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

Hemophilia A Clinical Trial

Official title:

A Non-randomized, Open-label Study To Evaluate The Pharmacokinetics, Safety And Efficacy Of Refacto Af In Previously Treated Pediatric Subjects Less Than Twelve Years Of Age With Severe Hemophilia A (Fviii:c <1%).

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00914459
• Other study ID #: 3082B2-4433
• Secondary ID: B18310052008-008
• Status: Active, not recruiting
• Phase: Phase 4
• First received: June 4, 2009
• Last updated: February 11, 2016
• Start date: December 2009
• Est. completion date: June 2016

Study information

• Verified date: February 2016
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Health authority: European Union: European Medicines Agency
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 50
• Est. completion date: June 2016
• Est. primary completion date: May 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 11 Years

Eligibility

Inclusion Criteria:

• 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).

Exclusion Criteria:

• 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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Biological:
• Moroctocog alfa ( AF-CC)
Dosing is at the discretion of the Investigator

Procedure:
• Laboratory tests
Factor VIII PK samples, Hematology, Chemistry and Coagulation testing, FactorVIII Inhibitor and Anti Factor VIII antibody

Locations

Country	Name	City	State
Finland	Kuopio University Hospital, Department of Pediatrics	Kuopio
Georgia	LTD Medinvesti- Institute of hematology and transfusiology	Tbilisi
Italy	Centro di Riferimento Regionale per la cura dell'Emofilia e delle Malattie Emorragiche Congenite	Parma
Romania	Sanador	Bucharest
Romania	Spitalul Clinic Judetean de Urgenta Craiova	Craiova	Dolj
Serbia	Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"	Belgrade
Serbia	University Children's Hospital	Belgrade
Spain	Hospital Universitario Puerta del Mar	Cadiz
Spain	Hospital Jerez de la Frontera Servicio de Hematologia	Jerez de la Frontera	Cádiz
Spain	Hospital La Paz	Madrid
Spain	Hospital Universitario Miguel Servet	Zaragoza
Sweden	Karolinska Universitetssjukhuset-Solna	Stockholm
Turkey	Cukurova University Department of Pediatrics, Pediatric Hematology Division	Adana	Balcali/Adana
Turkey	Akdeniz Universitesi Tip Fakultesi	Antalya	Kampus
Turkey	Ege University Department of Pediatrics, Pediatric Hematology Division	Bornova/Izmir
Ukraine	Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii medych	Lviv
Ukraine	Komunalna ustanova "Zaporizka oblasna klinichna dytiacha likarnia"	Zaporizhzhia

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

Finland,  Georgia,  Italy,  Romania,  Serbia,  Spain,  Sweden,  Turkey,  Ukraine, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of clinically significant FVIII inhibitor development.		2 years	Yes
Secondary	Annualized bleeding rates (ABRs) in subjects receiving treatment with ReFacto AF.		2 years	No
Secondary	Responses to the first on-demand treatment with ReFacto AF for all new bleeds (4 point scale of assessment) as assessed by the parent/legal representative.		2 years	No
Secondary	Number of ReFacto AF infusions to treat each new bleed.		2 years	No
Secondary	Number of bleeds within 48 hours of a preventive/prophylaxis dose of ReFacto AF.		2 years	No
Secondary	Average infusion dose and total factor consumption, and the incidence of less-than-expected therapeutic effect (LETE).		2 years	No
Secondary	Incidence of SAEs and nonserious AEs.		2 years	Yes

External Links

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