An observational study in Hemophilia A Patient, sponsored by Nantes University Hospital. Not yet recruiting at 8 sites in France. Per ClinicalTrials.gov, last updated 2026-09-25.
Sponsored by Nantes University Hospital · Observational
Among 157 studies in Hemophilia A with a site in France — 20 open to participants now, 33 under way, 15 of them phase 3. Hemophilia A · France.
Among the 157 studies in Hemophilia A with a site in France, this one is the 9th largest by planned enrollment of the 62 observational ones that give a count, is one of 2 that started in 2027, is one of 62 observational ones, reaches primary completion later than 25 of the 32 other live ones, and was the 156th registered.
844 studies on the registry are indexed under Hemophilia A; 136 are open to participants now.
Browse Hemophilia A studies →20 of the 157 studies in Hemophilia A with a site in France are open now. Recruiting first, the newest first:
Novo Nordisk A/S · Sites in France, United States, Argentina and 15 more countries
Ages 18+
Assistance Publique Hopitaux De Marseille · Sites in France
Ages 20–29
Hoffmann-La Roche · Sites in France, United States, Argentina and 15 more countries
Ages 12+
Hoffmann-La Roche · Sites in France, United States, Argentina and 19 more countries
Ages 12+
Octapharma · Sites in France, United States, Finland and 7 more countries
Ages 12+ · Women only
A study can be open while its sites in France have not opened yet or have stopped. Only those recruiting at a site in France →
Of the 86 Hemophilia A studies recruiting now, 51 (59%) set no upper age limit and 30 (35%) stop at 75 or younger; 54 take children under 18; 10 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
157 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 10 · Phase 2 15 · Phase 3 53 · Phase 4 12
First in 2001; this study’s year is marked. 2 started in 2027.
By drug class, each counting the studies that test any of its medicines:
This study's planned enrollment of 553 is above the median of 81 across 310 observational studies indexed under Hemophilia A.
Median enrollment 74 across the 95 interventional studies, 125 across the 62 observational. Of the 115 that completed or were terminated, 47 (41%) have results posted.
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107 of 157 have an industry lead sponsor. The 10 most frequent:
Nantes University Hospital is the lead sponsor of 839 studies on the registry; 195 are open to participants now.
Nantes University Hospital has 6 studies filed under Hemophilia A — fewer than ten, so there is no comparison to draw.
9 of the studies in Hemophilia A with a site in France that are under way reach primary completion within a year, 4 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04644575Phase 3Bioverativ, a Sanofi companyLong-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A | 2027-01-15 |
| NCT06716814Phase 3Swedish Orphan BiovitrumStudy to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa | 2027-03 |
| NCT05662319Phase 3SanofiA Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia | 2027-03-25 |
| NCT07416526Phase 3Hoffmann-La RocheA Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A | 2027-09-23 |
| NCT04472169University Hospital, LilleUPLC-MS/MS Monitoring of Emicizumab Therapy | 2026-11 |
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Among the 157 studies in Hemophilia A with a site in France, from their registry updates:
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Prophylactic management of severe Hemophilia A (HA) without inhibitors has historically relied on frequent intravenous infusions of standard or EHL FVIII concentrates or on subcutaneous emicizumab administered weekly to monthly intervals , yet both approaches maintain a notable treatment burden and variable factor utilization.
Efanesoctocog alfa (ALTUVOCT®, Swedish Orphan Biovitrum SOBI) was engineered as a fusion of FVIII, von Willebrand factor (VWF) binding domains, and XTEN polypeptides to decouple FVIII from endogenous VWF and extend its circulatory half-life beyond that of existing EHL concentrates.
In the pivotal XTEND-1 trial (NCT04161495) of 159 patients aged ≥ 12 years, once-weekly prophylaxis (50 IU/kg) yielded a mean ABR of 0.70 episodes per patient-year versus approximately 3.0 episodes on prior regimens. The study ended with superior bleeding protection, with FVIII activity above 40 IU/dL for the majority of each week and of 15 IU/dL at day 7. In children under 12, XTEND-Kids phase 3 data (n = 74) demonstrated comparable bleeding control; the average ABR for patients per year was 0.70. The XTEND-ed long-term extension (3-year data) assessed maintaining low ABRs, stable FVIII levels, and consistent tolerability. Efanesoctocog alfa was also well-tolerated in all trials, with adverse events similar to those of other FVIII products and no inhibitor development reported as a result of treatment. The analysis of pharmacokinetics indicated an extended half-life that supports once-weekly dosing and maintains FVIII activity in the normal to near-normal range (> 40 IU/dL) for the majority of each dosing interval.
While regulatory and health-technology assessments recognize its favorable pharmacokinetics and hemostatic efficacy, they underscore the non-randomized, intra-patient design of these studies, the paucity of robust head-to-head comparisons with standard FVIII or emicizumab, and the absence of real-world QoL and consumption data. Despite the promising results, critical questions remain unanswered in routine clinical practice: efanesoctocog alfa is designed for once-weekly dosing but how does switching to efanesoctocog alfa alter real-world FVIII consumption, what clinical or psychosocial factors drive clinicians and patients to transition, and how do patients perceive efficacy, convenience, and treatment burden post-switch?
This study is designed to bridge this knowledge gap by quantifying pre- and post-switch FVIII utilization, systematically capturing switch-motivations through patient interviews, and applying validated patient-reported outcome measures to assess satisfaction and QoL impacts. Addressing these dimensions will not only inform personalized prophylaxis regimens and clarify resource utilization but also enrich pharmaco-economic models and guide future therapeutic innovations in HA management.
Individual profit - The switched patient must benefit from a lower injection frequency for the same clinical outcome (change of prescription); this could also favorably impact his quality of life. The possibility of changing the replacement therapy with FVIII or emicizumab to efanesoctocog alfa will be proposed to the patient during a routine consultation.
The Patient-Reported Outcome Measures (PROMs) will make it possible to properly appraise the patient's view. QoL questionnaires (PERQOLATEUR), Hemophilia Functional Ability Scoring Tool questionnaires (Hemo-FAST©) and treatment evaluation questionnaires will be presented (See Appendice 8), particularly during the shared decision-making consultation on the switch and the following routine consultation.
Group profit - This study will allow a better understanding of the use of health resources (quantification of FVIII consumption before and after switching to efanesoctocog alfa), to better understand the motivations of a person with HA to change treatment, and to evaluate in a real situation the impact on the patient's QoL of a new substitutive treatment.
The research has no risks and constraints because only questionnaires (part of routine care or without health data) are distributed to patients.
The study population targets people with severe hemophilia A or moderate hemophilia A (FVIII:C \< 3 IU/dL), without inhibitors / with history of inhibitors, of any sex, any age. These subjects must be followed in one centre of the BERHLINGO network. The patients will be included in the flow, with routine consultations.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Patients who won't switch to efanesoctocog alfa.
Other: Study questionnaires · Other: Clinical/pharmacological data collection
Patients treated with FVIII who will be switched to efanesoctocog alfa
Other: Study questionnaires · Other: Clinical/pharmacological data collection
Patients treated with FVIII mimetic agents who will be switched to efanesoctocog alfa
Other: Study questionnaires · Other: Clinical/pharmacological data collection
The PERQOLATEUR project aims to develop a standardized yet personalized questionnaire to assess patients' perceived quality of life in relation to their condition and the treatments received. Patients are first asked to select the themes that matter most to them, and then to evaluate their well-being in relation to these themes, as well as the impact of their health problems and treatments on them. The questionnaire is particularly relevant for patients with chronic conditions, but well-being can be assessed in any individual (Co-development of the questionnaire with patients and healthcare professionals + Psychometric validation based on COSMIN recommendations + Item analysis using the Rasch model). The Hemo-FAST© questionnaire is a validated tool offering a quick and easy assessment of joint health and functionality by capturing both the adult patient and clinician perspectives, through the use of patient-reported-outcome and of physician-reported-outcome, with a score of 0 indic
Collection of data from the patient's medical file on clinical evaluation and the patient's treatment history.
Description and comparison of the Hemophilia Functional Ability Scoring Tool questionnaires (Hemo-FAST©) before/after marketing authorization of ALTUVOCT® (efanesoctocog alfa) in adult people with severe or moderate hemophilia A (with FVIII:C < 3 IU/dL)
The Hemo-FAST© questionnaire is a validated tool offering a quick and easy assessment of joint health and functionality by capturing both the adult patient and clinician perspectives, through the use of patient-reported-outcome and of physician-reported-outcome, with a score of 0 indicating the best possible joint health status and a score of 100 as the worst.
Time frame: 2 years
Plan to share: Undecided
No publications or documents are linked to this record.
Start is estimated for Jan 2027; primary completion for Dec 2028, 23 months later.
Of 78 non-industry observational studies under Hemophilia A, half reached primary completion within 36 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 96. This study's planned 23 months is below that.
Counting every sponsor, half of 131 studies reached it within 45 months; a quarter within 25 and three quarters within 79.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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