Hemophilia APhase 4NCT06940830
Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa
A Phase 4 study testing Efanesoctocog alfa in people with Hemophilia A, run by Swedish Orphan Biovitrum at 31 sites in 8 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Efanesoctocog alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 252 participants
- Where
- 31 sitesin 8 countries
- Study dates
- 21 May 2025to Dec 2028
- Phase
- Phase 4
- Are there results?
- Not yet: the study is under way
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
31 sites in 8 countries.
31 sites in 8 countries
France7 sites
Sobi Investigational Site
Bordeaux
Sobi Investigational Site
Marseille
Sobi Investigational Site
Montpellier
Sobi Investigational Site
Nantes
Sobi Investigational Site
Paris
Sobi Investigational Site
Rennes
Sobi Investigational Site
Rouen
Spain6 sites
Sobi Investigational Site
A Coruña
Sobi Investigational Site
Barcelona
Sobi Investigational Site
Málaga
Sobi Investigational Site
Palma de Mallorca
Sobi Investigational Site
Salamanca, Castille and León
Sobi Investigational Site
Zaragoza
Germany5 sites
Sobi Investigational Site
Berlin
Sobi Investigational Site
Bonn
Sobi Investigational Site
Frankfurt am Main
Sobi Investigational Site
Hamburg
Sobi Investigational Site
Munich
Italy4 sites
Sobi Investigational Site
Catania
Sobi Investigational Site
Padua
Sobi Investigational Site
Salerno
Sobi Investigational Site
Turin
United Kingdom4 sites
Sobi Investigational Site
Birmingham
Sobi Investigational Site
Cardiff
Sobi Investigational Site
London
Sobi Investigational Site
Manchester
Czechia3 sites
Sobi Investigational Site
Brno
Sobi Investigational Site
Ostrava
Sobi Investigational Site
Prague
About this study
Official title: 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)
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.
Read the detailed description
This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities [mild to severe], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII [FVIII]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment.
The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment.
The target is to enroll approximately 250 participants.
- Conditions
- Hemophilia A
- Being tested
- Efanesoctocog alfa
- Keywords
- Hemophilia A, Joint Health, Prophylaxis, Factor VIII Deficiency, Blood coagulation disorder, Efanesoctogoc alfa, Coagulation protein disorder, ALTUVOCT, ALTUVIIIO
- Medical subject headings
- Hemophilia A, Blood Coagulation Disorders, Coagulation Protein Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efanesoctocog alfaDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| Study Treatment Group | Other | Not described Receives: Drug: Efanesoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Annualized joint bleeding rate (AjBR) over the prospective observation period.
Measured: Enrolment to Month 12, 24 and 36
Secondary outcomes (27)
Annualized bleeding rate (ABR) over the prospective observation period.
Measured: Enrolment to Month 12, 24 and 36
Number of injections to control a bleeding event over the prospective observation period.
Measured: Enrolment to Month 12, 24 and 36
Total dose to control bleeding event over the prospective observation period.
Cumulative amount of medication administered to effectively manage bleeding episodes.
Measured: Enrolment to Month 12, 24 and 36
Haemophilia Joint Health Score (HJHS) version 2.1
Change from enrolment in total and domain scores of HJHS per patient.
Measured: Enrolment to Month 12, 24 and 36
Target joint development on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Measured: Month 12, 24 and 36
Target joint resolution on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Measured: Month 12, 24 and 36
Target joint recurrency on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Measured: Month 12, 24 and 36
Annualised injection frequency over the prospective observation period.
Measured: Month 12, 24 and 36
Annualised efanesoctocog alfa consumption for prophylaxis and treatment of bleeds (excluding surgery) over the prospective observation period.
Measured: Month 12, 24 and 36
Reason(s) for any changes in prescribed frequency and/or dose of efanesoctocog alfa prophylaxis over the prospective observation period.
Measured: Enrolment to Month 12, 24 and 36
Change in ABR in the period of using efanesoctocog alfa vs prior haemophilia treatment.
Measured: Prospective period vs Retrospective period
Change in annualised injection frequency during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Measured: Prospective period vs Retrospective period
Change in annualised consumption during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Measured: Prospective period vs Retrospective period
Reason(s) for Switching to efanesoctocog alfa treatment.
Measured: Retrospective period (12 to 18 months prior to enrollment)
Investigator or Surgeon assessment of patient's haemostatic response on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response for surgical procedures scale (Intraoperative and postoperative).
Measured: Within 96 hours following surgery
Number of injections and dose administered to maintain haemostasis during perioperative period for minor and major surgery.
Measured: Enrolment to 36 Months
Total efanesoctocog alfa consumption during perioperative period for minor/major surgery.
Measured: Enrolment to 36 Months
Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
Change from enrolment in total and domain score.
Measured: Enrolment to Month 12, 24 and 36
Treatment preference survey
Measured: Month 12
Patient Reported Outcome (PRO) of Pain Intensity
Use of PROMIS SF v2.0 to measure change in patient-reported pain intensity from enrolment.
Measured: Enrolment to Month 12, 24 and 36
Patient Reported Outcome (PRO) of Pain Interference
Use of PROMIS SF v2.0 to measure change in patient-reported pain interference from enrolment.
Measured: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of 5-level EuroQol-5 dimensions [EQ-5D-5L] score
Changes from enrolment of patient mobility, self-care, usual activities, pain/discomfort, anxiety/depression.
Measured: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of youth EuroQol 5-dimensions [EQ-5D-Y] score
Changes from enrolment of patient mobility, self-care, usual activities, pain/discomfort, anxiety/depression using child-appropriate wording.
Measured: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of Parent Proxy EuroQol 5-dimensions [EQ-5D-Y:1] score
Parent or caregiver's rating of the child's health outcomes based on mobility, self-care, usual activities, pain/discomfort, anxiety/depression.
Measured: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of ACTIVLIM-Hemo.
Measurement of activity limitations for patients with haemophilia (PwH).
Measured: Enrolment to Month 12, 24 and 36
Number of participants with occurrence of Serious Adverse Events (SAEs).
Measured: Enrolment to 36 months
Number of participants with occurrence of Adverse Events of Special Interest (AESIs).
Measured: Enrolment to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Apr 2025
- Study start21 May 2025
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 252 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Responsible party
- Sponsor
- Investigators
- Study Physician, Study director, Swedish Orphan Biovitrum AB (publ)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06940830
- Sponsor's protocol ID
- Sobi.BIVV001-005
- Other IDs
- 2024-517812-31-00, U1111-1312-9668
- Status last verified
- Sep 2026
- First submitted
- 16 Apr 2025
- Last updated
- 30 Sep 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 30 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT06940830
- Acronym / sponsor ID
- Sobi.BIVV001-005
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Joint Health, Prophylaxis, Factor VIII Deficiency, Blood coagulation disorder, Efanesoctogoc alfa, Coagulation protein disorder, ALTUVOCT
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 252 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Study start
- 21 May 2025 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 23 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities [mild to severe], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII [FVIII]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment.
The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment.
The target is to enroll approximately 250 participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Study Treatment Group | Other | Drug: Efanesoctocog alfa |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Efanesoctocog alfaParticipant 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. | Drug | Study Treatment Group |
Primary outcome
Annualized joint bleeding rate (AjBR) over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
27 secondary outcomes
Annualized bleeding rate (ABR) over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Number of injections to control a bleeding event over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Total dose to control bleeding event over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Haemophilia Joint Health Score (HJHS) version 2.1
Time frame: Enrolment to Month 12, 24 and 36
Target joint development on a patient and joint level over the prospective observation period.
Time frame: Month 12, 24 and 36
Target joint resolution on a patient and joint level over the prospective observation period.
Time frame: Month 12, 24 and 36
Target joint recurrency on a patient and joint level over the prospective observation period.
Time frame: Month 12, 24 and 36
Annualised injection frequency over the prospective observation period.
Time frame: Month 12, 24 and 36
Annualised efanesoctocog alfa consumption for prophylaxis and treatment of bleeds (excluding surgery) over the prospective observation period.
Time frame: Month 12, 24 and 36
Reason(s) for any changes in prescribed frequency and/or dose of efanesoctocog alfa prophylaxis over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Change in ABR in the period of using efanesoctocog alfa vs prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Change in annualised injection frequency during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Change in annualised consumption during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Reason(s) for Switching to efanesoctocog alfa treatment.
Time frame: Retrospective period (12 to 18 months prior to enrollment)
Investigator or Surgeon assessment of patient's haemostatic response on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response for surgical procedures scale (Intraoperative and postoperative).
Time frame: Within 96 hours following surgery
Number of injections and dose administered to maintain haemostasis during perioperative period for minor and major surgery.
Time frame: Enrolment to 36 Months
Total efanesoctocog alfa consumption during perioperative period for minor/major surgery.
Time frame: Enrolment to 36 Months
Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
Time frame: Enrolment to Month 12, 24 and 36
Treatment preference survey
Time frame: Month 12
Patient Reported Outcome (PRO) of Pain Intensity
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome (PRO) of Pain Interference
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of 5-level EuroQol-5 dimensions [EQ-5D-5L] score
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of youth EuroQol 5-dimensions [EQ-5D-Y] score
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of Parent Proxy EuroQol 5-dimensions [EQ-5D-Y:1] score
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of ACTIVLIM-Hemo.
Time frame: Enrolment to Month 12, 24 and 36
Number of participants with occurrence of Serious Adverse Events (SAEs).
Time frame: Enrolment to 36 months
Number of participants with occurrence of Adverse Events of Special Interest (AESIs).
Time frame: Enrolment to 36 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Sep 2026.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Physician study director
Swedish Orphan Biovitrum AB (publ)
31 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Efanesoctocog alfa in people with Hemophilia A, run by Swedish Orphan Biovitrum at 31 sites in 8 countries.
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 8 inclusion and 4 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: under way, not looking for participants, as of the sponsor's last update on 30 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in France, Spain, Germany, Italy, United Kingdom and 3 more countries. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Annualized joint bleeding rate (AjBR) over the prospective observation period. (time frame: Enrolment to Month 12, 24 and 36). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Sep 2026.