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%)

• Register: EUCTR
• Last refreshed on: 2 August 2021
• Main ID: EUCTR2008-008435-29-DK
• Date of registration: 25/02/2010
• Prospective Registration: Yes
• Primary sponsor: Wyeth Pharmaceuticals Inc.Acting through its division Wyeth Research, a Pfizer Company
• Public 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%)
• Scientific 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%)
• Date of first enrolment: 30/03/2010
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-008435-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: Open: Single blind: Double blind: Parallel group: Cross over: Other: If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Bulgaria, Czech Republic, Denmark, Finland, France, Greece, Italy, Spain, Sweden

Key inclusion & exclusion criteria

Inclusion criteria:
1) Male subjects <12 years of age with a documented history of severe hemophilia A (FVIII:C <1%).
a) Subjects who are <6 years of age must have had at least 50 exposure days to prior FVIII products (including blood products).
b) Subjects who are =6 years of age must have had greater than 150 exposure days to prior FVIII products (including blood products)



Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1) For laboratory assessments, any measured Bethesda inhibitor titer =0.6 BU, regardless of the laboratory normal range, or any Bethesda inhibitor titer > ULN for the testing laboratory at the time of screening.
2) Any other bleeding disorder in addition to hemophilia A.
3) Treatment with any investigational drug or device within 30 days before the time of signing the parental informed consent/assent form.
4) Major surgery planned to occur during the course of the study.
5) 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).
6) Regular, concomitant therapy with immunomodulating drugs (e.g., intravenous immunoglobulin [IVIG], routine systemic corticosteroids), or currently receiving immune tolerance induction (ITI) for inhibitor treatment.
7) The subject is receiving treatment for HIV or hepatitis infection (exceptions may be made for subjects on a stable antiviral regimen [i.e., consistent treatment regimen for at least 3 months before signing the parental informed consent/assent form]).
8) Platelet count <100,000/µL.
9) Prothrombin time (PT) =1.25 x ULN, or international normalized ratio (INR) =1.5.
10) Known hypersensitivity to hamster protein.
11) Any condition(s) that compromises the subject’s ability to comply with and/or perform study-related activities or that poses a clinical contraindication to study participation (these conditions include, but are not limited to, inadequate medical history available to assure study eligibility; inability to properly store study medication; expectation of poor compliance in study related documentation; poor venous access), in the opinion of the investigator.
12) Unwilling or unable to follow the terms of the protocol.



Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Severe hemophilia A (FVIII:C <1%) in male subjects <12 years of age.
MedDRA version: 9.1 Level: LLT Classification code 10018937 Term: Haemophilia A

Intervention(s)

Trade Name: ReFacto AF
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: MOROCTOCOG-ALFA
CAS Number: 284036-24-4
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 250-

Trade Name: ReFacto AF
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: MOROCTOCOG-ALFA
CAS Number: 284036-24-4
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 500-

Trade Name: ReFacto AF
Pharmaceutical Form: Powder and solvent for solution for injection
INN or Proposed INN: MOROCTOCOG-ALFA
CAS Number: 284036-24-4
Concentration unit: IU international unit(s)
Concentration type: equal
Concentration number: 1000-

Primary Outcome(s)

Primary end point(s): The primary safety outcome is the development of clinically significant FVIII inhibitors. Clinically significant inhibitors are defined as a central laboratory confirmed positive inhibitor (=0.6 BU using the Nijmegen modification of the Bethesda assay present at 2 consecutive blood draws within a 6 week interval) and the occurrence of one or more of the following clinical observations:
decreased FVIII recovery, need for alternative hemostatic products, increase in number of breakthrough bleeds while on preventative or prophylactic treatment, or more than one report of Less Than Expected Therapeutic Effect (LETE) in the absence of confounding factors.

Secondary Objective: To evaluate the efficacy and safety of ReFacto AF in pediatric subjects less than 12 years of age, including the frequency of less than expected therapeutic effect.

Main Objective: To evaluate the pharmacokinetics and incremental recovery of ReFacto AF in pediatic subjects less than 12 years of age after a single exposure to ReFacto AF.

Secondary ID(s)

3082B2-4433-WW

2008-008435-29-FR

Ethics review

Status: Approved
Approval date: 11/03/2010
Contact:

Results

• Results available: Yes
• Date Posted: 02/09/2016
• Date Completed: 05/04/2016
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2008-008435-29/results