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

NCT ID: NCT00543439

Last Updated: 2019-01-11

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

66 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-12-31

Study Completion Date

2018-04-30

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

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

Group Type EXPERIMENTAL

Moroctocog alfa (AF-CC)

Intervention Type BIOLOGICAL

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

2

Routine Prophylaxis Crossover

Group Type EXPERIMENTAL

Moroctocog alfa (AF-CC)

Intervention Type BIOLOGICAL

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.

Interventions

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Moroctocog alfa (AF-CC)

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

Intervention Type BIOLOGICAL

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 Type BIOLOGICAL

Other Intervention Names

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Xyntha Xyntha

Eligibility Criteria

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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.

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.
Minimum Eligible Age

6 Months

Maximum Eligible Age

15 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Pfizer

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Pfizer CT.gov Call Center

Role: STUDY_DIRECTOR

Pfizer

Locations

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Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

OHSU Investigational Pharmacy

Portland, Oregon, United States

Site Status

Oregon Health & Science University

Portland, Oregon, United States

Site Status

Childrens Medical Center Dallas

Dallas, Texas, United States

Site Status

University of Texas Southwestern Medical Center at Dallas

Dallas, Texas, United States

Site Status

University of Utah

Salt Lake City, Utah, United States

Site Status

Primary Children's Hospital

Salt Lake City, Utah, United States

Site Status

Fundacion de la Hemofilia

Buenos Aires, , Argentina

Site Status

Medizinische Universitaet Wien

Vienna, , Austria

Site Status

Liga Colombiana de Hemofílicos y otras Deficiencias Sanguíneas

Bogotá, , Colombia

Site Status

Clinical Hospital Centre Split

Split, , Croatia

Site Status

Jordan University of Science and Technology, King Abdullah University Hospital

Irbid, , Jordan

Site Status

Hospital Civil de Guadalajara Dr. Juan I. Menchaca

Guadalajara, Jalisco, Mexico

Site Status

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

Colonia Centro, Monterrey, Nuevo LEON, Mexico

Site Status

Hospital Universitario "Dr. Jose Eleuterio Gonzalez"

Monterrey, Nuevo León, Mexico

Site Status

Christchurch Hospital

Christchurch, South Island, New Zealand

Site Status

Canterbury District Health Board

Christchurch, , New Zealand

Site Status

Sultan Qaboos University Hospital

Muscat, , Oman

Site Status

Centro Medico Monte Carmelo

Urbanización La Victoria, Arequipa, Peru

Site Status

Samodzielny Publiczny Dzieciecy Szpital Kliniczny

Warsaw, , Poland

Site Status

Sanador

Bucharest, , Romania

Site Status

Cukurova University Tip Fakultesi

Balcali/Adana, Adana, Turkey (Türkiye)

Site Status

Ege Universitesi Tip Fakultesi Cocuk Hastanesi

Izmir, Bornova, Turkey (Türkiye)

Site Status

Istanbul Universtesi Istanbul Tip Fakultesi

Çapa, Istanbul, Turkey (Türkiye)

Site Status

On Dokuz Mayis University Faculty of Medicine

Samsun, Kurupelit, Turkey (Türkiye)

Site Status

Akdeniz Universitesi Tip Fakultesi

Antalya, , Turkey (Türkiye)

Site Status

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

Izmir, , Turkey (Türkiye)

Site Status

Erciyes Universitesi Tip Fakultesi

Kayseri, , Turkey (Türkiye)

Site Status

Countries

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United States Argentina Austria Colombia Croatia Jordan Mexico New Zealand Oman Peru Poland Romania Turkey (Türkiye)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

Other Identifiers

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B1831001

Identifier Type: OTHER

Identifier Source: secondary_id

2006-005575-17

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

3082B2-313

Identifier Type: -

Identifier Source: org_study_id

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