Clinical trial NCT03363321: PF-06741086 Long-term Treatment in Severe Hemophilia
Official title: A MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY AND EFFICACY OF SUBCUTANEOUS PF-06741086 IN SUBJECTS WITH SEVERE HEMOPHILIA
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hemophilia A or B
Keywords: hemophilia
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2017-12-06
Enrollment: 20
Sex: MALE
Minimum age (years): 12
Maximum age (years): 74
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SEQUENTIAL
Primary purpose: PREVENTION

Brief 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

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs), TEAEs by Severity, and Serious Adverse Events (SAEs) (All Causality and Treatment-Related)
timeFrame: Day 1 up to Day 393
measure: Number of Participants With Abnormal Laboratory Findings Without Regard to Baseline Abnormality (Including Hematology, Serum Chemistry, and Urinalysis)
timeFrame: 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.
measure: Number of Participants With Changes From Baseline in Vital Signs Measurements Meeting the Pre-Defined Categorical Summarization Criteria
timeFrame: Baseline, Days 1, 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365 and 393 visits.
measure: Number of Participants With Change From Baseline in Electrocardiogram (ECG) Parameters Meeting the Pre-defined Categorical Summarization Criteria
timeFrame: Baseline, Days 1 and 29 visits
measure: Number of Participants With Abnormalities in Physical Examination Findings
timeFrame: Day 1 to Day 393
measure: Number of Participants With Injection Site Reactions
timeFrame: Day 1 to Day 365, and Day 393 visit.
Secondary outcomes:
measure: Annualized Bleeding Rate (ABR)
timeFrame: 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.
Other outcomes:
Locations:
city: Aurora
country: United States
facility: UC Denver Hemophilia and Thrombosis Center
state: Colorado
city: Campinas
country: Brazil
facility: Centro de Hematologia e Hemoterapia de Campinas- Hemocentro de Campinas
state: SAO Paulo
city: Santiago
country: Chile
facility: Hospital Dr. Sotero del Rio
state: Puente ALTO
city: Zagreb
country: Croatia
facility: Klinicki bolnicki centar Zagreb
city: Gdansk
country: Poland
facility: Klinika Hematologii i Transplantologii Uniwersyteckie Centrum Kliniczne
city: Port Elizabeth
country: South Africa
facility: Phoenix Pharma (Pty) Ltd
state: Eastern CAPE
city: Johannesburg
country: South Africa
facility: Charlotte Maxeke Johannesburg Academic Hospital
state: Gauteng
city: Zurich
country: Switzerland
facility: UniversitatsSpital Zurich
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References:
citation: Mahlangu J, Luis Lamas J, Cristobal Morales J, Malan DR, Teeter J, Charnigo RJ, Hwang E, Arkin S. Long-term safety and efficacy of the anti-tissue factor pathway inhibitor marstacimab in participants with severe haemophilia: Phase II study results. Br J Haematol. 2023 Jan;200(2):240-248. doi: 10.1111/bjh.18495. Epub 2022 Oct 11.