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

NCT06738485 | Active Not Recruiting Phase 3

• 2 other identifiers: CSL627_30032023-001026-34
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 20

Brief Summary

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%).

Conditions

Congenital Hemophilia A

Outcome Measures

Primary Outcomes (8)

• Incremental Recovery (IR) of rVIII-SingleChain

  Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1

• Maximum Concentration (Cmax) of rVIII-SingleChain

  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

  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

  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

  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

  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)

  AsBR for treated bleeding episodes

  Up to 29 weeks after rVIII-SingleChain administration

• Number of participants who develop FVIII inhibitors

  During routine rVIII-SingleChain prophylaxis dosing, up to 29 weeks after rVIII-SingleChain administration.

Secondary Outcomes (20)

• Time to reach maximum concentration (Tmax) of rVIII-SingleChain

  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

  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])

  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)

  Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

• AUC0-last of rVIII-SingleChain (Repeat PK)

  Before, and at up to 72 hours after the end of, rVIII-SingleChain infusion at Week 28

Study Arms (1)

rVIII-SingleChain

EXPERIMENTAL

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)

Interventions

Recombinant single-chain factor VIII (rVIII-SingleChain) BIOLOGICAL

Lyophilized powder for solution for intravenous injection

Also known as: CSL627

rVIII-SingleChain

Eligibility Criteria

• Age: 0 Days - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (20)

The Second Hospital of Anhui Medical University

Hefei, Anhui, 230000, China

Location

Beijing Children's Hospital

Beijing, Beijing Municipality, 100045, China

Location

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, 400014, China

Location

Fujian Medical University Union Hospital

Fuzhou, Fujian, 350001, China

Location

Guangzhou Women and Children's Medical Center

Guangzhou, Guangdong, 510623, China

Location

Nanfang Hospital of Southern Medical University

Guangzhou, Guangdong, 515399, China

Location

Liuzhou People's Hospital

Liuchow, Guangxi, 545006, China

Location

North China University of Science and Technology Affiliated Hospital

Tangshan, Hebei, 63000, China

Location

Hospital of Hematology, Chinese Academy of Medical Sciences

Tianjin, Hebei, 300020, China

Location

Henan children's hospital Zhengzhou children's Hospital

Zhengzhou, Henan, 450053, China

Location

Hunan Provincial Children's Hospital

Changsha, Hunan, 410021, China

Location

Nanjing Children's Hospital

Nanjing, Jiangsu, 210008, China

Location

XuZhou Children's Hospital

Xuzhou, Jiangsu, 221006, China

Location

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, 212028, China

Location

Quinghai Provincial People's Hospital

Xining, Qinghai, 81000, China

Location

Jinan Central Hospital

Jinan, Shandong, 250013, China

Location

Qingdao Women and Children's Hospital

Qingdao, Shandong, 266071, China

Location

Shanghai Jiaotong University School of Medicine, Ruijin Hospital

Shanghai, Shanghai Municipality, 200020, China

Location

The second affiliated hospital of Kunming Medical University

Kunming, Yunan, 650011, China

Location

The Children's Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310005, China

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2024
• First Posted: December 17, 2024
• Study Start: January 9, 2025
• Primary Completion (Estimated): October 26, 2026
• Study Completion (Estimated): October 26, 2026
• Last Updated: April 23, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• 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.
• 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.

Locations

CN (20)