Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products

April 22, 2026 updated by: CSL Behring

A Phase 3, Open-label, Multicenter, Pharmacokinetics, Efficacy, and Safety Study of a Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Previously Treated Patients (PTPs) With Hemophilia A

For bridging the available global clinical data of rVIII-SingleChain, with the Chinese population, the aim of this study in China is to investigate the pharmacokinetics (PK) of rVIII-SingleChain after an initial and repeat dose and to assess efficacy and safety during 2 to 3 times weekly prophylaxis treatment with rVIII-SingleChain in male Chinese PTPs with severe hemophilia A (FVIII activity less than [<] 1%).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
60

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • China
    • Anhui
      • Hefei, Anhui, China, 230000
        • The Second Hospital of Anhui Medical University
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100045
        • Beijing Children's Hospital
    • Chongqing Municipality
      • Chongqing, Chongqing Municipality, China, 400014
        • Children's Hospital of Chongqing Medical University
    • Fujian
      • Fuzhou, Fujian, China, 350001
        • Fujian Medical University Union Hospital
    • Guangdong
      • Guangzhou, Guangdong, China, 510623
        • Guangzhou Women And Children's Medical Center
      • Guangzhou, Guangdong, China, 515399
        • Nanfang Hospital of Southern Medical University
    • Guangxi
      • Liuchow, Guangxi, China, 545006
        • Liuzhou People's Hospital
    • Hebei
      • Tangshan, Hebei, China, 63000
        • North China University of Science and Technology Affiliated Hospital
      • Tianjin, Hebei, China, 300020
        • Hospital of Hematology, Chinese Academy of Medical Sciences
    • Henan
      • Zhengzhou, Henan, China, 450053
        • Henan Children's Hospital Zhengzhou Children's Hospital
    • Hunan
      • Changsha, Hunan, China, 410021
        • Hunan Provincial Children's Hospital
    • Jiangsu
      • Nanjing, Jiangsu, China, 210008
        • Nanjing Children's Hospital
      • Xuzhou, Jiangsu, China, 221006
        • Xuzhou Children's Hospital
    • Jiangxi
      • Nanchang, Jiangxi, China, 212028
        • Jiangxi Provincial People's Hospital
    • Qinghai
      • Xining, Qinghai, China, 81000
        • Quinghai Provincial People's Hospital
    • Shandong
      • Jinan, Shandong, China, 250013
        • Jinan Central Hospital
      • Qingdao, Shandong, China, 266071
        • Qingdao Women and Children's Hospital
    • Shanghai Municipality
      • Shanghai, Shanghai Municipality, China, 200020
        • Shanghai Jiaotong University School of Medicine, Ruijin Hospital
    • Yunan
      • Kunming, Yunan, China, 650011
        • The Second Affiliated Hospital of Kunming Medical University
    • Zhejiang
      • Hangzhou, Zhejiang, China, 310005
        • The Children's Hospital Zhejiang University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • • Male Chinese participants <= 65 years of age.
  • • Participants with severe hemophilia A (FVIII activity < 1%).
  • • Participants who have received FVIII products for >= 150 EDs (>= 6 years of age) or >= 50 EDs (< 6 years of age).

Exclusion Criteria:

  • • Known hypersensitivity (allergic reaction or anaphylaxis) to any FVIII product or hamster protein.
  • • Known congenital or acquired coagulation disorder other than congenital FVIII deficiency.
  • • Currently receiving intravenous (IV) immunomodulating agents such as immunoglobulin or chronic systemic corticosteroid treatment.
  • • Receiving any cryoprecipitate, whole blood, or plasma within 30 days before administration of rVIII-SingleChain.
  • • Use of traditional or herbal Chinese medicine(s) with an impact on hemophilia, including coagulation, within 28 days before Day 1 and / or refusal to abstain from these during the study until the end of the participant's participation in the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: rVIII-SingleChain
Participants will receive rVIII-SingleChain as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs).
Biological: Recombinant single-chain factor VIII (rVIII-SingleChain)
Lyophilized powder for solution for intravenous injection
Other Names:
  • CSL627

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Incremental Recovery (IR) of rVIII-SingleChain
Time Frame: Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1
Maximum Concentration (Cmax) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Half-life (t1/2) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Clearance (Cl) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Annualized Spontaneous Bleeding Rate (AsBR)
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
AsBR for treated bleeding episodes
Up to 29 weeks after rVIII-SingleChain administration
Number of participants who develop FVIII inhibitors
Time Frame: During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.
During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Time to reach maximum concentration (Tmax) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Last Concentration (Clast) of rVIII-SingleChain
Time Frame: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
Last observed quantifiable plasma concentration
Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1
IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
AUC0-last of rVIII-SingleChain (Repeat PK)
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
AUC0-inf of rVIII-SingleChain (Repeat PK)
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)
Time Frame: Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28
Annualized Bleeding Rate (ABR)
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
Total ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous, traumatic, and unknown cause)
Up to 29 weeks after rVIII-SingleChain administration
Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
The investigator will rate the efficacy of the rVIII-SingleChain treatment for major and nonmajor bleeding episodes based on a hemostatic efficacy four point rating scale of "excellent, good, moderate or no efficacy".
Up to 29 weeks after rVIII-SingleChain administration
Consumption of rVIII-SingleChain - number of infusions (doses)
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
Up to 29 weeks after rVIII-SingleChain administration
Consumption of rVIII-SingleChain - IU/kg per participant per month
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
rVIII-SingleChain consumption (IU/kg) per participant per month, for the weekly regimen, on-demand treatment, and total treatment.
Up to 29 weeks after rVIII-SingleChain administration
Consumption of rVIII-SingleChain - IU/kg per participant per year
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
rVIII-SingleChain consumption (IU/kg) per participant per year, for the weekly regimen, on-demand treatment, and total treatment.
Up to 29 weeks after rVIII-SingleChain administration
Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
Number of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis
Up to 29 weeks after rVIII-SingleChain administration
Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis
Time Frame: Up to 29 weeks after rVIII-SingleChain administration
Percentage of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis
Up to 29 weeks after rVIII-SingleChain administration
Number of participants who develop noninhibitory antibodies against rVIII-SingleChain
Time Frame: Before, and up to 29 weeks after, rVIII-SingleChain administration
Before, and up to 29 weeks after, rVIII-SingleChain administration
Number of participants who develop antibodies against Chinese hamster ovary host cell protein
Time Frame: Before, and up to 29 weeks after, rVIII-SingleChain administration
Before, and up to 29 weeks after, rVIII-SingleChain administration
Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)
Time Frame: Up to 33 weeks after rVIII-SingleChain administration
Up to 33 weeks after rVIII-SingleChain administration
Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs)
Time Frame: Up to 33 weeks after rVIII-SingleChain administration
Up to 33 weeks after rVIII-SingleChain administration
Number of TEAEs (events)
Time Frame: Up to 33 weeks after rVIII-SingleChain administration
Up to 33 weeks after rVIII-SingleChain administration

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
CSL Behring

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Study Director, CSL Behring

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
January 9, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
October 26, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 26, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 12, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 12, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
December 17, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 23, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 22, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • CSL627_3003
  • 2023-001026-34 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

IPD Sharing Time Frame

Requests for IPD will generally be considered once review by major regulatory authorities (i.e., FDA, EMA) is complete and the primary publication is available.

IPD Sharing Access Criteria

Proposed research should seek to answer a previously unanswered important medical or scientific question.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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