DDAVP vs. Exercise in Patients With Mild Hemophilia A

What Was Tested

In this phase 4 trial, researchers set out to test Desmopressin.

Although both exercise and DDAVP (desmopressin) can raise Factor 8/Von Willebrand Factor (FVIII/VWF levels), it is not clear whether the pathophysiological mechanism is the same.

Consequently it is not known if DDAVP and exercise would have additive effects in raising FVIII:C and VWF levels or if one would one negate the effect of the other.

It had four arms (groups): Arm A: DDAVP followed by exercise (experimental), Arm B: DDAVP alone (active comparator), Arm C: Exercise alone (experimental) and ARM D: Exercise followed by DDAVP (experimental).

Intervention #1: Exercise Intervention #2: no further intervention (rest).

The registry classes the interventions as behavioral and drug.

More on the Plan

FVIII levels temporarily rise with stress, exercise and with DDAVP (1-desamino-8-D-arginine vasopressin, desmopressin).

Although both exercise and DDAVP can raise FVIII/VWF levels, it is not clear whether the pathophysiological mechanism in which they do this is the same.

Consequently it is not known if DDAVP and exercise would augment each other's effects in raising FVIII:C and VWF levels or if one would one negate the effect of the other.

Herein, the researchers propose a prospective, interventional study of exercise vs IN DDAVP in 40-50 post adolescent (13-21 yr) males with MHA to compare their impact on hemostasis and also to investigate the impact of sequentially administering these interventions on hemostatic indices.

Who Took Part

The registry lists 30 planned participants were the target.

It was open to men only aged from 13 years up to 21 years.

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

The full record lists 2 inclusion and 10 exclusion criteria; among the exclusions: co-existence of a congenital bleeding disorder other than mha (e.g. vwd); prior history of coronary artery disease or pulmonary disease, severe arthropathy interfering with ability to exercise; patients on beta-blockers, anti-platelet agents or regular non-steroidal anti-inflammatory medications (e.g. celebrex).

How the Study Was Run

The set-up was randomized, crossover and open-label.

The participants were allocated by chance, so the Desmopressin group and the comparison group should start out alike.

Each person switched between Desmopressin and the comparison at some point, usually with a gap in between.

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

It started in 2017-07-04 with completion listed for 2018-08.

It ran at 2 sites in Canada (2).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Factor 8 level after exercise (time frame: Baseline, 30 min post intervention #1, 30 min post intervention#2 and 90 minute post intervention#2).

It also tracked 2 secondary outcomes, for example Factor 8 level after sequential administration of exercise followed by IN DDAVP (or vice versa) and Associations between baseline physical activity scores and Factor 8 levels after exercise.

What This Means in Plain Language

Put simply, this is a phase 4, randomized trial of Desmopressin in 30 people.

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

Its status is unknown; no results are posted to the registry yet, so nothing here describes outcomes.

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