A study of the safety and effectiveness of a new treatment from human blood source that replaces the missing clotting factor VIII, including determination of drug level in hemophilia A patients.

A Phase I/III Open-label, Multicenter, Crossover Safety, Efficacy and Pharmacokinetic Study of Recombinant Coagulation Factor VIII (rFVIII) Compared to Recombinant Factor VIII (rFVIII; INN: octocog alfa) in Subjects with Hemophilia A, and a Repeat PK, Safety and Efficacy Study

Status

Active, not recruiting

Phases

Unknown

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-002393-23-DE

Enrollment

104

Registered

2011-07-28

Start date

2012-01-10

Completion date

Unknown

Last updated

2015-11-16

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A. MedDRA version: 17.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850

Interventions

Product Name: Recombinant Factor VIII (CSL627) 250 IU Product Code: CSL627 Pharmaceutical Form: Powder and solvent for solution for injection/infusion INN or Proposed INN: Lonoctocog alfa CAS Number:

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Diagnosis of severe hemophilia A defined as 150 EDs with a FVIII product - Written informed consent for study participation obtained before undergoing any study specific procedures. Are the trial subjects under 18? yes Number of subjects for this age range: 18 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 77 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 5

Exclusion criteria

Exclusion criteria: - Any history of or current FVIII inhibitors - Any first order family history of FVIII inhibitors - Use of an Investigational Medicinal Product within 30 days prior to the first CSL627 administration - Not capable of receiving treatment at home - Administration of any cryoprecipitate, whole blood or plasma within 30 days prior to administration of CSL627 or reference product. - Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein. - Any known congenital or acquired coagulation disorder other than congenital FVIII deficiency. - Platelet count 5 times (x) the upper limit of normal (ULN) at Screening. - Subjects with serum creatinine values > 2 x ULN at Screening. - Evidence of thrombosis, including deep vein thrombosis, stroke, pulmonary embolism, myocardial infarction and arterial embolus within 3 months prior to Day 1. - Experienced life-threatening bleeding episode or had major surgery or an orthopedic surgical procedure during the 3 months prior to Day 1. - Demonstrated inability (language problem or mental condition) or unwillingness to comply with study procedures or history of noncompliance. - Employee at the study site, or spouse/partner or relative of the investigator. - Re-entry of subjects previously enrolled or participating in the current study. - Suspected inability (language problems) or unwillingness to comply with study procedures. - Mental condition rendering the subject (or subject's legally acceptable representative[s]) unable to understand the nature, scope and possible consequences of the study). - Any condition that is likely to interfere with evaluation of the IMP or satisfactory conduct of the study.

Design outcomes

Primary

Measure	Time frame
Main Objective: To characterize the pharmacokinetic (PK) profile of CSL627 To demonstrate efficacy in the prevention and treatment of bleeding events To demonstrate the efficacy of routine prophylaxis treatment over on-demand treatment To demonstrate the efficacy of CSL627 in surgical prophylaxis. To characterize the rate of inhibitor formation.;Secondary Objective: To characterize the safety profile of CSL627. ;Primary end point(s): The rate of treatment success for bleeding episodes defined as a rating of “excellent” or “good” on the investigator’s overall clinical assessment of hemostatic efficacy four-point scale. Annualized spontaneous bleeding rate (AsBR). The rate of inhibitor formation to FVIII evaluated from the time of first dose through the End-of-Study visit. For surgical sub-study, the primary efficacy endpoint: -the rate of treatment success during the peri-operative surgical sub-study defined as an investigator rating of “excellent” or “good” on the four-point efficacy evaluation of surgical treatment scale.;Timepoint(s) of evaluation of this end point: The study design includes a 28 day screening period. Subjects participating in Part 3 will be dosed for at least 50 EDs. Subjects enrolled in Part 3 may continue treatment with CSL627 until an extension study is available, if necessary which may extend treatment for up to 24 months.	—

Measure

Time frame

Main Objective: To characterize the pharmacokinetic (PK) profile of CSL627 To demonstrate efficacy in the prevention and treatment of bleeding events To demonstrate the efficacy of routine prophylaxis treatment over on-demand treatment To demonstrate the efficacy of CSL627 in surgical prophylaxis. To characterize the rate of inhibitor formation.;Secondary Objective: To characterize the safety profile of CSL627. ;Primary end point(s): The rate of treatment success for bleeding episodes defined as a rating of “excellent” or “good” on the investigator’s overall clinical assessment of hemostatic efficacy four-point scale. Annualized spontaneous bleeding rate (AsBR). The rate of inhibitor formation to FVIII evaluated from the time of first dose through the End-of-Study visit. For surgical sub-study, the primary efficacy endpoint: -the rate of treatment success during the peri-operative surgical sub-study defined as an investigator rating of “excellent” or “good” on the four-point efficacy evaluation of surgical treatment scale.;Timepoint(s) of evaluation of this end point: The study design includes a 28 day screening period. Subjects participating in Part 3 will be dosed for at least 50 EDs. Subjects enrolled in Part 3 may continue treatment with CSL627 until an extension study is available, if necessary which may extend treatment for up to 24 months.

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Secondary

Measure	Time frame
Secondary end point(s): PK parameters for the repeat assessment of CSL627 collected during Part 3 of the study including incremental recovery, AUC0–t, AUC0–8 percent of area extrapolated, Cmax, Tmax, elimination constant, half-life (t1/2), AUMC0-8 , mean residence time (MRT), clearance (CL), and volume of distribution at steady-state ( Vss). Annualized bleeding rate (ABR) for total bleeds, traumatic bleeds, and joint bleeds. The proportion of bleeding episodes requiring 1, 2, 3 or > 3 infusions of CSL627 to achieve hemostasis. The safety assessment of CSL627 will include AEs, SAEs, laboratory safety parameters (serum chemistry and hematology), antibodies to CSL627, pre- and post-infusion vital signs, and physical examination. Safety data include: vital signs measurement (sitting blood pressure, heart rate, and temperature), standard safety laboratory data (hematology and chemistry [including liver function tests]), and physical examination. Inhibitors to FVIII and antibodies to CSL627 will be assessed prior to the first exposure to CSL627, 14 days after the first dose of CSL627, and every 4 weeks thereafter. A four day wash out period should occur before inhibitor and antibody assessments. Following the month six evaluation, subjects will return to the clinic for visits every 3 months. The overall assessment of efficacy will include all spontaneous bleeding events (regardless of the treatment regimen), and will include the dose administered, the number of infusions required to achieve hemostasis for a bleeding event and the subject’s and investigator’s assessment of efficacy. ;Timepoint(s) of evaluation of this end point: Blood draws for the PK analysis - Part 3 will be at pre-dose (immediately prior to the initial dose), then at intervals after infusion over 96 hours (10-15 min., 0.5, 1, 3, 6, 9, 24, 28, 32, 48, 72 and 96 hours).	—

Measure

Time frame

Secondary end point(s): PK parameters for the repeat assessment of CSL627 collected during Part 3 of the study including incremental recovery, AUC0–t, AUC0–8 percent of area extrapolated, Cmax, Tmax, elimination constant, half-life (t1/2), AUMC0-8 , mean residence time (MRT), clearance (CL), and volume of distribution at steady-state ( Vss). Annualized bleeding rate (ABR) for total bleeds, traumatic bleeds, and joint bleeds. The proportion of bleeding episodes requiring 1, 2, 3 or > 3 infusions of CSL627 to achieve hemostasis. The safety assessment of CSL627 will include AEs, SAEs, laboratory safety parameters (serum chemistry and hematology), antibodies to CSL627, pre- and post-infusion vital signs, and physical examination. Safety data include: vital signs measurement (sitting blood pressure, heart rate, and temperature), standard safety laboratory data (hematology and chemistry [including liver function tests]), and physical examination. Inhibitors to FVIII and antibodies to CSL627 will be assessed prior to the first exposure to CSL627, 14 days after the first dose of CSL627, and every 4 weeks thereafter. A four day wash out period should occur before inhibitor and antibody assessments. Following the month six evaluation, subjects will return to the clinic for visits every 3 months. The overall assessment of efficacy will include all spontaneous bleeding events (regardless of the treatment regimen), and will include the dose administered, the number of infusions required to achieve hemostasis for a bleeding event and the subject’s and investigator’s assessment of efficacy. ;Timepoint(s) of evaluation of this end point: Blood draws for the PK analysis - Part 3 will be at pre-dose (immediately prior to the initial dose), then at intervals after infusion over 96 hours (10-15 min., 0.5, 1, 3, 6, 9, 24, 28, 32, 48, 72 and 96 hours).

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Countries

Australia, Austria, Canada, Chile, Czech Republic, European Union, Germany, Hungary, Italy, Japan, Korea, Republic of, Lebanon, Malaysia, Netherlands, Philippines, Poland, Russian Federation, South Africa, Spain, Sweden, Switzerland, Ukraine, United Kingdom, United States

Contacts

Public ContactClinical Trial Registr. Coordinator

CSL Behring GmbH

clinicaltrials@cslbehring.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026