Study Evaluating Safety And Efficacy Of Moroctocog Alfa (AF-CC) In Previously Treated Hemophilia A Patients

NCT00914459 | Completed Phase 4 Results

• 3 other identifiers: 3082B2-4433B18310052008-008435-29
• Study Type: interventional
• Target: 37
• Locations: 8 countries
• Sites: 15

Brief Summary

The study will be investigating pharmacokinetics, safety and efficacy in patients less than 12 years of age with severe hemophilia A that have been previously treated with Factor VIII products ( including blood products).

Conditions

Hemophilia A

Keywords

hemophilia A

Outcome Measures

Primary Outcomes (4)

• Percentage of Participants With Clinically Significant Factor VIII Inhibitor Development

  Clinically significant factor VIII (FVIII) inhibitors were defined as a central laboratory confirmed positive inhibitor of greater than or equal to (\>=) 0.6 Bethesda units (BU) using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6-week interval and one of the following within 4 weeks before the initial or within 4 weeks following the second positive FVIII inhibitor sample collection: 1) the need for the participant to administer alternative hemostatic products in order to achieve sufficient efficacy, 2) \>=2 events indicating a decrease in the efficacy of the study treatment. Percentage of participants who developed clinically significant Factor VIII inhibitor after study drug administration were reported.

  Baseline up to Month 24

• Incremental Recovery

  Incremental recovery was the increase in circulating FVIII activity for every international unit (IU) of ReFacto AF administered per kilogram of body weight. It was measured in international units per deciliter (IU/dL) per international units per kilogram (IU/kg).

  Days 1, 15, 50, Months 6, 18 and Final visit (up to Month 24)

• Terminal Elimination Half Life of ReFacto AF (t1/2)

  T1/2 was the time for the plasma concentration of drug to decrease by one-half of its original concentration.

  Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1

• Clearance (CL)

  Drug clearance is a quantitative measure of the rate at which a drug substance is removed from the blood.

  Pre-dose, 0.5, 1, 3, 6, 9, 24, 28, 32, 48 hours post-dose on Day 1

Secondary Outcomes (16)

• Mean Annualized Bleeding Rates (ABRs): All Participants

  Baseline up to Month 24

• Response to First On-Demand Treatment for New Bleeds: All Participants

  Baseline up to Month 24

• Number of On-Demand ReFacto AF Infusions to Treat a New Bleed: All Participants

  Baseline up to Month 24

• Number of Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of ReFacto AF: All Participants

  Baseline up to Month 24

• Average Infusion Dose of ReFacto AF: All Participants

  Baseline up to Month 24

Study Arms (1)

Moroctocog alfa (AF-CC)

OTHER

Open Label

Biological: Moroctocog alfa ( AF-CC); Procedure: Laboratory tests

Interventions

Moroctocog alfa ( AF-CC) BIOLOGICAL

Dosing is at the discretion of the Investigator

Also known as: ReFacto AF

Moroctocog alfa (AF-CC)

Laboratory tests PROCEDURE

Factor VIII PK samples, Hematology, Chemistry and Coagulation testing, FactorVIII Inhibitor and Anti Factor VIII antibody

Moroctocog alfa (AF-CC)

Eligibility Criteria

• Age: Up to 11 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Male subjects less than 12 years of age with a documented history of severe hemophilia A (FVIII:C less than 1%).
• Subjects who are less than 6 years of age must have had at least 50 Exposure Days (EDs) to prior FVIII products (including blood products).
• Subjects who are equal to or greater than 6 years of age must have had greater than 150 EDs to prior FVIII products (including blood products).

You may not qualify if:

• For laboratory assessments, any measured Bethesda inhibitor titer equal to or greater than 0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer greater than ULN for the testing laboratory at the time of screening.
• Any other bleeding disorder in addition to hemophilia A.
• Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.
• Major surgery planned to occur during the course of the study.
• Regular (e.g., daily; every other day) use of agents or medications known to influence platelet function such as aspirin or certain nonsteroidal anti-inflammatory drugs (NSAIDS).
• Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin \[IVIG\], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
• The subject is receiving treatment for HIV or hepatitis infection (unless the subject is on a stable antiviral regimen \[i.e., consistent treatment regimen for at least 3 months before the parental informed consent/assent form is signed\]).
• Platelet count less than 100,000/µL.
• Prothrombin time (PT) equal to or greater than 1.25 x ULN, or international normalized ratio (INR) equal to or greater than 1.5.
• Known hypersensitivity to hamster protein.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (17)

Kuopio University Hospital

Kuopio, 70211, Finland

Location

LTD Medinvesti- Institute of hematology and transfusiology

Tbilisi, 0186, Georgia

Location

Centro di Riferimento Regionale per la cura dell'Emofilia e delle Malattie Emorragiche Congenite

Parma, 43100, Italy

Location

Spitalul Clinic Judetean de Urgenta Craiova

Craiova, Dolj, 200642, Romania

Location

Sanador

Bucharest, 011026, Romania

Location

University Children's Hospital

Belgrade, 11000, Serbia

Location

Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"

Belgrade, 11070, Serbia

Location

Hospital Jerez de la Frontera

Jerez de la Frontera, Cádiz, 11407, Spain

Location

Hospital Universitario Puerta del Mar

Cadiz, Spain, 11009, Spain

Location

Hospital Universitario Miguel Servet

Zaragoza, Zaragoza, 50009, Spain

Location

Hospital La Paz

Madrid, 28046, Spain

Location

Karolinska Universitetssjukhuset-Solna

Stockholm, 171 76, Sweden

Location

Cukurova University Department of Pediatrics, Pediatric Hematology Division

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

Location

Ege University Department of Pediatrics, Pediatric Hematology Division

Izmir, Bornova /izmir, 35100, Turkey (Türkiye)

Location

Akdeniz Universitesi Tip Fakultesi

Antalya, Kampus, 07059, Turkey (Türkiye)

Location

Komunalna ustanova "Zaporizka oblasna klinichna dytiacha likarnia"

Zaporizhzhia, Ukraine, 69063, Ukraine

Location

Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii medych

Lviv, 79044, Ukraine

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; Factor VIII; Clinical Laboratory Techniques

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

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors; Diagnostic Techniques and Procedures; Diagnosis; Investigative Techniques

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 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2009
• First Posted: June 5, 2009
• Study Start: December 1, 2009
• Primary Completion: March 1, 2016
• Study Completion: April 1, 2016
• Last Updated: February 10, 2017
• Results First Posted: December 21, 2016

Record last verified: 2016-12

Locations

SE (1); TU (3); RO (2); ES (4); IT (1); GE (1); UA (2); FI (1)