Hemophilia ACLHEARNCT04583930
Clinical and Health-related Outcome of rFVIIIFc Prophylaxis
An observational study of Hemophilia A, run by Prof. Dr. Dr. Thomas Hilberg at 2 sites in Germany.
Trial summary
Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 48 participants
- Where
- 2 sitesGermany
- Study dates
- 1 Feb 2021to 20 Apr 2023
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Patients with moderate or severe Haemophilia A will be included in this study consecutively, who will be switched from FVIII prophylaxis on rFVIIIFc prophylaxis.
Who may be able to join
- Patients suffering from moderate to severe haemophilia A
- Age ≥ 18-years
- Treatment with FVIII prophylaxis
- Submitted written informed consent
Who cannot join
- Patients suffering from other bleeding diseases
- Patients with inhibitors
- Patients without written informed consent
- Age < 18-year-old
- Any surgeries up to 6 months before the examination date
- Suffering from different rheumatologic diseases like M. Bechterew, Psoriasis or other local or generalized joint infections (Borreliosis, septic arthritis)
Trial locations and contacts
2 sites in 1 country.
Germany2 sites, 2 recruiting
- Recruiting
Department of Sports Medicine, University of Wuppertal
Wuppertal, North Rhine-Westphalia, 42117
Thomas Hilberg, Prof., 004920237320812, sportmedizin@uni-wuppertal.de
- Recruiting
Department of Sports Medicine
Wuppertal, Northwest, 42117
Thomas Hilberg, Prof., 004920237320812, hilberg@uni-wuppertal.de
Jamil Hmida, 0049202439, jamil.hmida@uni-wuppertal.de
About this study
Official title: Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A
Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. With the development of recombinant factor VIII Fc fusion protein (rFVIIIFc) the conventional routine prophylaxis regime is complemented by an extended half-life (EHL) factor replacement prophylaxis with the potential of improved bleed prevention and reduced injection frequency at similar factor consumption. Aim of this longitudinal multicentre study is to evaluate the influence of an EHL factor replacement regime with rFVIIIFc on haemophilic specific parameters (annual bleeding rate, bleeding localisation), joint status, pain, functional parameters, treatment adherence and health-related quality of life in PwH A.
- Conditions
- Hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Patients with Haemophilia A | * Patients suffering from moderate to severe haemophilia A * Age ≥ 18-years * Treatment with FVIII prophylaxis * Submitted written informed consent |
What is being measured
The primary outcome is the main question the study is designed to answer.
total annual bleeding rate
number of total bleedings
Measured: one year
orthopaedic joint status (Haemophilia health joint score)
The clinical joint status will be examined in all patients with haemophilia by the World Federation Joint Examination Score and Haemophilia Joint Health Score. Higher score points imply an increased deficit in the functional and structural joint status as a sign of more pronounced haemophilic arthropathy, with a maximum possible value of 124 (no deficits = 0).
Measured: one year
pressure pain thresholds
physiological Parameter in Newton
Measured: one year
Secondary outcomes (4)
spontaneous joint ABR
number of total joint bleedings
Measured: one year
subjective quality of life (SF-36)
The standarized questionnaire SF-36 includes a physical and psychological score. The higher the score, the higher the physical or psychological quality of life. A higher score implies a better physical and mental quality of life
Measured: two years
subjective physical performance (HEP-Test-Q)
The final version of the standarized questionnaire consists of 25 items pertaining to the domains 'mobility', 'strength \& coordination', 'endurance' and 'body perception'. The response options were a five-point Likert scale (ranging from 1 = never to 5 = always). Some of the items had to be re-coded; subscales and the total score were transformed to a scale of 0-100 with high scores indicating better physical performance
Measured: two years
self-perceived functional abilities (Haemophilia Activities List)
The Haemophilia Activities List measures the impact of hemophilia on self-perceived functional abilities in adults. It contains 42 multiple choice questions in seven domains: Lying/sitting/kneeling/standing (8 items), Functions of the legs (9 items), Functions of the arms (4 items), Use of transportation (3 items), Self-care (5 items), Household tasks (6 items), Leisure activities and sports (7 items)
Measured: two years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Oct 2020
- Study start1 Feb 2021
- Primary completion31 Mar 2023 (estimated)
- Study completion20 Apr 2023 (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.
- 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
- Prospective
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Prof. Dr. Dr. Thomas Hilberg
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Prof. Dr. Dr. Thomas Hilberg, University of Wuppertal
- Investigators
- Thomas Hilberg, Prof., Principal investigator, Head of Department
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04583930
- Sponsor's protocol ID
- Hae-Ger-2019-251-Hil
- Acronym
- CLHEAR
- Status last verified
- May 2021
- First submitted
- 28 Jul 2020
- Last updated
- 11 May 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT04583930
- Acronym / sponsor ID
- CLHEAR / Hae-Ger-2019-251-Hil
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- Prof. Dr. Dr. Thomas HilbergUniversity, hospital or other
- Study start
- 1 Feb 2021 (actual)
- Primary completion
- 31 Mar 2023 (estimated)
- Study completion
- 20 Apr 2023 (estimated)
- First posted
- 12 Oct 2020 (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.
Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. With the development of recombinant factor VIII Fc fusion protein (rFVIIIFc) the conventional routine prophylaxis regime is complemented by an extended half-life (EHL) factor replacement prophylaxis with the potential of improved bleed prevention and reduced injection frequency at similar factor consumption. Aim of this longitudinal multicentre study is to evaluate the influence of an EHL factor replacement regime with rFVIIIFc on haemophilic specific parameters (annual bleeding rate, bleeding localisation), joint status, pain, functional parameters, treatment adherence and health-related quality of life in PwH A.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with Haemophilia A | – | –* Patients suffering from moderate to severe haemophilia A * Age ≥ 18-years * Treatment with FVIII prophylaxis * Submitted written informed consent |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients with moderate or severe Haemophilia A will be included in this study consecutively, who will be switched from FVIII prophylaxis on rFVIIIFc prophylaxis.
Key inclusion criteria (4 listed)
- Patients suffering from moderate to severe haemophilia A
- Age ≥ 18-years
- Treatment with FVIII prophylaxis
- Submitted written informed consent
Key exclusion criteria (6 listed)
- Patients suffering from other bleeding diseases
- Patients with inhibitors
- Patients without written informed consent
- Age < 18-year-old
- Any surgeries up to 6 months before the examination date
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
total annual bleeding rate
Time frame: one year
number of total bleedings
orthopaedic joint status (Haemophilia health joint score)
Time frame: one year
The clinical joint status will be examined in all patients with haemophilia by the World Federation Joint Examination Score and Haemophilia Joint Health Score. Higher score points imply an increased deficit in the functional and structural joint status as a sign of more pronounced haemophilic arthropathy, with a maximum possible value of 124 (no deficits = 0).
pressure pain thresholds
Time frame: one year
physiological Parameter in Newton
4 secondary outcomes
spontaneous joint ABR
Time frame: one year
subjective quality of life (SF-36)
Time frame: two years
subjective physical performance (HEP-Test-Q)
Time frame: two years
self-perceived functional abilities (Haemophilia Activities List)
Time frame: two years
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 May 2021.
- 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.
Thomas Hilberg, Prof. principal investigator
Head of Department
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Prof. Dr. Dr. Thomas Hilberg at 2 sites in Germany.
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 4 inclusion and 6 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: status not confirmed, as of the sponsor's last update on 11 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Thomas Hilberg, Prof. (004920237320812, hilberg@uni-wuppertal.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
total annual bleeding rate (time frame: one year). The study lists 3 primary outcomes in all. 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 11 May 2021.