Long-term study evaluating joint health in people with haemophilia A receiving real-world prophylactic treatment with efanesoctocog alfa

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Swedish Orphan Biovitrum AB (publ)

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

21 May 2025 → ongoing

Decision date (initial)

2025-08-29

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Swedish Orphan Biovitrum AB (Publ) (Sobi)

External identifiers

EU CT number

2024-517812-31-00

WHO UTN

U1111-1312-9668

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy, Prophylaxis

To describe long-term effectiveness of efanesoctocog alfa prophylaxis in preventing joint bleeds in patients with Haemophilia A.

Secondary objectives 7

1. To describe effectiveness of efanesoctocog alfa prophylaxis on other outcomes such as annualized bleeding rate, number of injections used to control a bleeding event, change in HJHS score and target joint development
2. To describe the real-world usage of efanesoctocog alfa treatment
3. To assess the relative effectiveness and real-world usage of efanesoctocog alfa treatment to that of prior haemophilia treatment
4. To describe the effectiveness and usage of efanesoctocog alfa for perioperative management
5. To evaluate joint health structural outcomes under efanesoctocog alfa prophylaxis via ultrasound using Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
6. To describe treatment preference and health-related outcomes in terms of quality of life, pain, and physical activity in patients during real-world prophylactic treatment with efanesoctocog alfa
7. To describe long-term safety of efanesoctocog alfa prophylaxis in a real-world setting

Conditions and MedDRA coding

Haemophilia A

Study design 2 periods

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Male or female patient with a diagnosis of haemophilia A.
2. 2. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
3. 3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.
4. 4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
5. 5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.
6. 6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
7. 7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
8. 8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).

Exclusion criteria 4

1. 1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A
2. 2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit [BU]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.
3. 3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.
4. 4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Annualised joint bleeding rate (AjBR) over the prospective period

Secondary endpoints 4

1. 1. Annual Bleeding Rate over the prospective observation period
2. 2. Number of injections and total dose to control a bleeding event over the prospective observation period
3. 3. Change in HJHS from enrolment to Months 12, 24 and 36
4. 4. Target joint development, resolution, and recurrency on a patient and joint level during the period on efanesoctocog alfa

Investigational products

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

Test 6

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Swedish Orphan Biovitrum AB (publ)

Sponsor organisation

Swedish Orphan Biovitrum AB (publ)

Address

-

City

Stockholm

Postcode

112 76

Country

Sweden

Scientific contact point

Organisation

Swedish Orphan Biovitrum AB (publ)

Contact name

Contact point

Public contact point

Organisation

Swedish Orphan Biovitrum AB (publ)

Contact name

Contact point

Third parties 2

Locations

8 EU/EEA countries · 32 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

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

Documents 92 files

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

Application history

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