Study Evaluating Prophylaxis Treatment & Characterizing Efficacy, Safety, & PK Of B-Domain Deleted Recombinant FVIII

NCT00543439 | Completed Phase 3 Results DMC FDA Drug

• 3 other identifiers: 3082B2-313B18310012006-005575-17
• Study Type: interventional
• Target: 66
• Locations: 13 countries
• Sites: 28

Brief Summary

The purpose of this research study is to determine the effectiveness, safety, and pharmacokinetics (PK) of moroctocog alfa (AF-CC) in previously treated subjects, who are younger than 6 years of age, with severe or moderately severe hemophilia A.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Mean Annualized Bleed Rate (ABR) by Treatment: On Demand Cohort

  ABR for each participant was calculated as the number of bleeds requiring administration of moroctocog alfa (AF-CC) divided by the total therapy duration (in days), then multiplied by 365.25 (days in a year).

  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 Outcomes (22)

• Mean Annualized Bleed Rate (ABR) by Treatment: Routine Prophylaxis Cohort

  Day 1 up to Month 24 (RP Cohort, Period 1 and Period 2)

• Mean of Moroctocog Alfa (AF-CC) Infusions Administered To Treat Bleeding Episode: All Participants

  Day 1 up to Month 24

• 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)

  Day 1 up to Month 24

• Number of Treated Spontaneous Bleeds by Time Interval Between Bleed Onset and Prior Moroctocog Alfa (AF-CC) Prophylaxis Dose: Routine Prophylaxis Therapy

  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)

• Number of Participants Requiring Prophylaxis Regimen Escalation: Routine Prophylaxis Therapy

  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)

Study Arms (2)

1

EXPERIMENTAL

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

Biological: Moroctocog alfa (AF-CC)

2

EXPERIMENTAL

Routine Prophylaxis Crossover

Biological: Moroctocog alfa (AF-CC)

Interventions

Moroctocog alfa (AF-CC) BIOLOGICAL

On-demand therapy for 6 months, followed by routine prophylaxis 25 IU/kg, administered every other day for 1 year.

Also known as: Xyntha

1

Eligibility Criteria

• Age: 6 Months - 15 Years
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (28)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

OHSU Investigational Pharmacy

Portland, Oregon, 97239, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

Childrens Medical Center Dallas

Dallas, Texas, 75235, United States

Location

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, 75390-9063, United States

Location

University of Utah

Salt Lake City, Utah, 84108, United States

Location

Primary Children's Hospital

Salt Lake City, Utah, 84113, United States

Location

Fundacion de la Hemofilia

Buenos Aires, C1425BWE, Argentina

Location

Medizinische Universitaet Wien

Vienna, 1090, Austria

Location

Liga Colombiana de Hemofílicos y otras Deficiencias Sanguíneas

Bogotá, Colombia

Location

Clinical Hospital Centre Split

Split, 21000, Croatia

Location

Jordan University of Science and Technology, King Abdullah University Hospital

Irbid, 22110, Jordan

Location

Hospital Civil de Guadalajara Dr. Juan I. Menchaca

Guadalajara, Jalisco, 44340, Mexico

Location

Hospital y Clinica OCA S.A. de C.V.

Colonia Centro, Monterrey, Nuevo LEON, 64000, Mexico

Location

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, 64460, Mexico

Location

Christchurch Hospital

Christchurch, South Island, 8001, New Zealand

Location

Canterbury District Health Board

Christchurch, 8001, New Zealand

Location

Sultan Qaboos University Hospital

Muscat, 123, Oman

Location

Centro Medico Monte Carmelo

Urbanización La Victoria, Arequipa, 054, Peru

Location

Samodzielny Publiczny Dzieciecy Szpital Kliniczny

Warsaw, 00-576, Poland

Location

Sanador

Bucharest, 011026, Romania

Location

Cukurova University Tip Fakultesi

Balcali/Adana, Adana, 01330, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi Cocuk Hastanesi

Izmir, Bornova, 35100, Turkey (Türkiye)

Location

Istanbul Universtesi Istanbul Tip Fakultesi

Çapa, Istanbul, 34390, Turkey (Türkiye)

Location

On Dokuz Mayis University Faculty of Medicine

Samsun, Kurupelit, 55139, Turkey (Türkiye)

Location

Akdeniz Universitesi Tip Fakultesi

Antalya, 07059, Turkey (Türkiye)

Location

Dr. Behcet Uz Child and Diseases And Surgery Education and Research Hospital

Izmir, 35210, Turkey (Türkiye)

Location

Erciyes Universitesi Tip Fakultesi

Kayseri, 38039, Turkey (Türkiye)

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia A

Interventions

recombinant factor VIII SQ; F8 protein, human

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov_Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 11, 2007
• First Posted: October 15, 2007
• Study Start: December 1, 2007
• Primary Completion: April 1, 2018
• Study Completion: April 1, 2018
• Last Updated: January 11, 2019
• Results First Posted: January 11, 2019

Record last verified: 2018-12

Locations

PL (1); OM (1); CR (1); AR (1); AU (1); JO (1); CO (1); ME (3); US (7); PE (1); RO (1); TU (7); NZ (2)