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

The League of Clinical Research, Russia

Status and phase: Completed; Phase 4

Conditions: Hemophilia A

Treatments: Drug: Efmoroctocog Alfa Injection [Eloctate]

Study type: Interventional

Funder types: Other

Identifiers

NCT04728217

RELOQ

Details and patient eligibility

About

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.

Full 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

Enrollment: 30 patients

Sex: All

Ages: 12 to 25 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Patients receiving Efmoroctocog alfa

Experimental group

Description:

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.

Treatment:

Drug: Efmoroctocog Alfa Injection [Eloctate]