Hemophilia A With InhibitorNCT03951103
rFVIIIFc (Elocta®) ITI Chart Review in Patients With Haemophilia A
An observational study of Hemophilia A With Inhibitor, run by Swedish Orphan Biovitrum at 18 sites in 8 countries.
Trial summary
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Hemophilia A With Inhibitor
- What is being tested?
- rFVIIIFc
- How many people
- 44 participants
- Where
- 18 sitesin 8 countries
- Study dates
- 7 Nov 2018to 30 Sep 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
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
Study population: All haemophilia A patients who have been, or who are currently treated with rFVIIIFc for ITI meeting the inclusion and not meeting the exclusion criteria will be invited to participate. Approximately 45 patients from Europe and the Middle East are expected to be included in the study.
Who may be able to join
- Patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI.
- Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related data collection are undertaken. Assent should be obtained from paediatric patients according to local regulations.
Who cannot join
- Current participation in any investigational medicinal product trial.
Trial locations and contacts
18 sites in 8 countries.
18 sites in 8 countries
Germany4 sites
Swedish Orphan Biovitrum Research Site
Frankfurt
Swedish Orphan Biovitrum Research Site
Friedrichshain
Swedish Orphan Biovitrum Research Site
Hanover
Swedish Orphan Biovitrum Research Site
Mitte
Italy4 sites
Swedish Orphan Biovitrum Research Site
Catania
Swedish Orphan Biovitrum Research Site
Catanzaro
Swedish Orphan Biovitrum Research Site
Genova
Swedish Orphan Biovitrum Research Site
Naples
France3 sites
Swedish Orphan Biovitrum Research Site
Paris
Swedish Orphan Biovitrum Research Site
Rennes
Swedish Orphan Biovitrum Research Site
Tours
Saudi Arabia3 sites
Swedish Orphan Biovitrum Research Site
Riyadh, 12233
Swedish Orphan Biovitrum Research Site (a)
Riyadh, 12713
Swedish Orphan Biovitrum Research Site (p)
Riyadh, 12713
About this study
Official title: A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
Read the detailed description
A multicenter, international, non- interventional, retrospective and prospective medical chart review study. Data will be collected from medical records for patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI . The study will be descriptive in nature and report on baseline characteristics, treatment and outcomes for patients who have been, or who are currently, treated with rFVIIIFc for ITI.
- Conditions
- Hemophilia A With Inhibitor
- Being tested
- rFVIIIFc
- Keywords
- Elocta, rFVIIIFc
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rFVIIIFcOther
Also called Elocta
Drug according to prescription
| Study group | Role | What they receive |
|---|---|---|
| Hemophili A patients | Patients treated with rFVIIIFc for ITI Receives: Other: rFVIIIFc |
What is being measured
The primary outcome is the main question the study is designed to answer.
ITI with rFVIIIFc: Main dose
Main dose will be assessed on the prescribed dose (IU/kg)
Measured: From 2018 to 2022
ITI with rFVIIIFc: Main injection frequency
Main injection frequency will be assessed on the prescribed frequency
Measured: From 2018 to 2022
ITI with rFVIIIFc: Duration
Number of treatment months
Measured: From 2018 to 2022
ITI with rFVIIIFc: Concomitant by-passing agents
Product name and main dose will be used to describe any concomitant use of by-passing agents.
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Overall outcome
The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer
Treatment time to reach undetectable inhibitor levels (<0.6 BU/ml)
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to normal recovery
Treatment time to reach normal recovery levels (≥66% of the expected value)
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Time to success
Treatment time to reach success (see outcome #5)
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Inhibitor titer levels
BU/ml
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Half-life
FVIII half-life (hours)
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Recovery level
FVIII recovery level (%)
Measured: From 2018 to 2022
Outcome of ITI with rFVIIIFc: Bleeds
Number of bleeds per month during ITI-treatment
Measured: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
Occurrence of relapse (Yes/No) will be assessed by the investigator.
Measured: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Time to relapse
Time to occurrence of relapse (see outcome #13)
Measured: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Treatment regimen
Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.
Measured: From 2018 to 2022
Long-term outcome after ITI with rFVIIIFc: Bleeds
Number of bleeds per month.
Measured: From 2018 to 2022
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 May 2019
- Study start7 Nov 2018
- Primary completion30 Sep 2022
- Study completion30 Sep 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hemophilia A With InhibitorStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stefan Lethagen, Study director, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03951103
- Sponsor's protocol ID
- Sobi.Elocta-004
- Status last verified
- Sep 2024
- First submitted
- 19 Sep 2018
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Klamroth R, Al Saleh M, Glosli H, Schiavulli M, Guillet B, Bystricka L, Schonstein A, Lethagen S. Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study. Eur J Haematol. 2025 Aug;115(2):134-141. doi: 10.1111/ejh.14427. Epub 2025 Apr 27.
PubMed 40289300
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Chart Review Study of Patients With Haemophilia A With Inhibitors Treated With rFVIIIFc (Elocta®) for Immune Tolerance Induction
- ClinicalTrials.gov ID
- NCT03951103
- Acronym / sponsor ID
- Sobi.Elocta-004
- Conditions
- Hemophilia A With Inhibitor
- Keywords
- Elocta, rFVIIIFc
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Study start
- 7 Nov 2018 (actual)
- Primary completion
- 30 Sep 2022 (actual)
- Study completion
- 30 Sep 2022 (actual)
- First posted
- 15 May 2019 (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.
A chart review study of patients with haemophilia A with inhibitors treated with rFVIIIFc (Elocta®) for immune tolerance induction.
Detailed description
A multicenter, international, non- interventional, retrospective and prospective medical chart review study. Data will be collected from medical records for patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI . The study will be descriptive in nature and report on baseline characteristics, treatment and outcomes for patients who have been, or who are currently, treated with rFVIIIFc for ITI.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophili A patients | – | Other: rFVIIIFcPatients treated with rFVIIIFc for ITI |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- All haemophilia A patients who have been, or who are currently treated with rFVIIIFc for ITI meeting the inclusion and not meeting the exclusion criteria will be invited to participate. Approximately 45 patients from Europe and the Middle East are expected to be included in the study.
Key inclusion criteria (2 listed)
- Patients diagnosed with haemophilia A who have been, or who are currently, treated with rFVIIIFc for ITI.
- Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related data collection are undertaken. Assent should be obtained from paediatric patients according to local regulations.
Key exclusion criteria (1 listed)
- Current participation in any investigational medicinal product trial.
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 |
|---|---|---|
| rFVIIIFcAlso: EloctaDrug according to prescription | Other | Hemophili A patients |
Primary outcome
ITI with rFVIIIFc: Main dose
Time frame: From 2018 to 2022
Main dose will be assessed on the prescribed dose (IU/kg)
ITI with rFVIIIFc: Main injection frequency
Time frame: From 2018 to 2022
Main injection frequency will be assessed on the prescribed frequency
ITI with rFVIIIFc: Duration
Time frame: From 2018 to 2022
Number of treatment months
ITI with rFVIIIFc: Concomitant by-passing agents
Time frame: From 2018 to 2022
Product name and main dose will be used to describe any concomitant use of by-passing agents.
Outcome of ITI with rFVIIIFc: Overall outcome
Time frame: From 2018 to 2022
The Investigator will assess overall outcome as: success, partial success, failure, early withdrawal or other.
Outcome of ITI with rFVIIIFc: Time to undetectable inhibitor titer
Time frame: From 2018 to 2022
Treatment time to reach undetectable inhibitor levels (<0.6 BU/ml)
Outcome of ITI with rFVIIIFc: Time to normal recovery
Time frame: From 2018 to 2022
Treatment time to reach normal recovery levels (≥66% of the expected value)
Outcome of ITI with rFVIIIFc: Time to success
Time frame: From 2018 to 2022
Treatment time to reach success (see outcome #5)
Outcome of ITI with rFVIIIFc: Inhibitor titer levels
Time frame: From 2018 to 2022
BU/ml
Outcome of ITI with rFVIIIFc: Half-life
Time frame: From 2018 to 2022
FVIII half-life (hours)
Outcome of ITI with rFVIIIFc: Recovery level
Time frame: From 2018 to 2022
FVIII recovery level (%)
Outcome of ITI with rFVIIIFc: Bleeds
Time frame: From 2018 to 2022
Number of bleeds per month during ITI-treatment
Long-term outcome after ITI with rFVIIIFc: Occurrence of relapse
Time frame: From 2018 to 2022
Occurrence of relapse (Yes/No) will be assessed by the investigator.
Long-term outcome after ITI with rFVIIIFc: Time to relapse
Time frame: From 2018 to 2022
Time to occurrence of relapse (see outcome #13)
Long-term outcome after ITI with rFVIIIFc: Treatment regimen
Time frame: From 2018 to 2022
Treatment regimen will be described as: ITI, prophylaxis or on-demand; and also by product used.
Long-term outcome after ITI with rFVIIIFc: Bleeds
Time frame: From 2018 to 2022
Number of bleeds per month.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Derived from this study: PubMed 40289300Klamroth R, Al Saleh M, Glosli H, Schiavulli M, Guillet B, Bystricka L, Schonstein A, Lethagen S. Immune Tolerance Induction With a Recombinant Factor VIII Fc in Haemophilia A: Data From a Chart Review Study. Eur J Haematol. 2025 Aug;115(2):134-141. doi: 10.1111/ejh.14427. Epub 2025 Apr 27.
- Individual participant dataSharing planned: No
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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.
Stefan Lethagen study director
Swedish Orphan Biovitrum
18 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A With Inhibitor, run by Swedish Orphan Biovitrum at 18 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 2 inclusion and 1 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: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Germany, Italy, France, Saudi Arabia, Ireland 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
ITI with rFVIIIFc: Main dose (time frame: From 2018 to 2022). The study lists 16 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03951103, which also lists 1 linked publication.
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 19 Sep 2024.