Exercise Versus DDAVP in Patients With Mild Hemophilia A

NCTNCT03379974Other idIRB17-00290

Open at ClinicalTrials.govAsk about this trial

Overview

Status: Completed
Phase: Phase 4
Enrollment: 32 participants (actual)
Sponsor: Nationwide Children's Hospital · Other
Design: Randomized · Open label (no masking)
Countries: 1 country
United States
Start: 31 Jul 2018
Primary completion: 30 Aug 2019
Completion: 5 Dec 2019
First posted: 20 Dec 2017
Last update posted: 25 Feb 2020
Conditions: Hemophilia A

Timeline

1 trial · 1 completed

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

201820192020

Ph 4
Exercise Versus DDAVP in Patients With Mild Hemophilia A
NCT03379974
31 Jul 2018 to 5 Dec 2019

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 Nationwide Children's), 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

DDAVP Inhalant ProductDrug
Exercise InterventionBehavioral

Arms4

Experimental
ARM A: DDAVP followed by exercise
1. Intervention #1: DDAVP Inhalant Product intervention 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). 2. Intervention #2: Exercise intervention
Active comparator
ARM B: DDAVP alone
1. Intervention #1: DDAVP Inhalant Product intervention 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). 2. Intervention #2: no further intervention (rest)
Experimental
ARM C: Exercise intervention
1. Intervention #1: Exercise intervention 2. Intervention #2: no further intervention (rest)
Experimental
ARM D: Exercise followed by DDAVP
1. Intervention #1: Exercise intervention 2. Intervention #2: DDAVP Inhalant Product intervention 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
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 FVIII:C (measured as absolute and fold increase) associated with a standardized moderate intensity aerobic exercise regimen (3-minutes of exercise at 85% of the predicted maximum heart rate) to intranasal 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
Time frame · Baseline, 30 min post intervention #1, 30 min post intervention #2 and 90 minute post intervention
Registry description
To determine the absolute and fold increase in FVIII:C associated with the sequential administration of exercise followed by intranasal 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
Time frame · Baseline, 30 min post intervention #1, 30 min post intervention #2 and 90 minute post intervention
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 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.

Completed trials do not always post results.

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: * 1\) Patients of ≥13 years of age and ≤21 years of age with MHA (FVIII:C level of ≥6% to ≤50%). Exclusion Criteria: 1. A currently circulating or a history of inhibitor (0.5 BU on two or more occasions). Inhibitor development is rare in MHA. 2. History of FVIII infusion (both standard-acting and extended half-life products) or DDAVP use in preceding 1 week. Patients will be instructed to hold factor use or DDAVP for 1-week prior to participation in study, except for management of acute bleeds, in which case they will be instructed to inform the PI via telephone or e-mail. 3. Patients with severe arthropathy (as determined by the principal investigator) interfering with ability to exercise. Severe arthropathy is rare in MHA. 4. Patients on beta-blockers, anti-platelet agents or regular non-steroidal anti-inflammatory medications (e.g. Celebrex). 5. Patients who are active smokers (cigarettes, marijuana). 6. 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. 7. Co-existence of a congenital bleeding disorder other than MHA (e.g. VWD). 8. Patients with an active infectious or inflammatory condition. This includes previously identified HIV, active hepatitis B or C as reflected in elevated AST, ALT, RNA positivity for hepatitis B or C. HIV, hepatitis B and C are very rare in the age group (13-21 years) we hope to accrue in the proposed study. 9. 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).

PepBank is a research reference. Talk to a doctor before you use any peptide.