Hemophilia ANCT03286153
Comparison of Ideal vs. Actual Weight Base Factor Dosing
A study testing Ideal Body Weight First and Actual Body Weight First in people with Hemophilia A, run by Bloodworks at 4 sites in United States.
Trial summary
This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and… Read more about this study
- Who is it for?
- Ages 12 years and older, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Ideal Body Weight First and Actual Body Weight First
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 4 sitesUnited States
- Study dates
- Jan 2017to Dec 2018
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Hemophilia A
- Able and willing to comply with pharmacokinetic testing schedule
- Either overweight or obese BMI using CDC definitions by age
Who cannot join
- Inhibitor of > 0.6 BU twice in the past, or documented abnormal recovery of less than 66% (of expected) in the past
- Known other bleeding disorder
- Known other prolongation in aPTT (lupus anticoagulant, FXII deficiency)
- Female
Trial locations and contacts
4 sites in 1 country.
United States4 sites, 4 recruiting
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239-3098
Michael Recht, MD, PhD, 503-494-8311, rechtm@ohsu.edu
- Recruiting
Washington Center for Bleeding Disorders at Bloodworks Northwest
Seattle, Washington, 98104
Rebecca Kruse-Jarres, MD, MPH, 206-689-6593, RebeccaKr@BloodWorksNW.org
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Amanda Blair, MD, 206-987-2106, Amanda.Blair@seattlechildrens.org
- Recruiting
Providence Sacred Heart Children's Hospital
Spokane, Washington, 99220-2555
Judy Felgenhauer, MD, 509-474-2777, Judy.Felgenhauer@providence.org
About this study
This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and cessation. This study also examines ways to prevent delivering excessive factor by using a patient's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in protective factor levels.
Read the detailed description
This is a randomized, prospective, multicenter, open-label, crossover study to examine whether or not the current recommended factor dosing strategy, i.e., dosing by actual body weight in overweight and obese patients, may deliver more clotting factor than necessary to cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This study also examines ways to prevent delivering too much factor by using a participant's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in hemostatic factor levels.
The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at Bloodworks Northwest, Oregon Health \& Science University (OHSU), Seattle Children's Hospital (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four sites, up to 20 participants will be enrolled. Randomization will be performed centrally at WCBD.
Participants will provide their own factor. Prior to the first study-related dose, participants will stop taking any FVIII products for either 48 hours if currently using a short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be measured immediately before and at multiple points after two different factor doses. Subjects will be randomized to start their dosage based either on actual body weight or ideal body weight first and then crossover to receive dosage based on the other category.
- Conditions
- Hemophilia A
- Being tested
- Ideal Body Weight First, Actual Body Weight First
- Keywords
- Hemophilia A, FVIII, Ideal body weight, Actual body weight
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ideal Body Weight FirstOther
Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)].
Actual Body Weight FirstOther
Randomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight.
| Study group | Role | What they receive |
|---|---|---|
| Ideal Body Weight First | Experimental | Randomized to receive factor product based on ideal body weight first Receives: Other: Ideal Body Weight First |
| Actual Body Weight First | Experimental | Randomized to receive factor product based on actual body weight first Receives: Other: Actual Body Weight First |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recovery
Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A
Measured: Change from baseline at up to two months
Underdosing
Determine the likelihood of underdosing when using ideal body weight
Measured: Change from baseline at up to two months
Overdosing
Determine the likelihood of overdosing when using actual body weight
Measured: Change from baseline at up to two months
Secondary outcomes (4)
Effect of half-life
Determine the effect on half-life of these dosing strategies
Measured: Change from baseline at up to two months
Effect on hemophilia severity
Determine the effect of pharmacokinetic differences on hemophilia severity
Measured: Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life
Regular half-life vs. extended half-life Regular half-life vs. extended half-life
Determine differences in participants receiving regular half-life versus extended half-life products
Measured: Change from baseline at up to two months
Overweight vs. obese
Determine the differences, if any, between overweight and obese participants
Measured: Change from baseline at up to two months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Sep 2017
- Study startJan 2017
- Primary completionDec 2018 (estimated)
- Study completionDec 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Health services research
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Subjects are randomized to receive factor based either on ideal body weight or actual body weight first followed by receiving factor based on the other category next
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bloodworks
- Sponsor type
- University, hospital or other
- Collaborators
- Seattle Children's Hospital, Oregon Health and Science University, Providence Health & Services
- Responsible party
- Sponsor
- Investigators
- Rebecca Kruse-Jarres, MD, MPH, Principal investigator, Washington Center for Bleeding Disorders at Bloodworks Northwest
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03286153
- Sponsor's protocol ID
- BDC Ideal Body Weight Dosing
- Status last verified
- Sep 2017
- First submitted
- 9 Aug 2017
- Last updated
- 18 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Comparison of Ideal vs. Actual Weight Base Factor Dosing
- ClinicalTrials.gov ID
- NCT03286153
- Acronym / sponsor ID
- BDC Ideal Body Weight Dosing
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, FVIII, Ideal body weight, Actual body weight
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- BloodworksUniversity, hospital or other
- Collaborators
- Seattle Children's Hospital, Oregon Health and Science University, Providence Health & Services
- Study start
- Jan 2017 (actual)
- Primary completion
- Dec 2018 (estimated)
- Study completion
- Dec 2018 (estimated)
- First posted
- 18 Sep 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, prospective, multicenter study to examine whether or not the current recommended factor dosing strategy - i.e., dosing by actual body weight - in overweight and obese patients with Hemophilia A may deliver excessive clotting factor to achieve the desired result of bleeding prevention and cessation. This study also examines ways to prevent delivering excessive factor by using a patient's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in protective factor levels.
Detailed description
This is a randomized, prospective, multicenter, open-label, crossover study to examine whether or not the current recommended factor dosing strategy, i.e., dosing by actual body weight in overweight and obese patients, may deliver more clotting factor than necessary to cause bleeding to stop in participants with Hemophilia A who use Factor VIII (FVIII). This study also examines ways to prevent delivering too much factor by using a participant's ideal body weight as a new dosing strategy compared to the current dosing strategy. The hypothesis being tested is that factor dosing based on ideal body weight will result in hemostatic factor levels.
The study will be conducted at the Washington Center for Bleeding Disorders (WCBD) at Bloodworks Northwest, Oregon Health \& Science University (OHSU), Seattle Children's Hospital (SCH), and Providence Sacred Heart Children's Hospital (SH). Cumulatively across the four sites, up to 20 participants will be enrolled. Randomization will be performed centrally at WCBD.
Participants will provide their own factor. Prior to the first study-related dose, participants will stop taking any FVIII products for either 48 hours if currently using a short-acting FVIII product or 72 hours for a long acting FVIII product. Factor levels will be measured immediately before and at multiple points after two different factor doses. Subjects will be randomized to start their dosage based either on actual body weight or ideal body weight first and then crossover to receive dosage based on the other category.
Study design
- Primary purpose
- Health services research
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Subjects are randomized to receive factor based either on ideal body weight or actual body weight first followed by receiving factor based on the other category next
| Arm | Type | Receives |
|---|---|---|
| Ideal Body Weight First | Experimental | Other: Ideal Body Weight FirstRandomized to receive factor product based on ideal body weight first |
| Actual Body Weight First | Experimental | Other: Actual Body Weight FirstRandomized to receive factor product based on actual body weight first |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Hemophilia A
- Able and willing to comply with pharmacokinetic testing schedule
- Either overweight or obese BMI using CDC definitions by age
Key exclusion criteria (4 listed)
- Inhibitor of > 0.6 BU twice in the past, or documented abnormal recovery of less than 66% (of expected) in the past
- Known other bleeding disorder
- Known other prolongation in aPTT (lupus anticoagulant, FXII deficiency)
- Female
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ideal Body Weight FirstRandomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on ideal body weight. For participants age 12-19, ideal weight is calculated using the McLaren method. For participants age 20 and over, ideal weight is calculated using the following equation: [50kg + (2.3kg*every inch over 5 feet)]. | Other | Ideal Body Weight First |
| Actual Body Weight FirstRandomized to receive 50 U/kg (+/- 20%) of the factor product participants routinely use based on actual body weight. | Other | Actual Body Weight First |
Primary outcome
Recovery
Time frame: Change from baseline at up to two months
Compare the recovery with FVIII between doses calculated on actual body weight versus ideal body weight in subjects with Hemophilia A
Underdosing
Time frame: Change from baseline at up to two months
Determine the likelihood of underdosing when using ideal body weight
Overdosing
Time frame: Change from baseline at up to two months
Determine the likelihood of overdosing when using actual body weight
4 secondary outcomes
Effect of half-life
Time frame: Change from baseline at up to two months
Effect on hemophilia severity
Time frame: Change from baseline at 20-40 minutes, 5-7 hours, 20-26 hours, and 44-50 hours for both half-life and extended half-life and also at 69-75 hours, and 93-99 hours for extended half-life
Regular half-life vs. extended half-life Regular half-life vs. extended half-life
Time frame: Change from baseline at up to two months
Overweight vs. obese
Time frame: Change from baseline at up to two months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2017.
- Individual participant dataSharing planned: No
Related trials for Hemophilia A
Not yet recruitingPhase 1
Lentiviral FVIII Gene Therapy
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A First-in Human Study of ARD001 in Healthy Volunteers and People With Hemophilia A
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A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A
Haemophilia A
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
RecruitingObservational study
An Observational Study to Learn More About How Well Damoctocog Alfa Pegol Works in Previously Treated Children With Hemophilia A
Hemophilia A
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Rebecca Kruse-Jarres, MD, MPH principal investigator
Washington Center for Bleeding Disorders at Bloodworks Northwest
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Ideal Body Weight First and Actual Body Weight First in people with Hemophilia A, run by Bloodworks at 4 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Heidi Thielmann, PhD (206-689-6234, hthielmann@bloodworksnw.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recovery (time frame: Change from baseline at up to two months). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Sep 2017.