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Completed

NCT Number: NCT04728217

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

12 year–25 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

League of Clinical Research (LeagueCRR)

Moscow, 119590, Russia

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

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).

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

The League of Clinical Research, Russia

Other

Registry information

Official study title

Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia

Acronym: RELOQ

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Jan 28, 2021
Registry last updated
Dec 12, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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