Trial · NCT03363321
PF-06741086 Long-term Treatment in Severe Hemophilia
NCT03363321, Phase 2, Completed, Pfizer; results first posted 2021-07-27
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. PF-06741086 Long-term Treatment in Severe Hemophilia. Trials, record NCT03363321. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03363321
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- PF-06741086 Long-term Treatment in Severe Hemophilia
- NCT number
- NCT03363321
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 20 participants, actual
- Lead sponsor
- Pfizer
- Conditions
- Hemophilia A or B
- Interventions
- PF-06741086
- First posted
- 2017-12-06
- Last updated
- 2021-07-27
- Other registries' IDs
- eudract:2017-001255-31
- Results first posted
- 2021-07-27
Source ClinicalTrials.gov
Registered outcomes7
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Day 1 up to Day 393 | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product; the event did not need to have a causal relationship with the treatment. A serious adverse event (SAE) was any untoward medical occurrence at any dose that resulted in death; was life threatening; required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both SAEs and non-serious AEs. TEAEs were AEs occurred following the start of treatment or AEs increasing in severity during treatment. Treatment-related TEAEs were determined by the investigator. Grades 3 AEs were severe and undesirable adverse events. Grades 4 AEs were life threatening or disabling adverse events. |
| Primary | Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis) | Hematology and serum chemistry: Baseline, Days 1, 29, 57, 85, 169, 253, and 365 visits. Urinalysis: Baseline, Days 1, 85, 169, 253, and 365 visits. | Following parameters were analyzed for laboratory examination: hematology, clinical chemistry, and urinalysis. The hematology parameters and pre-defined criteria included: neutrophils (10\^3/millimeter\[mm\]\^3) \<0.8\*lower limit of normal (LLN), and basophils (10\^3/mm\^3) \>1.2\*upper limit of normal (ULN). The clinical chemistry parameter and pre-defined criteria included: bilirubin (milligrams \[mg\]/decilitre \[dL\]) \>1.5 ULN, aspartate aminotransferase (units \[U\]/liter \[L\]) \>3.0 ULN, glucose (mg/dL) \>1.5\*ULN. The urinalysis parameter and pre-defined criteria included: urine glucose ≥1, ketones (scalar) ≥1, urine protein ≥1, urine hemoglobin (scalar) ≥1, and hyaline casts per low power field (/LPF). Participants met criteria at any time point were included. |
| Primary | Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria | Baseline, Days 1, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365 and 393 visits. | Following parameters were analyzed for vital sign examination: blood pressure (BP), pulse rate (PR), temperature, respiration rate. Categorical vital signs: Temperature \>38.5 degree(s) Celsius (℃), Supine PR: \<40 or \>120 beats per minute (BPM), Systolic BP: \<90 mm Hg, \>=30 mm Hg change from baseline, Diastolic BP: \<50 mm Hg, \>=20 mm Hg change from baseline. |
| Primary | Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria | Baseline, Days 1 and 29 visits | Baseline was defined as the average of triplicate ECG measurements collected prior to dosing on Day 1 in B7841003. Criteria for potentially clinically important changes in ECG were defined as: PR interval value \>=300 millisecond (msec); PR interval baseline \>200 msec and change \>=25%; PR interval baseline \<=200 msec and change \>=50%; QRS complex value \>=140 msec and change \>=50%; QTcF value \>=450 msec and change \>=30 msec. Only the number of participants meeting pre-defined criteria was reported below. |
| Primary | Number of Participants With Abnormalities in Physical Examination Findings | Day 1 to Day 393 | Physical examinations were conducted by a physician, trained physician's assistant, or nurse practitioner as acceptable according to local regulation. A full physical examination included head, ears, eyes, nose, mouth, skin, heart and lung examinations, lymph nodes, gastrointestinal, musculoskeletal, and neurological systems. The limited or abbreviated physical examination was focused on general appearance, the respiratory and cardiovascular systems, as well as towards participant reported symptoms. For measuring weight, a scale with appropriate range and resolution was used and must have been placed on a stable, flat surface. Participants removed shoes, bulky layers of clothing, and jackets so that only light clothing remains. They also removed the contents of their pockets and remain still during measurement of weight. |
| Primary | Number of Participants With Injection Site Reactions | Day 1 to Day 365, and Day 393 visit. | Injection site reactions included but were not limited to: erythema, induration, ecchymosis, pain and pruritus. Grade of severity was defined as follows: Mild: Transient or mild discomfort (\< 48 hours); no medical intervention/therapy required. Moderate: Mild to moderate limitation in activity - some assistance may be needed; no or minimal medical intervention/therapy required. Severe: Marked limitation in activity, some assistance usually required; medical intervention/therapy required, hospitalizations possible. |
| Secondary | Annualized Bleeding Rate (ABR) | Day 1 to Day 365, and Day 393 visit. Pre-Treatment summarized the data up to 6 months prior to participation in B7841003 for de novo participants and up to 6 months prior to participation in B7841002 for roll over participants. | The ABR was calculated as (\[number of bleeding events × 365.25\] / observed treatment period in days) |
Source ClinicalTrials.gov
Outcomes7
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Day 1 up to Day 393 | The analysis population included all participants who received at least 1 dose of investigational product. Here Overall Number of Participants Analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis) | Hematology and serum chemistry: Baseline, Days 1, 29, 57, 85, 169, 253, and 365 visits. Urinalysis: Baseline, Days 1, 85, 169, 253, and 365 visits. | This analysis population included all participants who received at least 1 dose of investigational product. Here Overall Number of Participants Analyzed signifies number of participants who were evaluable for this outcome measure. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria | Baseline, Days 1, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365 and 393 visits. | This analysis population included all participants who received at least 1 dose of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria | Baseline, Days 1 and 29 visits | ECG was only evaluated in de novo participants. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Abnormalities in Physical Examination Findings | Day 1 to Day 393 | The analysis population included all participants who received at least 1 dose of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Number of Participants With Injection Site Reactions | Day 1 to Day 365, and Day 393 visit. | This analysis population included all participants who received at least 1 dose of investigational product. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Annualized Bleeding Rate (ABR) | Day 1 to Day 365, and Day 393 visit. Pre-Treatment summarized the data up to 6 months prior to participation in B7841003 for de novo participants and up to 6 months prior to participation in B7841002 for roll over participants. | This efficacy analysis was conducted in the Per Protocol Analysis Set (PPAS) that included all participants who received at least 1 dose of investigational product and did not have any major protocol deviations. | Bleeding episodes/participant/year | MEAN |
Source ClinicalTrials.gov
Results by group255
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 1: 300mg - 300mg Non-Inhibitor | All-causalities TEAE | 5 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | All-causalities TEAE | 2 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | All-causalities TEAE | 4 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 4: 300mg - 300mg Inhibitor | All-causalities TEAE | 2 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | All-causalities TEAE | 1 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 1: 300mg - 300mg Non-Inhibitor | Treatment-related TEAE | 2 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | Treatment-related TEAE | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | Treatment-related TEAE | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 4: 300mg - 300mg Inhibitor | Treatment-related TEAE | 0 | Participants | |
| Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | Treatment-related TEAE | 1 | Participants |
1 to 10 of 255Source ClinicalTrials.gov
See all 255 results by groupParticipant flow15
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Cohort 1: 300mg - 300mg Non-Inhibitor | 5 | |
| Overall Study | STARTED | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 4 | |
| Overall Study | STARTED | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 4 | |
| Overall Study | STARTED | Cohort 4: 300mg - 300mg Inhibitor | 5 | |
| Overall Study | STARTED | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 2 | |
| Overall Study | COMPLETED | Cohort 1: 300mg - 300mg Non-Inhibitor | 5 | |
| Overall Study | COMPLETED | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 3 | |
| Overall Study | COMPLETED | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 4 | |
| Overall Study | COMPLETED | Cohort 4: 300mg - 300mg Inhibitor | 4 | |
| Overall Study | COMPLETED | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 2 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 participant flowAdverse events165
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 | Food poisoning | Gastrointestinal disorders | Cohort 1: 300mg - 300mg Non-Inhibitor | 0 | 5 | 0 |
| other | Food poisoning | Gastrointestinal disorders | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 | 0 |
| other | Food poisoning | Gastrointestinal disorders | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 | 0 |
| other | Food poisoning | Gastrointestinal disorders | Cohort 4: 300mg - 300mg Inhibitor | 0 | 5 | 0 |
| other | Food poisoning | Gastrointestinal disorders | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 1 | 2 | 1 |
| other | Nausea | Gastrointestinal disorders | Cohort 1: 300mg - 300mg Non-Inhibitor | 0 | 5 | 0 |
| other | Nausea | Gastrointestinal disorders | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 | 0 |
| other | Nausea | Gastrointestinal disorders | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 | 0 |
| other | Nausea | Gastrointestinal disorders | Cohort 4: 300mg - 300mg Inhibitor | 0 | 5 | 0 |
| other | Nausea | Gastrointestinal disorders | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 1 | 2 | 1 |
1 to 10 of 165Source ClinicalTrials.gov
See all 165 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: 300mg - 300mg Non-Inhibitor | 0 | 5 |
| Total, all-cause mortality | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 |
| Total, all-cause mortality | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 0 | 4 |
| Total, all-cause mortality | Cohort 4: 300mg - 300mg Inhibitor | 0 | 5 |
| Total, all-cause mortality | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 0 | 2 |
| Total, other adverse events | Cohort 1: 300mg - 300mg Non-Inhibitor | 5 | 5 |
| Total, other adverse events | Cohort 2: 300mg Loading + 150mg - 300mg Loading + 150mg Non-Inhibitor | 2 | 4 |
| Total, other adverse events | Cohort 3: 450mg - 300mg Loading + 150mg Non-Inhibitor | 4 | 4 |
| Total, other adverse events | Cohort 4: 300mg - 300mg Inhibitor | 2 | 5 |
| Total, other adverse events | Cohort 5: De Novo 300mg Loading + 150mg Inhibitors | 1 | 2 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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/NCT03363321
GET https://conductscience.org/api/trials/v1/NCT03363321/results?page=2