Trial · NCT00543439
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
NCT00543439, Phase 3, Completed, Pfizer; results first posted 2019-01-11
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII. Trials, record NCT00543439. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT00543439
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
- NCT number
- NCT00543439
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 66 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Hemophilia A
- Interventions
- Moroctocog alfa (AF-CC), Moroctocog alfa (AF-CC)
- First posted
- 2007-10-15
- Last updated
- 2019-01-11
- Other registries' IDs
- eudract:2006-005575-17
- Results first posted
- 2019-01-11
Source ClinicalTrials.gov
Registered outcomes23
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort | Day 1 up to Month 6 (OD Cohort, OD Therapy, Period 1); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ABR for each participant was calculated as the number of bleeds requiring administration of moroctocog alfa (AF-CC) divided by the total therapy duration (in days), then multiplied by 365.25 (days in a year). |
| Secondary | Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort | Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2) | ABR for each participant was calculated as the number of bleeds requiring administration of moroctocog alfa (AF-CC) divided by the total therapy duration (in days), then multiplied by 365.25 (days in a year). |
| Secondary | Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants | Day 1 up to Month 24 | In this outcome measure, the mean of total number of moroctocog alfa (AF-CC) on-demand infusions administered to treat each bleeding episode was reported, regardless of participant cohort or period during which it occurred. |
| Secondary | Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Day 1 up to Month 24 | Number (no.) of bleeds treated are reported on basis of response to first infusion of study drug, at 4-point scale: excellent, good, moderate, no response. Excellent:definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good:definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate:probable or slight improvement starting after 8 hr following infusion,at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen. Bleeds for which response not recorded, reported as:Data Not Recorded. Total no. of first infusions may not be equal to total no. of bleeds if bleed was: missing start date/dose information or treated initially with non-study FVIII. |
| Secondary | Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | In this outcome measure number of treated spontaneous bleeds are reported according to the time interval between bleed onset and prior moroctocog alfa (AF-CC) routine prophylaxis dose. Following time intervals used to report this outcome measure: lesser than or equal to (\<=) 24 hours, greater than (\>) 24 hours to \<=48 hours, \>48 hours to \<=72 hours, \>72 hours. For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg" cumulative data for routine prophylaxis cohort (Day 1 up to Month 24, Period 1 and Period 2) and on demand cohort (Month 7 up to Month 18, Period 2) is reported. |
| Secondary | Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | During prophylaxis, criteria for prophylaxis regimen escalation are the occurrence, over a 4-week duration (and in the absence of a confirmed FVIII inhibitor), of (a) 2 or more spontaneous bleeds into a major joint and/or target joint, or (b) 3 or more spontaneous bleeds (consisting of joint bleeds and/or significant soft tissue/muscle or other site bleeds). If either criterion was met, the participant was escalated to a more intense prophylaxis regimen of 45 IU/kg, administered every other day. Participant who meet dose escalation criteria while on prophylaxis regimen of 45 IU/kg, were escalated to a higher intensity regimen designated by the investigator. Significant spontaneous bleeds were those that led to a transient or persistent loss of function. For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg", cumulative data for RP cohort (Day 1 up to Month 24, Period 1 and Period 2) and OD cohort (Month 7 up to Month 18, Period 2) is reported. |
| Secondary | Mean Routine Prophylaxis Dose (IU/kg) of Moroctocog Alfa (AF-CC) Received: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | Mean RP dose (by weight) for each participant was calculated as his total moroctocog alfa (AF-CC) consumption (in IU) divided by weight (in kg). For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg", cumulative data for routine prophylaxis cohort (Day 1 up to Month 24, Period 1 and Period 2) and on demand cohort (Month 7 up to Month 18, Period 2) is reported. |
| Secondary | Mean of Total Number Moroctocog Alfa (AF-CC) Infusions Received: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | In this outcome measure mean of total number of infusions of moroctocog alfa (AF-CC) received by participant is reported. For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg", cumulative data for routine prophylaxis cohort (Day 1 up to Month 24, Period 1 and Period 2) and on demand cohort (Month 7 up to Month 18, Period 2) is reported. |
| Secondary | Mean of Total Number of Days Participants Exposed to Moroctocog Alfa (AF-CC): Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg", cumulative data for routine prophylaxis cohort (Day 1 up to Month 24, Period 1 and Period 2) and on demand cohort (Month 7 up to Month 18, Period 2) is reported. |
| Secondary | Mean of Total Number of Infusions of Moroctocog Alfa (AF-CC) Received Per Week to Assess Compliance: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | Participants' compliance to their assigned prophylaxis regimen was measured by following: a) number of infusions received per week and b) dose received. In this outcome measure mean of total number of infusions of moroctocog alfa (AF-CC) received by participants per week is reported. For reporting arm: "Moroctocog alfa (AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg" cumulative data for routine prophylaxis cohort (Day 1 up to Month 24, Period 1 and Period 2) and on demand cohort (Month 7 up to Month 18, Period 2) is reported. |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 registered outcomesOutcomes23
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort | Day 1 up to Month 6 (OD Cohort, OD Therapy, Period 1); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | Intent-to-treat (ITT) analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Bleeds per year | MEAN |
| SECONDARY | Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort | Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Bleeds per year | MEAN |
| SECONDARY | Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants | Day 1 up to Month 24 | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Infusions | MEAN |
| SECONDARY | Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Day 1 up to Month 24 | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Bleeds | NUMBER |
| SECONDARY | Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | Analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form and who reported a spontaneous bleeding episode following a routine prophylaxis dose. | Bleeds | NUMBER |
| SECONDARY | Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Routine Prophylaxis Dose (IU/kg) of Moroctocog Alfa (AF-CC) Received: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | IU/kg | MEAN |
| SECONDARY | Mean of Total Number Moroctocog Alfa (AF-CC) Infusions Received: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Infusions | MEAN |
| SECONDARY | Mean of Total Number of Days Participants Exposed to Moroctocog Alfa (AF-CC): Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Days | MEAN |
| SECONDARY | Mean of Total Number of Infusions of Moroctocog Alfa (AF-CC) Received Per Week to Assess Compliance: Routine Prophylaxis Therapy | Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); Month 7 up to Month 18 (OD Cohort, RP 25 IU/kg, Period 2) | ITT analysis population included all participants for whom a legal acceptable representative had signed the informed consent/assent form. Here, Overall Number of Participants Analyzed signifies participants who were evaluable for this outcome measure. | Infusions per week | MEAN |
1 to 10 of 23Source ClinicalTrials.gov
See all 23 outcomesResults by group54
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort | Moroctocog Alfa (AF-CC), On Demand Cohort: On Demand Therapy | 47.0 | Standard Deviation 32.2 | Bleeds per year | |
| Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort | Moroctocog Alfa (AF-CC), On Demand Cohort: RP Therapy 25 IU/kg | 1.5 | Standard Deviation 2.2 | Bleeds per year | |
| Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort | Moroctocog Alfa (AF-CC), RP Cohort: RP Therapy 45 IU/kg | 3.3 | Standard Deviation 5.3 | Bleeds per year | |
| Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort | Moroctocog Alfa (AF-CC), RP Cohort: RP Therapy 25 IU/kg | 2.2 | Standard Deviation 4.1 | Bleeds per year | |
| Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants | Moroctocog Alfa (AF-CC): All Participants | 1.5 | Standard Deviation 1.38 | Infusions | |
| Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Moroctocog Alfa (AF-CC): On Demand Therapy | Excellent | 376 | Bleeds | |
| Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Moroctocog Alfa (AF-CC): On Demand Therapy | Good | 150 | Bleeds | |
| Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Moroctocog Alfa (AF-CC): On Demand Therapy | Moderate | 27 | Bleeds | |
| Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Moroctocog Alfa (AF-CC): On Demand Therapy | No Response | 2 | Bleeds | |
| Number of Treated Bleeds Classified on Basis of Response to First Infusion of Moroctocog Alfa (AF-CC) as On-Demand Treatment: OD Therapy (OD and RP Cohort) | Moroctocog Alfa (AF-CC): On Demand Therapy | Data Not Recorded | 4 | Bleeds |
1 to 10 of 54Source ClinicalTrials.gov
See all 54 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Period 1 | STARTED | Moroctocog Alfa (AF-CC),OD Cohort:OD Therapy Then RP Therapy | 10 | |
| Period 1 | STARTED | Moroctocog Alfa(AF-CC),RP Cohort:RP 25 IU/kg Then RP 45IU/kg | 29 | |
| Period 1 | STARTED | Moroctocog Alfa(AF-CC), RP Cohort:RP 45 IU/kg Then RP25IU/kg | 27 | |
| Period 1 | Treated | Moroctocog Alfa (AF-CC),OD Cohort:OD Therapy Then RP Therapy | 9 | |
| Period 1 | Treated | Moroctocog Alfa(AF-CC),RP Cohort:RP 25 IU/kg Then RP 45IU/kg | 29 | |
| Period 1 | Treated | Moroctocog Alfa(AF-CC), RP Cohort:RP 45 IU/kg Then RP25IU/kg | 27 | |
| Period 1 | COMPLETED | Moroctocog Alfa (AF-CC),OD Cohort:OD Therapy Then RP Therapy | 9 | |
| Period 1 | COMPLETED | Moroctocog Alfa(AF-CC),RP Cohort:RP 25 IU/kg Then RP 45IU/kg | 26 | |
| Period 1 | COMPLETED | Moroctocog Alfa(AF-CC), RP Cohort:RP 45 IU/kg Then RP25IU/kg | 25 | |
| Period 1 | NOT COMPLETED | Moroctocog Alfa (AF-CC),OD Cohort:OD Therapy Then RP Therapy | 1 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events49
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Upper respiratory tract infection | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 16 | 51 | |
| other | Nasopharyngitis | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 14 | 51 | |
| other | Influenza | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 8 | 51 | |
| other | Tonsillitis | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 7 | 51 | |
| other | Pharyngitis | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 5 | 51 | |
| other | Varicella | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 4 | 51 | |
| other | Viral infection | Infections and infestations | Moroctocog Alfa (AF-CC): All Participants | 3 | 51 | |
| other | Pyrexia | General disorders | Moroctocog Alfa (AF-CC): All Participants | 24 | 51 | |
| other | Fatigue | General disorders | Moroctocog Alfa (AF-CC): All Participants | 4 | 51 | |
| other | Joint injury | Injury, poisoning and procedural complications | Moroctocog Alfa (AF-CC): All Participants | 10 | 51 |
1 to 10 of 49Source ClinicalTrials.gov
See all 49 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Moroctocog Alfa (AF-CC): All Participants | 0 | 51 |
| Total, other adverse events | Moroctocog Alfa (AF-CC): All Participants | 49 | 51 |
| Total, serious adverse events | Moroctocog Alfa (AF-CC): All Participants | 13 | 51 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
Moroctocog Alfa (AF-CC), RP Cohort: RP Therapy 45 IU/kg vs Moroctocog Alfa(AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg: Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): Routine Prophylaxis Therapy | Trial comparisons |
Moroctocog Alfa (AF-CC), RP Cohort: RP Therapy 45 IU/kg vs Moroctocog Alfa(AF-CC),OD and RP Cohort: RP Therapy 25 IU/kg: Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT00543439
GET https://conductscience.org/api/trials/v1/NCT00543439/registered?page=2