Efanesoctocog alfa treatment in patients with congenital hemophilia A suffering from inflammation of joint lining

Overview

Sponsor-declared trial summary

Key facts

Sponsor

GWT-Tud GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Decision date (initial)

2026-06-08

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

SOBI (Swedish Orphan Biovitrum AB (publ)

External identifiers

EU CT number

2025-523896-44-00

WHO UTN

U1111-1328-6888

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

The primary objective of the study is to assess the efficacy of Efanesoctocog alfa intensified replacement therapy (ERT) to reduce or resolve signs of synovitis in patients with congenital hemophilia A.

Secondary objectives 3

1. Assess pharmacokinetics of ERT
2. Assess the efficacy of ERT to prevent bleeding
3. Assess safety of ERT

Conditions and MedDRA coding

Congenital hemophilia A (HA)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

1. Written informed consent must be obtained before any study-specific tests or procedures are performed
2. Male patients aged between 18 and 70 years at the first screening visit
3. Patients must be capable of giving informed consent and have the ability to understand and follow study-related instructions
4. Patients with severe to moderate congenital hemophilia A
5. Regular prophylaxis with licensed FVIII products (SHL-FVIII, EHL-FVIII or HS-FVIII) at its recommended regimen during the last 6 months
6. Synovitis of elbow, knee, and/or ankle joint (score ≥ 1 for synovial hypertrophy in HEAD-US) confirmed by Blinded ultrasound examination (BLUE) at the time of screening

Exclusion criteria 15

1. Acute hemarthrosis at time of screening (clinical or ultrasound detected) or within the past 4 weeks before screening (clinical)
2. Participation in another clinical interventional trial in the 3 months before screening
3. Criteria which in the opinion of the investigator preclude participation for scientific reasons, for reasons of compliance, or for reasons of the patient’s safety
4. Patient is in custody by virtue of an order issued either by the judicial or the administrative authorities
5. Current factor VIII inhibitor ≥0.6 BU/mL
6. Current immune tolerance therapy
7. Planned intensification of FVIII prophylaxis above the allowed doses specified in the protocol (Table 1, section 5.1)
8. Radiosynovectomy (RSO) or orthopedic surgery during the past 3 months or planned within the next 12 months
9. History of thrombosis, myocardial infarction, other clinically relevant vascular diseases, or atrial fibrillation, or combination of risk factors that would significantly increase the cardiovascular risk during ERT or standard of care (SOC)
10. Use of anticoagulant or antiplatelet drugs at the time of screening
11. Known bleeding disorder other than hemophilia A
12. Life expectancy <12 months at the time of screening
13. Hypersensitivity to the active substance or to any of the excipients of the IMP
14. Patients of Asian ethnicity
15. Close affiliation with the investigator (e.g. a close relative) or persons working at the study site, the sponsor or involved CRO

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Proportion of patients with treatment response (TR) after 12 months. TR is defined as a reduction or resolution of synovial hypertrophy in at least one affected joint according to the synovial hypertrophy domain of the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) score performed by blinded ultrasound expert (BLUE) team

Secondary endpoints 3

1. related to pharmacokinetics and efficacy for bleed prevention of ERT: ■ FVIII trough level ■ Time spent at FVIII activity >30 IU/dL ■ Time spent at FVIII activity >50 IU/dL ■ Annualized bleeding rate (ABR, all treated bleeds) and annualized joint bleeding rate (AjBR, treated joint bleeds only)
2. related to assessments and clinical relevance of synovitis: ■ Proportion of joints with TR ■ Proportion of patients with complete resolution of synovitis in all of their joints
3. related to safety of efanesoctocog alfa: ■ Treatment-emergent adverse events (TEAE)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 5

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

GWT-Tud GmbH

Sponsor organisation

GWT-Tud GmbH

Address

Freiberger Strasse 33, Wilsdruffer Vorstadt/seevorstadt-West Wilsdruffer Vorstadt/seevorstadt-West

City

Dresden

Postcode

01067

Country

Germany

Scientific contact point

Organisation

GWT-Tud GmbH

Contact name

FB Medizin

Public contact point

Organisation

GWT-Tud GmbH

Contact name

FB Medizin

Third parties 3

Locations

2 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications