DDAVP vs. Exercise in Patients With Mild Hemophilia A

NCTNCT03136003Other id1000054948

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Overview

Status: Status unknown
Phase: Phase 4
Enrollment: 30 participants (estimated)
Sponsor: The Hospital for Sick Children · Other
Design: Randomized · Open label (no masking)
Countries: 1 country
Canada
Start: 4 Jul 2017
Primary completion: Aug 2018 (estimated)
Completion: Aug 2018 (estimated)
First posted: 2 May 2017
Last update posted: 4 Dec 2017
Conditions: Mild Haemophilia A Without Inhibitor

Timeline

1 trial

Completed
Ongoing
Not yet recruiting
Stopped
Estimated dates (est.)
Results posted

201720182019

Ph 4
DDAVP vs. Exercise in Patients With Mild Hemophilia A
NCT03136003
4 Jul 2017 to est. Aug 2018

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

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. The aim of this 2 center (Sickkids and Columbus, Ohio), prospective, cross-over design study is to compare the impact of exercise vs. DDAVP on hemostasis in patients with MHA and also to investigate the impact of sequentially administering these interventions on their hemostatic indices.

Interventions

DDAVPDrug
ExerciseBehavioral

Arms4

Experimental
Arm A: DDAVP followed by exercise
Intervention #1: DDAVP. The participant will take either 1 or 2 nasal sprays of IN DDAVP. For patients weighing \<50 kg: 150 ug (i.e. 1 spray into one nostril) and patients weighing ≥50 kg: 300 ug (i.e. 2 sprays - one into each nostril). Intervention #2: Exercise
Active comparator
Arm B: DDAVP alone
Intervention #1: DDAVP. The participant will take either 1 or 2 nasal sprays of IN DDAVP. For patients weighing \<50 kg: 150 ug (i.e. 1 spray into one nostril) and patients weighing ≥50 kg: 300 ug (i.e. 2 sprays - one into each nostril). Intervention #2: no further intervention (rest)
Experimental
Arm C: Exercise alone
Intervention #1: Exercise Intervention #2: no further intervention (rest)
Experimental
ARM D: Exercise followed by DDAVP
Intervention #1: Exercise Intervention #2: DDAVP. The participant will take either 1 or 2 nasal sprays of IN DDAVP. For patients weighing \<50 kg: 150 ug (i.e. 1 spray into one nostril) and patients weighing ≥50 kg: 300 ug (i.e. 2 sprays - one into each nostril).

Primary outcomes1

Outcomes the sponsor listed as primary.

Factor 8 level after exercise
Time frame · Baseline, 30 min post intervention #1, 30 min post intervention#2 and 90 minute post intervention#2
Registry description
To compare the increase in Factor 8 levels (FVIII:C) (measured as absolute and fold increase) associated with moderate intensity aerobic exercise (approximately 15 minutes of gradually increasing aerobic exercise culminating in 3-minutes of exercise at 85% of the predicted maximum heart rate) vs. IN DDAVP in post-adolescent males with MHA.

Secondary outcomes(2)

Factor 8 level after sequential administration of exercise followed by IN DDAVP (or vice versa)
Time frame · Baseline, 30 min post intervention #1, 30 min post intervention#2 and 90 minute post intervention#2
Registry description
To determine the absolute and fold increase in Factor 8 levels (FVIII:C) associated with the sequential administration of exercise followed by IN DDAVP (or vice versa).
Associations between baseline physical activity scores and Factor 8 levels after exercise
Time frame · Baseline, 30 min post intervention #1, 30 min post intervention#2 and 90 minute post intervention#2
Registry description
To explore the impact of baseline physical activity (measured using the International Physical Activity Questionnaire \[IPAQ\]-short form) on exercise induced increase in Factor 8 levels (FVIII:C).

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

No results are posted for this trial.

A trial that is still running, or that stopped early, may post results later.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Eligibility

Who could join, as posted.

Sex: Male
Age: from 13 Years up to 21 Years
Healthy volunteers: Not accepted

Inclusion and exclusion criteria, as posted

Inclusion Criteria: * Patients ≥13 years of age and ≤21 years of age with Mild Hemophilia A (MHA), with a historical baseline FVIII:C level of ≥5% to ≤40% followed at either the Hospital for Sick Children or St. Michael's Hospital (Toronto). * Patients ≥13 years of age and ≤21 years of age with genetically confirmed Mild Hemophilia A (MHA), with FVIII:C level of ≥5% to ≤50% followed at either the Hospital for Sick Children or St. Michael's Hospital (Toronto). Exclusion Criteria: * A currently circulating or history of a previous inhibitor ( ≥0.5 BU) within the past 5 years. As inhibitor development in MHA is rare, it is not expected that any patient will be excluded for this reason. * Any FVIII infusion or DDAVP use in the preceding week. This is to avoid an residual FVIII still being present in a patient who has taken an extended half-life FVIII. * 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). * Patients with an active infectious or inflammatory condition. This includes HIV, active hepatitis B or C as reflected in elevated AST, ALT, RNA positivity for hepatitis B or C. * Patients who are active (defined as smoking daily) smokers (cigarettes, marijuana). This exclusion is put into place as we do not know if daily smoking will impact on the hemostatic response to either exercise or DDAVP. * Patients with limited exercise tolerance for any reason. * Patients with a history of a recent bleed (in preceding 2 weeks) in any location, or a joint/muscle bleed in the lower limbs in the preceding 4 weeks. * Patients who for medical reasons should not receive DDAVP \[those with renal or CNS disease (e.g. brain tumor)\] or have previously experienced adverse events with DDAVP (e.g. hypotensive event; seizure).

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