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

December 11, 2023 updated by: The League of Clinical Research, Russia

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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
30

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Month 12

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Spontaneous bleedings
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Number from previous visit
Month 1 - Month 3 - Month 6 - Month 12
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.
Month 1 - Month 3 - Month 6 - Month 12
Post-traumatic bleedings
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Number from previous visit
Month 1 - Month 3 - Month 6 - Month 12
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.
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
Dose of efmorococtog alfa: IU.
Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: major
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Number from previous visit.
Month 1 - Month 3 - Month 6 - Month 12
Surgery/ Invasive Procedures: minor
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Number from previous visit.
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
Dose of efmorococtog alfa: IU.
Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Number from previous visit.
Month 1 - Month 3 - Month 6 - Month 12
Hospitalisations: duration
Time Frame: Month 1 - Month 3 - Month 6 - Month 12
Total duration of all hospitalisations: days.
Month 1 - Month 3 - Month 6 - Month 12
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.
Day 0 - Month 6 - Month 12
Target Joints
Time Frame: Day 0 - Month 6 - Month 12
Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.
Day 0 - Month 6 - Month 12
Ultrasound Scoring
Time Frame: Day 0 - Month 6 - Month 12
Ultrasound scoring (HEAD-US): score.
Day 0 - Month 6 - Month 12
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.
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
Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.
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, frequency: no. of injections per week.
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
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
Time Frame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Generic drug name.
Day 0 - Month 1 - Month 3 - Month 6 - Month 12

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
The League of Clinical Research, Russia

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 15, 2021

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 20, 2023

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
February 20, 2023

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
January 20, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
January 23, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 28, 2021

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
December 12, 2023

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 11, 2023

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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