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Congenital Hemophilia APhase 3NCT06738485

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

A Phase 3 study testing Recombinant single-chain factor VIII (rVIII-SingleChain) in people with Congenital Hemophilia A, run by CSL Behring at 20 sites in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 0 days to 65 years, men only
Conditions the study is for
Congenital Hemophilia A
What is being tested?
Recombinant single-chain factor VIII (rVIII-SingleChain)
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
62 participants
Where
20 sitesChina
Study dates
9 Jan 2025to 23 Sep 2026
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 0 days to 65 years
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • • 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).

Who cannot join

  • • 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.

Trial locations and contacts

20 sites in 1 country.

20 sites in 1 country

China20 sites

  • Beijing Children's Hospital

    Beijing, Beijing Municipality, 100045

  • Hunan Provincial Children's Hospital

    Changsha, Hunan, 410021

  • Children's Hospital of Chongqing Medical University

    Chongqing, Chongqing Municipality, 400014

  • Fujian Medical University Union Hospital

    Fuzhou, Fujian, 350001

  • Guangzhou Women and Children's Medical Center

    Guangzhou, Guangdong, 510623

  • Nanfang Hospital of Southern Medical University

    Guangzhou, Guangdong, 515399

  • The Children's Hospital Zhejiang University School of Medicine

    Hangzhou, Zhejiang, 310005

  • The Second Hospital of Anhui Medical University

    Hefei, Anhui, 230000

  • Jinan Central Hospital

    Jinan, Shandong, 250013

  • The second affiliated hospital of Kunming Medical University

    Kunming, Yunan, 650011

  • Liuzhou People's Hospital

    Liuzhou, Guangxi, 545006

  • Jiangxi Provincial People's Hospital

    Nanchang, Jiangxi, 212028

  • Nanjing Children's Hospital

    Nanjing, Jiangsu, 210008

  • Qingdao Women and Children's Hospital

    Qingdao, Shandong, 266071

  • Shanghai Jiaotong University School of Medicine, Ruijin Hospital

    Shanghai, Shanghai Municipality, 200020

  • North China University of Science and Technology Affiliated Hospital

    Tangshan, Hebei, 63000

  • Hospital of Hematology, Chinese Academy of Medical Sciences

    Tianjin, Hebei, 300020

  • Quinghai Provincial People's Hospital

    Xining, Qinghai, 81000

  • XuZhou Children's Hospital

    Xuzhou, Jiangsu, 221006

  • Henan children's hospital Zhengzhou children's Hospital

    Zhengzhou, Henan, 450053

Other trials in China

About this study

Official title: 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%).

Being tested
Recombinant single-chain factor VIII (rVIII-SingleChain)
Medical subject headings
Hemophilia A

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Recombinant single-chain factor VIII (rVIII-SingleChain)Biological

    Also called CSL627

    Lyophilized powder for solution for intravenous injection

Study groups in NCT06738485
Study groupRoleWhat they receive
rVIII-SingleChainExperimentalParticipants will receive rVIII-SingleChain as an intravenous (IV) infusion for a minimum of 50 exposure days (EDs).
Receives: Biological: Recombinant single-chain factor VIII (rVIII-SingleChain)

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incremental Recovery (IR) of rVIII-SingleChain

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

  2. Maximum Concentration (Cmax) of rVIII-SingleChain

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  3. Area Under the Plasma Concentration Time Curve from Time Zero to the Last Measurable Concentration (AUC0-last) of rVIII-SingleChain

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  4. Area Under the Plasma Concentration Time Curve from Time Zero to Infinity (AUC0-inf) of rVIII-SingleChain

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  5. Half-life (t1/2) of rVIII-SingleChain

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  6. Clearance (Cl) of rVIII-SingleChain

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  7. Annualized Spontaneous Bleeding Rate (AsBR)

    AsBR for treated bleeding episodes

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  8. Number of participants who develop FVIII inhibitors

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

Secondary outcomes (20)

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

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  2. Last Concentration (Clast) of rVIII-SingleChain

    Last observed quantifiable plasma concentration

    Measured: Before, and up to 72 hours (participants ≥ 12 years ) and 48 hours (participants < 12 years) after rVIII-SingleChain administration on Day 1

  3. IR (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat pharmacokinetic [PK])

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

  4. Cmax (participants ≥ 12 years of age) of rVIII-SingleChain (Repeat PK)

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

  5. AUC0-last of rVIII-SingleChain (Repeat PK)

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

  6. AUC0-inf of rVIII-SingleChain (Repeat PK)

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

  7. t1/2 of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)

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

  8. Cl of rVIII-SingleChain (participants ≥ 12 years of age) (Repeat PK)

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

  9. Annualized Bleeding Rate (ABR)

    Total ABR for treated, untreated, and both treated and untreated bleeding episodes (spontaneous, traumatic, and unknown cause)

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  10. Hemostatic Efficacy for Major and Nonmajor Bleeding Episodes

    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".

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  11. Consumption of rVIII-SingleChain - number of infusions (doses)

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  12. Consumption of rVIII-SingleChain - IU/kg per participant per month

    rVIII-SingleChain consumption (IU/kg) per participant per month, for the weekly regimen, on-demand treatment, and total treatment.

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  13. Consumption of rVIII-SingleChain - IU/kg per participant per year

    rVIII-SingleChain consumption (IU/kg) per participant per year, for the weekly regimen, on-demand treatment, and total treatment.

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  14. Number of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

    Number of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  15. Percentage of bleeding episodes requiring rVIII-SingleChain to achieve hemostasis

    Percentage of bleeding episodes requiring 1, <= 2, or > 2 infusions (doses) of rVIII-SingleChain to achieve hemostasis

    Measured: Up to 29 weeks after rVIII-SingleChain administration

  16. Number of participants who develop noninhibitory antibodies against rVIII-SingleChain

    Measured: Before, and up to 29 weeks after, rVIII-SingleChain administration

  17. Number of participants who develop antibodies against Chinese hamster ovary host cell protein

    Measured: Before, and up to 29 weeks after, rVIII-SingleChain administration

  18. Number of participants with Treatment-emergent Adverse Events (TEAEs), including related TEAEs, and serious adverse events (SAEs)

    Measured: Up to 33 weeks after rVIII-SingleChain administration

  19. Percentage of participants with TEAEs, including related TEAEs, and serious adverse events (SAEs)

    Measured: Up to 33 weeks after rVIII-SingleChain administration

  20. Number of TEAEs (events)

    Measured: Up to 33 weeks after rVIII-SingleChain administration

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted17 Dec 2024
  2. Study start9 Jan 2025
  3. Primary completion23 Sep 2026
  4. Study completion23 Sep 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Prevention
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
62 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06738485
Sponsor's protocol ID
CSL627_3003
Other IDs
2023-001026-34, 2023
Status last verified
Oct 2026
First submitted
12 Dec 2024
Last updated
23 Oct 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: 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.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Recombinant single-chain factor VIII (rVIII-SingleChain) in people with Congenital Hemophilia A, run by CSL Behring at 20 sites in China.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 20 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incremental Recovery (IR) of rVIII-SingleChain (time frame: Before, and at 30 minutes after the end of, rVIII-SingleChain administration on Day 1). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06738485.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.

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