Trial Outcomes & Findings for Weight-based Dosing in Hemophilia A (NCT NCT02586012)
NCT ID: NCT02586012
Last Updated: 2020-09-25
Results Overview
Desired peak FVIII recovery value of 2 +/- 0.2 IU/dl per IU/kg (100 +/- 10%) at 10 minutes following infusion based on LBM and TBW. FVIII recovery values will be calculated as follows: \[(weight in kg x observed FVIII recovery in IU/dL)/(dose in IU)\] and expressed as IU/dL per IU/kg.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
3 weeks
Results posted on
2020-09-25
Participant Flow
Participant milestones
| Measure |
TBW, LBM, IBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on TBW (Total Body Weight); Week 2, the dose will be based on LBM (Lean Body Mass); and Week 3, the dose will be based on IBW (Ideal Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
LBM, IBW, TBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on LBM (Lean Body Mass); Week 2, the dose will be based on IBW (Ideal Body Weight); and Week 3, the dose will be based on TBW (Total Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
IBW, TBW, LBM
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on IBW (Ideal Body Weight); Week 2, the dose will be based on TBW (Total Body Weight); and Week 3, the dose will be based on LBM (Lean Body Mass).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
TBW, IBW, LBM
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on TBW (Total Body Weight); Week 2, the dose will be based on IBW (Ideal Body Weight); and Week 3, the dose will be based on LBM (Lean Body Mass).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
LBM, TBW, IBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on LBM (Lean Body Mass); Week 2, the dose will be based on TBW (Total Body Weight); and Week 3, the dose will be based on IBW (Ideal Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
IBW, LBM, TBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on, IBW (Ideal Body Weight); Week 2, the dose will be based on LBM (Lean Body Mass); and Week 3, the dose will be based on TBW (Total Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
|---|---|---|---|---|---|---|
|
Overall Study
STARTED
|
5
|
5
|
5
|
5
|
5
|
5
|
|
Overall Study
COMPLETED
|
4
|
2
|
4
|
3
|
4
|
2
|
|
Overall Study
NOT COMPLETED
|
1
|
3
|
1
|
2
|
1
|
3
|
Reasons for withdrawal
| Measure |
TBW, LBM, IBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on TBW (Total Body Weight); Week 2, the dose will be based on LBM (Lean Body Mass); and Week 3, the dose will be based on IBW (Ideal Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
LBM, IBW, TBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on LBM (Lean Body Mass); Week 2, the dose will be based on IBW (Ideal Body Weight); and Week 3, the dose will be based on TBW (Total Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
IBW, TBW, LBM
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on IBW (Ideal Body Weight); Week 2, the dose will be based on TBW (Total Body Weight); and Week 3, the dose will be based on LBM (Lean Body Mass).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
TBW, IBW, LBM
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on TBW (Total Body Weight); Week 2, the dose will be based on IBW (Ideal Body Weight); and Week 3, the dose will be based on LBM (Lean Body Mass).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
LBM, TBW, IBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on LBM (Lean Body Mass); Week 2, the dose will be based on TBW (Total Body Weight); and Week 3, the dose will be based on IBW (Ideal Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
IBW, LBM, TBW
Subjects will be randomized to receive rFVIII doses calculated by 3 separate methods over a 3 week period. Week 1, the dose will be based on, IBW (Ideal Body Weight); Week 2, the dose will be based on LBM (Lean Body Mass); and Week 3, the dose will be based on TBW (Total Body Weight).
rFVIII: Recombinant FVIII concentrate is an FDA approved, and both efficacious and safe, therapy for the treatment and prevention of bleeding in hemophilia A. The rFVIII infusion dose will be calculated as follows: \[(weight in kg x desired FVIII increase of 100 IU/dL)/(2)\].
|
|---|---|---|---|---|---|---|
|
Overall Study
Withdrawal by Subject
|
1
|
1
|
0
|
1
|
0
|
2
|
|
Overall Study
Protocol Violation
|
0
|
0
|
0
|
0
|
0
|
1
|
|
Overall Study
study terminated
|
0
|
2
|
1
|
1
|
1
|
0
|
Baseline Characteristics
Weight-based Dosing in Hemophilia A
Baseline characteristics by cohort
| Measure |
Subjects
n=30 Participants
All subjects randomized to 1 of 6 possible dosing sequence scenarios based on 3 different weight-based dosing regimens (TBW, LBM, and IBW).
|
|---|---|
|
Age, Continuous
|
34.6 years
STANDARD_DEVIATION 11.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
30 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
30 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Height
|
178.5 cm
STANDARD_DEVIATION 6.9 • n=5 Participants
|
|
Weight
|
93.0 kg
STANDARD_DEVIATION 13.6 • n=5 Participants
|
|
BMI
|
29.2 kg/m^2
STANDARD_DEVIATION 3.5 • n=5 Participants
|
PRIMARY outcome
Timeframe: 3 weeksPopulation: Subjects receiving rFVIII based on LBM and TBW.
Desired peak FVIII recovery value of 2 +/- 0.2 IU/dl per IU/kg (100 +/- 10%) at 10 minutes following infusion based on LBM and TBW. FVIII recovery values will be calculated as follows: \[(weight in kg x observed FVIII recovery in IU/dL)/(dose in IU)\] and expressed as IU/dL per IU/kg.
Outcome measures
| Measure |
Lean Body Mass
n=20 Participants
Subjects receiving rFVIII based on LBM.
|
Total Body Weight
n=20 Participants
Subjects receiving rFVIII based on TBW.
|
|---|---|---|
|
Proportion of Subjects Dosed Based on LBM (Lean Body Mass) and TBW (Total Body Weight) Achieving Desired Peak FVIII Recovery.
|
9 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: 3 weeksPopulation: Subjects receiving rFVIII based on IBW and TBW.
Desired peak FVIII recovery value of 2 +/- 0.2 IU/dl per IU/kg (100 +/- 10%) at 10 minutes following infusion based on IBW and TBW. FVIII recovery values will be calculated as follows: \[(weight in kg x observed FVIII recovery in IU/dL)/(dose in IU)\] and expressed as IU/dL per IU/kg.
Outcome measures
| Measure |
Lean Body Mass
n=20 Participants
Subjects receiving rFVIII based on LBM.
|
Total Body Weight
n=20 Participants
Subjects receiving rFVIII based on TBW.
|
|---|---|---|
|
Proportion of Subjects Dosed Based on IBW (Ideal Body Weight) and TBW (Total Body Weight) Achieving Desired Peak FVIII Recovery.
|
10 Participants
|
6 Participants
|
SECONDARY outcome
Timeframe: 3 weeksPopulation: Subjects receiving rFVIII based on LBM and IBW.
Desired peak FVIII recovery value of 2 +/- 0.2 IU/dl per IU/kg (100 +/- 10%) at 10 minutes following infusion based on LBM and IBW. FVIII recovery values will be calculated as follows: \[(weight in kg x observed FVIII recovery in IU/dL)/(dose in IU)\] and expressed as IU/dL per IU/kg.
Outcome measures
| Measure |
Lean Body Mass
n=20 Participants
Subjects receiving rFVIII based on LBM.
|
Total Body Weight
n=20 Participants
Subjects receiving rFVIII based on TBW.
|
|---|---|---|
|
Proportion of Subjects Dosed Based on LBM (Lean Body Mass) and IBW (Ideal Body Weight) Achieving Desired Peak FVIII Recovery.
|
9 Participants
|
10 Participants
|
Adverse Events
Total Body Weight
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Ideal Body Weight
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Lean Body Mass
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place