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Health Related Quality of Life of Youth and Young Adults With Haemophilia A (RELOQ)

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ClinicalTrials.gov Identifier: NCT04728217
Recruitment Status : Recruiting
First Posted : January 28, 2021
Last Update Posted : April 7, 2022
Sponsor:
Information provided by (Responsible Party):
The League of Clinical Research, Russia

Study Description
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Brief 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. 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.


Condition or disease Intervention/treatment Phase
Hemophilia A Drug: Efmoroctocog Alfa Injection [Eloctate] Phase 4

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
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: A 12-month prospective open-label, single-arm multicentre study.
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia
Actual Study Start Date : April 15, 2021
Estimated Primary Completion Date : August 15, 2022
Estimated Study Completion Date : November 15, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: Patients receiving Efmoroctocog alfa
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.
 Drug: 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).


Outcome Measures
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Primary Outcome Measures :
  1. 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.



Secondary Outcome Measures :
  1. Spontaneous bleedings [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Number from previous visit

  2. Spontaneous bleedings - localisation [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Localisation of spontaneous bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.

  3. Post-traumatic bleedings [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Number from previous visit

  4. Post-traumatic bleedings - localisation [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Localisation of post-traumatic bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.

  5. Administration of efmorococtog alfa for correction of bleedings [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Dose of efmorococtog alfa: IU.

  6. Surgery/ Invasive Procedures: major [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Number from previous visit.

  7. Surgery/ Invasive Procedures: minor [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Number from previous visit.

  8. Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Dose of efmorococtog alfa: IU.

  9. Hospitalisations [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Number from previous visit.

  10. Hospitalisations: duration [ Time Frame: Month 1 - Month 3 - Month 6 - Month 12 ]
    Total duration of all hospitalisations: days.

  11. Hemophilia Joint Health Score [ Time Frame: Day 0 - Month 6 - Month 12 ]
    Hemophilia Joint Health Score 2.1 for knee, elbow, ankle (left, right): score.

  12. Target Joints [ Time Frame: Day 0 - Month 6 - Month 12 ]
    Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.

  13. Ultrasound Scoring [ Time Frame: Day 0 - Month 6 - Month 12 ]
    Ultrasound scoring (HEAD-US): score.

  14. Factor VIII Activity [ Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12 ]
    Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL.

  15. Factor VIII Inhibitor Activity [ Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12 ]
    Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.

  16. Administration of efmoroctocog alfa [ Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12 ]
    Administration of efmoroctocog alfa, frequency: no. of injections per week.

  17. Administration of efmoroctocog alfa: factor consumption [ Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12 ]
    Administration of efmoroctocog alfa, factor consumption: IU per week, IU/kg per week (calculation).

  18. Previous treatment/ prophylaxis of haemophilia [ Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12 ]
    Generic drug name.


Eligibility Criteria
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Ages Eligible for Study:   12 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Signed consent form.
  2. Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis.

Exclusion Criteria:

  1. Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product.
  2. History of Factor VIII inhibitors.
  3. Patients who have other haemostatic disorders.
  4. Patients participating in interventional studies.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04728217


Contacts
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Contact: Oleg Melikhov, MD 0079166950530 melikhov.oleg@gmail.com
Contact: Svetlana Elkonina 0079164454877 elkonina.svetlana@gmail.com

Locations
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Russian Federation
League of Clinical Research (LeagueCRR) Recruiting
Moscow, Russian Federation, 119590
Contact: Oleg G Melikhov, MD, PhD    0079166950530    melikhov.oleg@gmail.com   
Contact: Svetlana I Elkonina, MD    0079164454877    elkonina.svetlana@gmail.com   
Sponsors and Collaborators
The League of Clinical Research, Russia
More Information
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Responsible Party: The League of Clinical Research, Russia
ClinicalTrials.gov Identifier: NCT04728217    
Other Study ID Numbers: RELOQ
First Posted: January 28, 2021    Key Record Dates
Last Update Posted: April 7, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by The League of Clinical Research, Russia:
Haemophilia A
Health-related Quality of Life
Efmoroctocog alfa
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
 Genetic Diseases, Inborn
Factor VIII
Immunoglobulin Fc Fragments
Coagulants
Immunologic Factors
Physiological Effects of Drugs