Exercise Versus DDAVP in Patients With Mild Hemophilia A

What Was Tested

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

Individuals with mild hemophilia A (MHA) bleed infrequently but can in the setting of trauma which often is when participating in sports/exercise.

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 intervention (experimental) and ARM D: Exercise followed by DDAVP (experimental).

1. Intervention #1: Exercise intervention 2. 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-Darginine 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 investigators 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

Enrollment: 32 actual participants took part.

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 1 inclusion and 9 exclusion criteria; among the exclusions: A currently circulating or a history of inhibitor (0.5 BU on two or more occasions). Inhibitor development is rare in MHA; patients with severe arthropathy (as determined by the principal investigator) interfering with ability to exercise. severe arthropathy is rare in mha; 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.

Random assignment meant chance, not the researchers, decided who got Desmopressin and who got the comparison, which limits bias.

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 2018-07-31 with completion listed for 2019-08-30.

It ran at 4 sites in United States (4).

Its stated primary purpose is treatment.

What Was 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 (time frame: Baseline, 30 min post intervention #1, 30 min post intervention #2 and 90 minute post intervention #2).

What This Means in Plain Language

Put simply, this is a phase 4, randomized trial of Desmopressin in 32 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.