Adults 7 to 65, any sex, with Hemophilia A. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Mean Transformed Annualized Bleed Rate Estimates From Each of the 1-year Prophylaxis RegimensPrimary· 12 months ±2 weeks
Participants were Randomized to Receive 1 of the 2 Following Prophylaxis Regimens (Study Part 2): 1. Standard prophylaxis (20-40 IU/kg (every 48 ±6 hour), exact regimen determined by investigator) 2. PK-driven prophylaxis (20-80 IU/kg (every 72 ±6 hour), exact regimen determined by sponsor) Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X = bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the t-test.
Group
Value
95% CI
PK-Driven Prophylaxis
1.61
± 1.10
Standard Prophylaxis
1.46
± 0.98
Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and Standard Prophylaxis Treatment RegimensSecondary· On-demand 6 months (± 2 weeks); followed by Prophylaxis 12 months (± 2 weeks)
Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the paired t-test. Mean Difference of Transformed Annualized Bleeding Rate (TABR) = (On-Demand Treatment TABR) - (Standard Prophylaxis Treatment TABR). Participants from the On-Demand portion of the study were subsequently randomized to either Standard Prophylaxis or PK-Driven Prophylaxis, (i.e the same participan
Group
Value
95% CI
On-Demand Versus Standard Prophylaxis
5.29
± 1.46
Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and PK-Driven Prophylaxis Treatment RegimensSecondary· On-demand 6 months (± 2 weeks); followed by Prophylaxis 12 months (± 2 weeks)
Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the paired t-test. Mean Difference of Transformed Annualized Bleeding Rate (TABR) = (On-Demand Treatment TABR) - (PK-Driven Prophylaxis Treatment TABR) Participants from the On-Demand portion of the study were subsequently randomized to either Standard Prophylaxis or PK-Driven Prophylaxis, (i.e the same participan
Group
Value
95% CI
On-Demand Versus PK-Driven Prophylaxis
5.00
± 1.85
Mean Difference of Transformed Annualized Bleeding Rate Between On-Demand and Any Prophylaxis Treatment RegimensSecondary· On-demand 6 months (± 2 weeks); Prophylaxis 12 months (± 2 weeks)
Annualized bleed rates were transformed using the square root of the number of bleeding episodes observed (X bleeds/year), X' = √(X + 0.5). This transformation was performed to stabilize the variance and align the sample distribution with the assumption of normality inherent in using the paired t-test. Mean Difference of Transformed Annualized Bleeding Rate (TABR) = (On-Demand Treatment TABR) - (Any Prophylaxis Treatment TABR). Any Prophylaxis = Standard or PK-Driven Prophylaxis Participants from the On-Demand portion of the study were subsequently randomized to either Standard Prophylaxis or
Group
Value
95% CI
On-Demand Versus Any Prophylaxis
5.14
± 1.66
Total Weight-Adjusted Dose of rAHF-PFM Used Per Year for Each Prophylaxis ArmSecondary· 12 months ±2 weeks
Participants were Randomized to Receive 1 of the 2 Following Prophylaxis Regimens (Part 2 of the study): 1. Standard prophylaxis- infusions every 48 ±6 hours, dosed at 20 to 40 IU/kg. 2. PK-driven prophylaxis- infusions every 72 ±6 hours dosed at 20 to 80 IU/kg.
Group
Value
95% CI
PK-Driven Prophylaxis
5197.8
3268.4 – 8273.5
Standard Prophylaxis
5768.2
4728.0 – 6425.4
Bleeding Episodes Treated With 1 to ≥4 InfusionsSecondary· Throughout the study period (4 years and 5 months)
The number of bleeding episodes treated with 1, 2, 3, or ≥4 infusions of rAHF-PFM to achieve adequate hemostasis
1 infusion (n = 62, 13, 22)
Group
Value
95% CI
On-Demand Treatment
1168
Standard Prophylaxis Treatment
68
PK-Driven Prophylaxis
90
2 infusions (n = 51, 6, 9)
Group
Value
95% CI
On-Demand Treatment
277
Standard Prophylaxis Treatment
12
PK-Driven Prophylaxis
37
3 infusions (n = 27, 2, 4)
Group
Value
95% CI
On-Demand Treatment
128
Standard Prophylaxis Treatment
4
PK-Driven Prophylaxis
5
4 or more infusions (n = 21, 5, 5)
Group
Value
95% CI
On-Demand Treatment
50
Standard Prophylaxis Treatment
9
PK-Driven Prophylaxis
7
Assessment of Hemostasis for Treatment of Bleeding EpisodesSecondary· On-demand 6 months (± 2 weeks); Prophylaxis 12 months (± 2 weeks)
Number of rAHF-PFM-treated bleeding episodes with an assessment of hemostasis (4-point ordinal scale): Excellent: Full pain relief \& bleeding cessation within \~8 hrs of 1 infusion. Additional infusions may have been given to maintain hemostasis; Good: Definite pain relief and/or improvement in bleeding within \~8 hrs after infusion. Possibly requires \>1 infusion for complete resolution; Fair: Probable or slight relief of pain \& slight improvement in bleeding within \~8 hrs after infusion. Requires \>1 infusion for complete resolution; None: No improvement or condition worsens
Excellent
Group
Value
95% CI
On-Demand Regimen
547
Standard Prophylaxis
39
PK-Driven Prophylaxis
33
Good
Group
Value
95% CI
On-Demand Regimen
943
Standard Prophylaxis
38
PK-Driven Prophylaxis
75
Fair
Group
Value
95% CI
On-Demand Regimen
167
Standard Prophylaxis
16
PK-Driven Prophylaxis
11
None
Group
Value
95% CI
On-Demand Regimen
3
Standard Prophylaxis
0
PK-Driven Prophylaxis
20
Unknown
Group
Value
95% CI
On-Demand Regimen
13
Standard Prophylaxis
0
PK-Driven Prophylaxis
0
Total Area Under the Curve (AUC)Secondary· Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Total AUC estimated by AUC 0-48h plus an area extrapolated from the log-linear regression model
Group
Value
95% CI
≥14 Years of Age
1334.45
± 454.33
<14 Years of Age
1061.26
± 452.87
Area Under the CurveSecondary· Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Area under the factor VIII (FVIII) plasma concentration versus time curve (AUC) from 0 to 48 hours estimated using the linear trapezoidal method
Group
Value
95% CI
≥14 Years of Age
1213.98
± 323.96
<14 Years of Age
966.68
± 330.83
Maximum Plasma Concentration (C-max)Secondary· Within 1 hour post-infusion
Change in factor VIII concentration from pre- to post-infusion at initial and termination study visits. Adjusted IR defined as: \[Cmax (IU/dL) - pre-infusion FVIII (IU/dL)\]/dose (IU/kg)
Group
Value
95% CI
≥14 Years of Age
1.81
± 0.41
<14 Years of Age
1.47
± 0.27
Terminal Half-lifeSecondary· Pharmacokinetic evaluations: 30 minutes pre-infusion up to 48 hours post-infusion
Computed from the regression slope in the terminal phase of the model. Terminal half life is the time it takes for the plasma concentration or the amount of drug in the body to be reduced by 50%.
Group
Value
95% CI
≥14 Years of Age
13.91
± 5.07
<14 Years of Age
14.66
± 5.21
Adverse events — posted to ClinicalTrials.gov
Time frame: Throughout the study period (4 years and 5 months).
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40 IU/kg every 48 ± 6 hours; actual dose determined by the investigator) with that of alternate prophylaxis (20-80 IU/kg every 72 + 6 hours; actual dose determined by Baxter utilizing an algorithm and the patient's pharmacokinetic data). The rates of bleeding episodes for the on-demand regimen and the prophylaxis regimens will also be compared for the cross-over portion of the study. Enrolled patients will be treated originally on demand for a period of 6 months and then they will be randomized into one of the prophylaxis arms. Prophylactic treatment will last for a period of 12 months +/- 2 weeks.
Publications & conference data
2 peer-reviewed publications reference this trial (live from Europe PMC):
Other trials of Antihemophilic factor, recombinant, manufactured protein-free
Trials testing the same drug.
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Currently open trials in the same condition.
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Other Baxalta now part of Shire trials
Trials by the same sponsor.
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Baxalta now part of Shire
Last refreshed: 19 May 2021
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT00243386.