An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A

• Register: ClinicalTrials.gov
• Last refreshed on: 14 June 2021
• Main ID: NCT02172950
• Date of registration: 23/06/2014
• Prospective Registration: Yes
• Primary sponsor: CSL Behring
• Public title: An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
• Scientific title: A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII-SingleChain, CSL627) in Subjects With Severe Hemophilia A
• Date of first enrolment: October 13, 2014
• Target sample size: 245
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02172950
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Canada, Czech Republic, Czechia, France, Georgia, Germany, Hungary, Ireland, Italy, Japan, Lebanon, Malaysia, Netherlands, Philippines, Poland, Portugal, Romania, South Africa, Spain, Switzerland, Thailand, Turkey, Ukraine, United Kingdom, United States

Contacts

• Name: Program Director
• Affiliation: CSL Behring

Key inclusion & exclusion criteria

Inclusion Criteria:

PTPs:

- Males of any age who have been diagnosed with severe congenital hemophilia A (FVIII
activity levels < 1%) and who participated in a previous CSL-sponsored clinical study
with rVIII-SingleChain.

- Males 0 to <65 years age who have been diagnosed with severe congenital hemophilia A
(FVIII activity levels < 1%), who have at least 50 EDs to any FVIII product, and who
are not currently enrolled in a CSL-sponsored clinical study with rVIII-SingleChain.

PUPs:

- Males 0 to <18 years of who have been diagnosed with severe congenital hemophilia A
(FVIII activity levels < 1%)

- No prior exposure to any Factor VIII product (with the exception of short-term use of
blood products).

ITI substudy:

- PUPs who have developed a confirmed inhibitor to rVIII-SingleChain in the main study.

Exclusion Criteria:

- Known or suspected hypersensitivity to rVIII-SingleChain or to any excipients of
rVIII-SingleChain or Chinese hamster ovary (CHO) proteins.

- Currently receiving a therapy not permitted during the study.

- Serum creatinine > 2 x upper limit of normal, alanine aminotransferase or aspartate
aminotransferase > 5 x upper limit of normal at Screening (if specified)

- Any first-order family (eg, siblings) history of FVIII inhibitors

- For PTPs not rolling over directly from a CSL-sponsored clinical study with
rVIII-SingleChain: any history of or current FVIII inhibitors



Age minimum: N/A
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Hemophilia A

Severe Hemophilia A

Intervention(s)

Biological: rVIII-SingleChain

Primary Outcome(s)

Treatment success for major bleeding episodes in PUPs [Time Frame: Up to 5 years]

Incidence of high-titer inhibitor formation to FVIII in PUPs with at least 50 EDs of rVIII-SingleChain [Time Frame: At the closest visit after 50 EDs (up to 5 years).]

Incidence of inhibitor formation to FVIII in PTPs with 100 EDs to rVIII-SingleChain [Time Frame: At the closest visit after 100 EDs (up to 5 years).]

Annualized spontaneous bleeding rate in PUPs [Time Frame: Up to 5 years]

Secondary Outcome(s)

Consumption of rVIII-SingleChain in PTPs and PUPs - injections [Time Frame: Up to 5 years]

Incidence of inhibitor formation to FVIII after 10 EDs and after 50 EDs in PTPs [Time Frame: After 10 and after 50 exposure days]

Percentage of PUPs with clinically significant abnormal vital signs values after first rVIII-SingleChain injection [Time Frame: At 1, 2, 3, and 6 hours after first rVIII-SingleChain injection]

Annualized bleeding rate in PTPs and PUPs [Time Frame: Up to 5 years]

Incidence of low-titer inhibitor formation to FVIII in PUPs [Time Frame: At the closest visit after 10 and after 50 EDs (up to 5 years)]

Hemostatic efficacy of rVIII-SingleChain for PTPs and PUPs who undergo surgery [Time Frame: From the start of surgery through the post-operative recovery (generally up to 14 days after surgery)]

Incidence of high-titer inhibitor formation to FVIII in PUPs after 10 EDs with rVIII-SingleChain [Time Frame: At the closest visit after 10 EDs]

Incidence of total inhibitor formation to FVIII in PUPs [Time Frame: At the closest visit after 10 and after 50 EDs (up to 5 years)]

Percentage of PTPs and PUPs developing antibodies to Chinese hamster ovary (CHO) proteins [Time Frame: PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).]

Percentage of PUPs with treatment-emergent clinically significant abnormal vital signs values [Time Frame: Up to 5 years]

Treatment success for non-major bleeding episodes in PUPs [Time Frame: Up to 5 years]

Treatment success in PTPs [Time Frame: Up to 5 years]

Consumption of rVIII-SingleChain in PTPs and PUPs - IU/kg [Time Frame: Up to 5 years]

Percentage of bleeding episodes requiring 1, 2, 3, or > 3 injections of rVIII-SingleChain to achieve hemostasis in PTPs and PUPs [Time Frame: Up to 5 years]

Percentage of PTPs and PUPs developing antibodies against rVIII-SingleChain [Time Frame: PTPs: At the closest visit after 100 EDs (up to 5 years). PUPs: At the closest visit after 50 EDs (up to 5 years).]

Secondary ID(s)

2013-003262-13

CSL627_3001

Source(s) of Monetary Support

Please refer to primary and secondary sponsors