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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00543439




Registration number
NCT00543439
Ethics application status
Date submitted
11/10/2007
Date registered
15/10/2007
Date last updated
11/01/2019

Titles & IDs
Public title
Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII
Scientific title
An Open-label Study To Evaluate Prophylaxis Treatment, And To Characterize The Efficacy, Safety, And Pharmacokinetics Of B-domain Deleted Recombinant Factor Viii Albumin Free (Moroctocog Alfa [Af-cc]) In Children With Hemophilia A
Secondary ID [1] 0 0
B1831001
Secondary ID [2] 0 0
3082B2-313
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 - Moroctocog alfa (AF-CC)
Treatment: Other - Moroctocog alfa (AF-CC)

Experimental: 1 - On-Demand therapy for 6 months, followed by Routine Prophylaxis treatment for 1 year.

Experimental: 2 - Routine Prophylaxis Crossover


Treatment: Other: Moroctocog alfa (AF-CC)
On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.

Treatment: Other: Moroctocog alfa (AF-CC)
Routine prophylaxis crossover:

45 IU/kg, administered 2 times a week for 1 year followed by 25 IU/kg administered every other day for 1 year, or, 25 IU/kg, administered every other day for 1 year, followed by 45 IU/kg, administered 2 times a week for 1 year.

Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort
Timepoint [1] 0 0
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)
Secondary outcome [1] 0 0
Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort
Timepoint [1] 0 0
Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2)
Secondary outcome [2] 0 0
Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants
Timepoint [2] 0 0
Day 1 up to Month 24
Secondary outcome [3] 0 0
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)
Timepoint [3] 0 0
Day 1 up to Month 24
Secondary outcome [4] 0 0
Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy
Timepoint [4] 0 0
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)
Secondary outcome [5] 0 0
Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy
Timepoint [5] 0 0
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)
Secondary outcome [6] 0 0
Mean Routine Prophylaxis Dose (IU/kg) of Moroctocog Alfa (AF-CC) Received: Routine Prophylaxis Therapy
Timepoint [6] 0 0
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)
Secondary outcome [7] 0 0
Mean of Total Number Moroctocog Alfa (AF-CC) Infusions Received: Routine Prophylaxis Therapy
Timepoint [7] 0 0
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)
Secondary outcome [8] 0 0
Mean of Total Number of Days Participants Exposed to Moroctocog Alfa (AF-CC): Routine Prophylaxis Therapy
Timepoint [8] 0 0
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)
Secondary outcome [9] 0 0
Mean of Total Number of Infusions of Moroctocog Alfa (AF-CC) Received Per Week to Assess Compliance: Routine Prophylaxis Therapy
Timepoint [9] 0 0
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)
Secondary outcome [10] 0 0
Terminal Phase Half Life (t1/2) of Factor VIII (FVIII) Activity
Timepoint [10] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [11] 0 0
Clearance (CL) of Factor VIII Activity
Timepoint [11] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [12] 0 0
Incremental Recovery of Factor VIII Activity
Timepoint [12] 0 0
Day 1, Month 6
Secondary outcome [13] 0 0
Maximum Concentration of Factor VIII Activity
Timepoint [13] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [14] 0 0
Area Under the Curve From Time Zero to Extrapolated Infinite Time (AUCinf) of Factor VIII Activity
Timepoint [14] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [15] 0 0
Area Under the Curve From Time Zero to Last Measurable Concentration (AUClast) of Factor VIII Activity
Timepoint [15] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [16] 0 0
Steady-State Volume of Distribution (Vss) of Factor VIII Activity
Timepoint [16] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [17] 0 0
Mean Residence Time (MRT) of Factor VIII Activity
Timepoint [17] 0 0
0.5, 8, 24, 28 and 32 hours post dose on Day 1
Secondary outcome [18] 0 0
Number of Participants With Treatment Emergent Adverse Events (AEs) According to Severity
Timepoint [18] 0 0
Day 1 up to Month 25
Secondary outcome [19] 0 0
Number of Participants With Treatment-Related Adverse Events
Timepoint [19] 0 0
Day 1 up to Month 25
Secondary outcome [20] 0 0
Number of Participants With Confirmed FVIII Inhibitor Development
Timepoint [20] 0 0
Day 1 up to Month 24
Secondary outcome [21] 0 0
Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): On Demand Therapy
Timepoint [21] 0 0
Day 1 up to Month 24 (RP Cohort, RP 25 IU/kg and 45 IU/kg, Period 1 and 2); 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)
Secondary outcome [22] 0 0
Number of Participants With Incidence of Less Than Expected Therapeutic Effect (LETE): Routine Prophylaxis Therapy
Timepoint [22] 0 0
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)

Eligibility
Key inclusion criteria
* Male subjects, aged less than 6 years, with moderately severe to severe hemophilia A.
* A negative FVIII inhibitor titer at screening, and a medical history negative for a past FVIII inhibitor.
* At least 20 exposure days to any FVIII replacement product.
* Adequate hepatic and renal function
* CD4 count > 400 cells/uL, and if receiving antiviral therapy must be on a stable regimen

Additional criteria for subjects participating in the PK assessment:

* Male subjects as described immediately above except they must have a FVIII Activity of less than or equal to 1% confirmed by the central laboratory screening test
* Age < 6 years at time of PK assessment.
* The subject's size is sufficient to permit PK-related phlebotomy.
* The subject is able to comply with the procedures conducted during the PK assessment, including a mandatory 72-hour washout period preceding the PK assessment.
Minimum age
6 Months
Maximum age
15 Years
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* A history of FVIII inhibitor.
* Presence of a bleeding disorder in addition to hemophilia A.
* Treatment with any investigational drug or device within 30 days before the time of signing the informed consent form.
* Major or orthopedic surgery planned to occur during the course of the study.
* Regular (e.g., daily, every other day) use of antifibrinolytic agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDs), or regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids).
* Known hypersensitivity to hamster protein.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Crossover
Other design features
Phase
Phase 3
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
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Ohio
Country [2] 0 0
United States of America
State/province [2] 0 0
Oregon
Country [3] 0 0
United States of America
State/province [3] 0 0
Texas
Country [4] 0 0
United States of America
State/province [4] 0 0
Utah
Country [5] 0 0
Argentina
State/province [5] 0 0
Buenos Aires
Country [6] 0 0
Austria
State/province [6] 0 0
Wien
Country [7] 0 0
Colombia
State/province [7] 0 0
Bogota
Country [8] 0 0
Croatia
State/province [8] 0 0
Split
Country [9] 0 0
Jordan
State/province [9] 0 0
Irbid
Country [10] 0 0
Mexico
State/province [10] 0 0
Jalisco
Country [11] 0 0
Mexico
State/province [11] 0 0
Monterrey, Nuevo LEON
Country [12] 0 0
Mexico
State/province [12] 0 0
Nuevo LEON
Country [13] 0 0
New Zealand
State/province [13] 0 0
South Island
Country [14] 0 0
New Zealand
State/province [14] 0 0
Christchurch
Country [15] 0 0
Oman
State/province [15] 0 0
Muscat
Country [16] 0 0
Peru
State/province [16] 0 0
Arequipa
Country [17] 0 0
Poland
State/province [17] 0 0
Warszawa
Country [18] 0 0
Romania
State/province [18] 0 0
Bucuresti
Country [19] 0 0
Turkey
State/province [19] 0 0
Adana
Country [20] 0 0
Turkey
State/province [20] 0 0
Bornova
Country [21] 0 0
Turkey
State/province [21] 0 0
Istanbul
Country [22] 0 0
Turkey
State/province [22] 0 0
Kurupelit
Country [23] 0 0
Turkey
State/province [23] 0 0
Antalya
Country [24] 0 0
Turkey
State/province [24] 0 0
Izmir
Country [25] 0 0
Turkey
State/province [25] 0 0
Kayseri

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

Results publications and other study-related documents

No documents have been uploaded by study researchers.