Characterization of Laboratory Response to DDAVP in Adult Hemophilia A Carriers

What Was Tested

This registered phase 1 trial tested Desmopressin.

The purpose of this study is to determine how female hemophilia A carriers respond to a medication called DDAVP (Desmopressin).

It had three arms (groups): Hemophilia A carriers with mild mutation (active comparator), Hemophilia A Carriers with severe mutation (active comparator) and Control (active comparator).

The registry classes the interventions as drug.

More on the Plan

DDAVP (Desmopressin) is commonly used in the treatment of persons with bleeding disorders such as hemophilia, von Willebrand disease, or qualitative platelet disorders to help them clot better.

The investigator will compare the response to DDAVP (Desmopressin) in adult hemophilia A carriers to women with a diagnosis of qualitative platelet dysfunction.

Who Took Part

Enrollment: 2 actual participants took part.

It was open to women only aged from 18 years up to 60 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Females 18-60 years of age at time of enrollment. Females 18-60 years of age at time of enrollment. Age-matched by 10 years to carrier enrolled.

The full record lists 8 inclusion and 4 exclusion criteria; among the exclusions: personal history of concomitant bleeding or clotting disorder; cardiac condition that requires the daily use of aspirin or clopidogrel; inability to comply with fluid restriction protocol for 24 hours following desmopressin (ddavp).

How the Study Was Run

Design: non-randomized, parallel-group and open-label.

The Desmopressin group and the comparison group ran side by side rather than switching.

Being open-label, the Desmopressin study did not mask who received what.

It started in 2015-07 with completion listed for 2018-06-15.

It ran at 2 sites in United States (2).

Its stated primary purpose is diagnostic.

What Was Measured

It also tracked 5 secondary outcomes, for example Change in the time-course response of Factor VIII antigen levels.

What This Means in Plain Language

Put simply, this is a phase 1, non-randomized trial of Desmopressin in 2 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Desmopressin compound page, all human trials.