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

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.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Efmoroctocog Alfa Injection [Eloctate]

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 Type: Interventional
• Enrollment (Actual): 30
• Phase: Phase 4

Contacts and Locations

Study Locations

• Russian Federation
  • Moscow, Russian Federation, 119590
    • League of Clinical Research (LeagueCRR)

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 25 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	Month 12

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Spontaneous bleedings	Number from previous visit	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.	Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings	Number from previous visit	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.	Month 1 - Month 3 - Month 6 - Month 12
Administration of efmorococtog alfa for correction of bleedings	Dose of efmorococtog alfa: IU.	Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: major	Number from previous visit.	Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: minor	Number from previous visit.	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.	Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations	Number from previous visit.	Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations: duration	Total duration of all hospitalisations: days.	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.	Day 0 - Month 6 - Month 12
Target Joints	Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.	Day 0 - Month 6 - Month 12
Ultrasound Scoring	Ultrasound scoring (HEAD-US): score.	Day 0 - Month 6 - Month 12
Factor VIII Activity	Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL.	Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Factor VIII Inhibitor Activity	Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.	Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Administration of efmoroctocog alfa	Administration of efmoroctocog alfa, frequency: no. of injections per week.	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).	Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Previous treatment/ prophylaxis of haemophilia	Generic drug name.	Day 0 - Month 1 - Month 3 - Month 6 - Month 12

Collaborators and Investigators

• Sponsor: The League of Clinical Research, Russia

Study record dates

Study Major Dates

• Study Start (Actual): April 15, 2021
• Primary Completion (Actual): February 20, 2023
• Study Completion (Actual): February 20, 2023

Study Registration Dates

• First Submitted: January 20, 2021
• First Submitted That Met QC Criteria: January 23, 2021
• First Posted (Actual): January 28, 2021

Study Record Updates

• Last Update Posted (Estimated): December 12, 2023
• Last Update Submitted That Met QC Criteria: December 11, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Health-related Quality of Life; Haemophilia A; Efmoroctocog alfa
• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Physiological Effects of Drugs; Immunologic Factors; Coagulants; Immunoglobulin Fc Fragments; Factor VIII
• Other Study ID Numbers: RELOQ

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No