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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT01439971




Registration number
NCT01439971
Ethics application status
Date submitted
26/08/2011
Date registered
23/09/2011

Titles & IDs
Public title
Phase 1 Safety, Pharmacokinetics And Pharmacodynamics Study Of Recombinant Factor VIIa Variant (813d) In Adult Subjects With Hemophilia
Scientific title
An Ascending Single Dose Study To Evaluate The Safety, Tolerability And Pharmacokinetics/Pharmacodynamics Of Pf-05280602, A Recombinant Factor Viia Variant (813d), In Adult Hemophilia A And B Subjects With Or Without Inhibitors
Secondary ID [1] 0 0
2011-002170-23
Secondary ID [2] 0 0
B3051001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - PF-05280602
Treatment: Other - PF-05280602
Treatment: Other - PF-05280602
Treatment: Other - PF-05280602
Treatment: Other - PF-05280602

Experimental: 1 -

Experimental: 2 -

Experimental: 3 -

Experimental: 4 -

Experimental: 5 -


Treatment: Other: PF-05280602
0.5 micrograms per kilogram of PF-05280602, IV infusion, single dose

Treatment: Other: PF-05280602
4.5 micrograms per kilogram of PF-05280602, IV infusion, single dose

Treatment: Other: PF-05280602
9.0 micrograms per kilogram of PF-05280602, IV infusion, single dose

Treatment: Other: PF-05280602
18.0 micrograms per kilogram of PF-05280602, IV infusion, single dose

Treatment: Other: PF-05280602
30.0 micrograms per kilogram of PF-05280602, IV infusion, single dose
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Systolic Blood Pressure (SBP) and Diastolic Blood Pressure (DBP)
Timepoint [1] 0 0
Baseline, Day 2, Day 3, and Day 15
Primary outcome [2] 0 0
Change From Baseline in Body Weight
Timepoint [2] 0 0
Baseline, Day 2, Day 3, and Day 15
Primary outcome [3] 0 0
Change From Baseline in Body Temperature
Timepoint [3] 0 0
Baseline, Day 2, Day 3, and Day 15
Primary outcome [4] 0 0
Change From Baseline in Respiration Rate
Timepoint [4] 0 0
Baseline, Day 2, Day 3, and Day 15
Primary outcome [5] 0 0
Change From Baseline in Supine Pulse Rate
Timepoint [5] 0 0
Baseline, Day 2, Day 3, and Day 15
Primary outcome [6] 0 0
Number of Participants With Changes Since Previous Physical Examination
Timepoint [6] 0 0
Baseline (Day 0), Day 1, Day 2, Day 3, Day 15
Primary outcome [7] 0 0
Number of Participants With Potentially Clinically Significant Electrocardiogram (ECG) Findings
Timepoint [7] 0 0
Baseline through Day 15
Primary outcome [8] 0 0
Number of Participants With Treatment-Emergent Adverse Events (AEs), Serious Adverse Events (SAEs), and Withdrawals Due to AEs (Except Hemophilia AEs)
Timepoint [8] 0 0
Baseline through Day 60
Primary outcome [9] 0 0
Number of Participants With Treatment-Emergent Hemophilia AEs and Withdrawals Due to Hemophilia AEs
Timepoint [9] 0 0
Baseline through Day 60
Primary outcome [10] 0 0
Number of Treatment-Emergent AEs and SAEs by Severity (Except Hemophilia AEs)
Timepoint [10] 0 0
Baseline through Day 60
Primary outcome [11] 0 0
Number of Treatment-Emergent Hemophilia AEs by Severity
Timepoint [11] 0 0
Baseline through Day 60
Primary outcome [12] 0 0
Number of Participants With Treatment-Emergent Abnormal Troponin-T Levels by Magnitude
Timepoint [12] 0 0
Baseline through Day 15
Primary outcome [13] 0 0
Number of Participants With Treatment-Emergent Abnormal Anti-Thrombin III (ATIII) Levels by Magnitude
Timepoint [13] 0 0
Baseline through Day 3
Primary outcome [14] 0 0
Number of Participants With Treatment-Emergent Abnormal Tissue Factor Pathway Inhibitor (TFPI) Levels by Magnitude
Timepoint [14] 0 0
Baseline through Day 3
Primary outcome [15] 0 0
Number of Participants With Treatment-Emergent Laboratory Test Abnormalities (Normal Baseline)
Timepoint [15] 0 0
Baseline through Day 15
Primary outcome [16] 0 0
Number of Participants With Clinically Significant Laboratory Abnormalities Meeting Stopping Criteria
Timepoint [16] 0 0
Baseline through Day 15
Primary outcome [17] 0 0
Number of Participants With Positive Immune Response (Anti-Drug Antibodies [ADA], PF-05280602 Inhibitor, Factor VIIa Inhibitor, Factor VII Inhibitor, and Depletion of Factor VII Activity)
Timepoint [17] 0 0
Baseline through Day 60
Secondary outcome [1] 0 0
Maximum Observed Plasma Concentration (Cmax)
Timepoint [1] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [2] 0 0
Area Under the Curve From Time Zero to Last Quantifiable Concentration (AUClast)
Timepoint [2] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [3] 0 0
Terminal Elimination Half-Life (t1/2)
Timepoint [3] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [4] 0 0
Incremental Recovery (IncRec)
Timepoint [4] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [5] 0 0
Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf)
Timepoint [5] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [6] 0 0
Mean Residence Time (MRT)
Timepoint [6] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [7] 0 0
Volume of Distribution at Steady State (Vss)
Timepoint [7] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [8] 0 0
Clearance (CL)
Timepoint [8] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [9] 0 0
Time to Reach Maximum Observed Plasma Concentration (Tmax)
Timepoint [9] 0 0
Day 1 (pre-dose and 5 min, 10 min, 20 min, 30 min, 1 hr, 3 hr, 6 hr, 9 hr, and 12 hrs post-dose), Day 2 (24 hrs post-dose), Day 3 (48 hrs post-dose)
Secondary outcome [10] 0 0
Maximum Mean Decrease From Baseline in Prothrombin Time (PT)
Timepoint [10] 0 0
Baseline through Day 15
Secondary outcome [11] 0 0
Maximum Mean Decrease From Baseline in Activated Partial Thromboplastin Time (aPTT)
Timepoint [11] 0 0
Baseline through Day 15
Secondary outcome [12] 0 0
Maximum Mean Increase From Baseline in Thrombin Anti-Thrombin (TAT) Complexes
Timepoint [12] 0 0
Baseline through Day 3
Secondary outcome [13] 0 0
Maximum Mean Increase From Baseline in Prothrombin Fragments 1+2
Timepoint [13] 0 0
Baseline through Day 3
Secondary outcome [14] 0 0
Maximum Mean Increase From Baseline in D-Dimers
Timepoint [14] 0 0
Baseline through Day 15
Secondary outcome [15] 0 0
Maximum Mean Increase From Baseline in Endogenous Thrombin Potential (ETP)
Timepoint [15] 0 0
Baseline through Day 3
Secondary outcome [16] 0 0
Maximum Mean Decrease From Baseline in Thrombin Generation Lag Time
Timepoint [16] 0 0
Baseline through Day 3
Secondary outcome [17] 0 0
Maximum Mean Increase From Baseline in Peak Thrombin Generation
Timepoint [17] 0 0
Baseline through Day 3

Eligibility
Key inclusion criteria
* Male subjects 18 to <65 years old with severe hemophilia A or B with or without inhibitors to FVIII or FIX.
* Subjects is willing and able to comply with the mandatory washout periods prior to screening and prior to dosing and through 48 hours post dosing. At screening this includes a washout of FIX for 96 hours and FVIII for 72 houts. At dosing this includes a washout of FIX for 96 hours and FVIII and other hemostatic agents for 72 hours through 48 hours post dosing.
* Subjects must agree and commit to using a a highly effective method of birth control from the time of screening through four weeks after study drug administration.
Minimum age
18 Years
Maximum age
64 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Presence of a bleeding disorder in addition to hemophilia A or B.
* Regular, concomitant therapy with immunomodulating drugs (eg, intravenous immunoglobulin, and routine systemic corticosteroids).
* History of coronary artery disease, thrombolic disease or diagnosis of prothrombic disorder.

Study design
Purpose of the study
Other
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/12/2011
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
1/10/2015
Sample size
Target
Accrual to date
Final
29
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Connecticut
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Oregon
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
Belgium
State/province [7] 0 0
Bruxelles
Country [8] 0 0
Hungary
State/province [8] 0 0
Budapest
Country [9] 0 0
Italy
State/province [9] 0 0
Castelfranco Veneto (TV)
Country [10] 0 0
Italy
State/province [10] 0 0
Castelfranco Veneto - Treviso
Country [11] 0 0
Italy
State/province [11] 0 0
Milano
Country [12] 0 0
Italy
State/province [12] 0 0
Vicenza
Country [13] 0 0
New Zealand
State/province [13] 0 0
Christchurch
Country [14] 0 0
South Africa
State/province [14] 0 0
Eastern Cape
Country [15] 0 0
Turkey
State/province [15] 0 0
Izmir
Country [16] 0 0
United Kingdom
State/province [16] 0 0
London
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Manchester

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Catalyst Biosciences
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT01439971