NCT02412943

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2015

Shorter than P25 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 1, 2015

Completed
Same day until next milestone

Study Start

First participant enrolled

April 1, 2015

Completed
8 days until next milestone

First Posted

Study publicly available on registry

April 9, 2015

Completed
22 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2015

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2015

Completed
Last Updated

May 28, 2015

Status Verified

May 1, 2015

Enrollment Period

1 month

First QC Date

April 1, 2015

Last Update Submit

May 27, 2015

Conditions

Short Stature, Idiopathic

Outcome Measures

Primary Outcomes (1)

  • Decrease in IGF-1

    Decrease in either total IGF-1 or IGFBP-3 of 20% or greater as a clinically meaningful decrease in levels

    5 minutes

Secondary Outcomes (1)

  • Length of decrease in IGF-1

    up to 30 days

Study Arms (1)

PAPP-A2 Enzyme Replacement

EXPERIMENTAL

A 20 cc/kg transfusion of Fresh Frozen Plasma will be given over 3 hours on day 0.

Other: Fresh Frozen Plasma

Interventions

Fresh Frozen PlasmaOTHER

Liquid portion of human blood that was centrifuged, separated, and frozen solid within 6 hours of collection

PAPP-A2 Enzyme Replacement

Eligibility Criteria

Age18 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Homozygous for a defect in PAPPA2.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Dwarfism

Condition Hierarchy (Ancestors)

Bone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesEndocrine System Diseases

Study Officials

  • Andrew Dauber, MD

    Children's Hospital Medical Center, Cincinnati

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
BASIC SCIENCE
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 1, 2015

First Posted

April 9, 2015

Study Start

April 1, 2015

Primary Completion

May 1, 2015

Study Completion

May 1, 2015

Last Updated

May 28, 2015

Record last verified: 2015-05

Locations

US(1)