Hemophilia APhase 4RELOQNCT04728217
Health Related Quality of Life of Youth and Young Adults With Haemophilia A
A Phase 4 study testing Efmoroctocog Alfa Injection [Eloctate] in people with Hemophilia A, run by The League of Clinical Research, Russia at 1 site in Russia.
Trial summary
Research question: Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis? A 12-month prospective open-label, single-arm multicentre study. Read more about this study
- Who is it for?
- Ages 12 to 25 years
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Efmoroctocog Alfa Injection [Eloctate]
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteRussia
- Study dates
- 15 Apr 2021to 20 Feb 2023
- Phase
- Phase 4
- 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 12 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Signed consent form.
- Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis.
Who cannot join
- Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product.
- History of Factor VIII inhibitors.
- Patients who have other haemostatic disorders.
- Patients participating in interventional studies.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.
Read the detailed description
Quality of life is among the key factors when medical decision about management of haemophilia is taken. There are no scientific data on the HRQoL in patients after switching from Standard Half Life (SHL) FVIII prophylaxis to efmoroctocog alfa (Extended Half Life, EHL) prophylaxis in Russia.
It will be a 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations. Providing the centers/ patients with the study drug (efmoroctocog alfa) may be considered as interventional component, whereas all administrations should be in accordance with Instruction on Medical Use approved by Russian Ministry of Health.
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
No hypothesis is formulated for this exploratory study. The obtained data will be used to assess real world data on quality of life and clinical outcomes in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis in routine medical practice in Russia
- Conditions
- Hemophilia A
- Being tested
- Efmoroctocog Alfa Injection [Eloctate]
- Keywords
- Haemophilia A, Health-related Quality of Life, Efmoroctocog alfa
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efmoroctocog Alfa Injection [Eloctate]Drug
Efmoroctocog alfa should be administered according to Summary of Product Characteristics (SmPC)/ Instruction on Medical Use approved by Russian Ministry of Health ЛП-006034 (13.01.2020).
| Study group | Role | What they receive |
|---|---|---|
| Patients receiving Efmoroctocog alfa | Experimental | For long term prophylaxis, the recommended starting dose is 50 IU of factor VIII per kg body weight at intervals of 3 to 5 days. The dose may be adjusted based on patient response in the range of 25 to 65 IU/kg. In some cases, especially in younger patients, shorter dosage intervals or higher doses may be necessary. Receives: Drug: Efmoroctocog Alfa Injection [Eloctate] |
What is being measured
The primary outcome is the main question the study is designed to answer.
Health-related quality of life
Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) - for patients of 18-25 y.o.; total score, domain scores.
or Haemophilia-specific Quality of Life assessment for children and adolescents Short Form (Haemo-QoL SF) - for patients 12-17 y.o.; total score, domain scores.
Measured: Month 12
Secondary outcomes (18)
Spontaneous bleedings
Number from previous visit
Measured: Month 1 - Month 3 - Month 6 - Month 12
Spontaneous bleedings - localisation
Localisation of spontaneous bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings
Number from previous visit
Measured: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings - localisation
Localisation of post-traumatic bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings
Dose of efmorococtog alfa: IU.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: major
Number from previous visit.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: minor
Number from previous visit.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures
Dose of efmorococtog alfa: IU.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations
Number from previous visit.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations: duration
Total duration of all hospitalisations: days.
Measured: Month 1 - Month 3 - Month 6 - Month 12
Hemophilia Joint Health Score
Hemophilia Joint Health Score 2.1 for knee, elbow, ankle (left, right): score.
Measured: Day 0 - Month 6 - Month 12
Target Joints
Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.
Measured: Day 0 - Month 6 - Month 12
Ultrasound Scoring
Ultrasound scoring (HEAD-US): score.
Measured: Day 0 - Month 6 - Month 12
Factor VIII Activity
Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL.
Measured: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Factor VIII Inhibitor Activity
Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.
Measured: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa
Administration of efmoroctocog alfa, frequency: no. of injections per week.
Measured: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa: factor consumption
Administration of efmoroctocog alfa, factor consumption: IU per week, IU/kg per week (calculation).
Measured: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Previous treatment/ prophylaxis of haemophilia
Generic drug name.
Measured: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Jan 2021
- Study start15 Apr 2021
- Primary completion20 Feb 2023
- Study completion20 Feb 2023
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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A 12-month prospective open-label, single-arm multicentre study.
- Masking
- None (open label)
- Enrolment
- 30 participants (actual)
Sponsor and investigators
- Lead sponsor
- The League of Clinical Research, Russia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04728217
- Sponsor's protocol ID
- RELOQ
- Acronym
- RELOQ
- Status last verified
- Dec 2023
- First submitted
- 20 Jan 2021
- Last updated
- 12 Dec 2023
- 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 12 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia
- ClinicalTrials.gov ID
- NCT04728217
- Acronym / sponsor ID
- RELOQ / RELOQ
- Conditions
- Hemophilia A
- Keywords
- Haemophilia A, Health-related Quality of Life, Efmoroctocog alfa
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (actual)
- Lead sponsor
- The League of Clinical Research, RussiaUniversity, hospital or other
- Study start
- 15 Apr 2021 (actual)
- Primary completion
- 20 Feb 2023 (actual)
- Study completion
- 20 Feb 2023 (actual)
- First posted
- 28 Jan 2021 (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.
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.
Detailed description
Quality of life is among the key factors when medical decision about management of haemophilia is taken. There are no scientific data on the HRQoL in patients after switching from Standard Half Life (SHL) FVIII prophylaxis to efmoroctocog alfa (Extended Half Life, EHL) prophylaxis in Russia.
It will be a 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations. Providing the centers/ patients with the study drug (efmoroctocog alfa) may be considered as interventional component, whereas all administrations should be in accordance with Instruction on Medical Use approved by Russian Ministry of Health.
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
No hypothesis is formulated for this exploratory study. The obtained data will be used to assess real world data on quality of life and clinical outcomes in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis in routine medical practice in Russia
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A 12-month prospective open-label, single-arm multicentre study.
| Arm | Type | Receives |
|---|---|---|
| Patients receiving Efmoroctocog alfa | Experimental | Drug: Efmoroctocog Alfa Injection [Eloctate]For long term prophylaxis, the recommended starting dose is 50 IU of factor VIII per kg body weight at intervals of 3 to 5 days. The dose may be adjusted based on patient response in the range of 25 to 65 IU/kg. In some cases, especially in younger patients, shorter dosage intervals or higher doses may be necessary. |
Who may qualify
- Ages
- 12 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (2 listed)
- Signed consent form.
- Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis.
Key exclusion criteria (4 listed)
- Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product.
- History of Factor VIII inhibitors.
- Patients who have other haemostatic disorders.
- Patients participating in interventional studies.
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 |
|---|---|---|
| Efmoroctocog Alfa Injection [Eloctate]Efmoroctocog alfa should be administered according to Summary of Product Characteristics (SmPC)/ Instruction on Medical Use approved by Russian Ministry of Health ЛП-006034 (13.01.2020). | Drug | Patients receiving Efmoroctocog alfa |
Primary outcome
Health-related quality of life
Time frame: Month 12
Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) - for patients of 18-25 y.o.; total score, domain scores. or Haemophilia-specific Quality of Life assessment for children and adolescents Short Form (Haemo-QoL SF) - for patients 12-17 y.o.; total score, domain scores.
18 secondary outcomes
Spontaneous bleedings
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Spontaneous bleedings - localisation
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings - localisation
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: major
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: minor
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations: duration
Time frame: Month 1 - Month 3 - Month 6 - Month 12
Hemophilia Joint Health Score
Time frame: Day 0 - Month 6 - Month 12
Target Joints
Time frame: Day 0 - Month 6 - Month 12
Ultrasound Scoring
Time frame: Day 0 - Month 6 - Month 12
Factor VIII Activity
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Factor VIII Inhibitor Activity
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa: factor consumption
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Previous treatment/ prophylaxis of haemophilia
Time frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
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 12 Dec 2023.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Efmoroctocog Alfa Injection [Eloctate] in people with Hemophilia A, run by The League of Clinical Research, Russia at 1 site in Russia.
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 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: finished, as of the sponsor's last update on 12 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Russia. 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?
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?
Health-related quality of life (time frame: Month 12). 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 12 Dec 2023.