Phase 2
N=20
PF-06741086 Long-term Treatment in Severe Hemophilia
Hemophilia A or B
Bottom Line
View on ClinicalTrials.gov: NCT03363321 ↗Enrolled (actual)
20
Serious AEs
5.0%
Results posted
Jul 2021
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) — 5; 2; 4; 2 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- PF-06741086 (Biological)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- Male
- Sponsor
- Pfizer
- Primary completion
- Aug 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related) |
5; 2; 4; 2; 1; 2 | — |
| PRIMARY Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis) |
1; 1; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria |
0; 0; 0; 0; 0; 0 | — |
| PRIMARY Number of Participants With Abnormalities in Physical Examination Findings |
5; 2; 3; 4; 2 | — |
| PRIMARY Number of Participants With Injection Site Reactions |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Annualized Bleeding Rate (ABR) |
22.000; 14.000; 22.000; 18.400; 15.000; 2.971 | — |
Summary
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study. Additionally, de novo subjects will be recruited into this study.
Eligibility Criteria
Inclusion Criteria
- Severe hemophilia A or B (Factor VIII or Factor IX activity ≤ 1%)
- Subjects enrolled as Factor VIII or Factor IX inhibitor patients must have a positive inhibitor test result (above the upper limit of normal) at the local laboratory and must receive a bypass agent as primary treatment for bleeding episodes.
- Episodic (on-demand) treatment regimen prior to screening
- At least 6 acute bleeding episodes during the 6-month period prior to screening
Exclusion Criteria
- Known coronary artery, thrombotic, or ischemic disease
- Concomitant treatment with activated prothrombin complex concentrate
Data sourced from ClinicalTrials.gov (NCT03363321). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.