Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)
3 other identifiers
interventional
250
8 countries
27
Brief Summary
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_4
Started May 2025
Longer than P75 for phase_4
27 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 16, 2025
CompletedFirst Posted
Study publicly available on registry
April 23, 2025
CompletedStudy Start
First participant enrolled
May 21, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2028
March 24, 2026
March 1, 2026
3.5 years
April 16, 2025
March 20, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Annualized joint bleeding rate (AjBR) over the prospective observation period.
Enrolment to Month 12, 24 and 36
Secondary Outcomes (27)
Annualized bleeding rate (ABR) over the prospective observation period.
Enrolment to Month 12, 24 and 36
Number of injections to control a bleeding event over the prospective observation period.
Enrolment to Month 12, 24 and 36
Total dose to control bleeding event over the prospective observation period.
Enrolment to Month 12, 24 and 36
Haemophilia Joint Health Score (HJHS) version 2.1
Enrolment to Month 12, 24 and 36
Target joint development on a patient and joint level over the prospective observation period.
Month 12, 24 and 36
- +22 more secondary outcomes
Study Arms (1)
Study Treatment Group
OTHERInterventions
Participant in this study must be prescribed efanesoctocog alfa prophylactic treatment per their standard of care from their physician. No drug will be provided by the Sponsor.
Eligibility Criteria
You may qualify if:
- Male or female patient with a diagnosis of haemophilia A.
- 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.
- 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.
- Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
- 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.
- Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
- Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
- For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).
You may not qualify if:
- Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
- 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.
- 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.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Swedish Orphan Biovitrumlead
- PSI CROcollaborator
Study Sites (27)
Sobi Investigational Site
Zagreb, Croatia
Sobi Investigational Site
Brno, Czechia
Sobi Investigational Site
Ostrava, Czechia
Sobi Investigational Site
Prague, Czechia
Sobi Investigational Site
Bordeaux, France
Sobi Investigational Site
Marseille, France
Sobi Investigational Site
Montpellier, France
Sobi Investigational Site
Nantes, France
Sobi Investigational Site
Paris, France
Sobi Investigational Site
Rennes, France
Sobi Investigational Site
Rouen, France
Sobi Investigational Site
Berlin, Germany
Sobi Investigational Site
Bonn, Germany
Sobi Investigational Site
Frankfurt am Main, Germany
Sobi Investigational Site
Hamburg, Germany
Sobi Investigational Site
Munich, Germany
Sobi Investigational Site
Dublin, Ireland
Sobi Investigational Site
Turin, Italy
Sobi Investigational Site
A Coruña, Spain
Sobi Investigational Site
Barcelona, Spain
Sobi Investigational Site
Málaga, Spain
Sobi Investigational Site
Palma de Mallorca, Spain
Sobi Investigational Site
Zaragoza, Spain
Sobi Investigational Site
Birmingham, United Kingdom
Sobi Investigational Site
Cardiff, United Kingdom
Sobi Investigational Site
London, United Kingdom
Sobi Investigational Site
Manchester, United Kingdom
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Physician
Swedish Orphan Biovitrum AB (publ)
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 16, 2025
First Posted
April 23, 2025
Study Start
May 21, 2025
Primary Completion (Estimated)
December 1, 2028
Study Completion (Estimated)
December 1, 2028
Last Updated
March 24, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will share
Qualified researchers may request access to patient level data and related study documents. Patient level data will be anonymized and study documents, if applicable will be redacted to protect the privacy of trial participants.