IGF binding proteins. The investigator's hypothesize that transfusion of plasma with donor PAPP-A2 will lead to the unbinding of IGF-1 from its binding proteins and that they will be able to measure free IGF-1 in the blood of this female adult.

Key facts

Study ID

NCT02412943

Run by

Children's Hospital Medical Center, Cincinnati

People needed

1

Starts

2015-04-01

Expected to finish

2015-05-01

Last updated by the study team

2015-05-28

Who can join

Age: 18 and older. Sex: female. Healthy volunteers: not accepted.

You may qualify if…

• Homozygous for a defect in PAPPA2.
• Exclusion Criteria: None

Where it is running

• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States

Full record on ClinicalTrials.gov

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