NCT04728217

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
30

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2021

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 20, 2021

Completed
8 days until next milestone

First Posted

Study publicly available on registry

January 28, 2021

Completed
3 months until next milestone

Study Start

First participant enrolled

April 15, 2021

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 20, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 20, 2023

Completed
Last Updated

December 12, 2023

Status Verified

December 1, 2023

Enrollment Period

1.9 years

First QC Date

January 20, 2021

Last Update Submit

December 11, 2023

Conditions

Hemophilia A

Keywords

Haemophilia AHealth-related Quality of LifeEfmoroctocog alfa

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (18)

  • Spontaneous bleedings

    Month 1 - Month 3 - Month 6 - Month 12

  • Spontaneous bleedings - localisation

    Month 1 - Month 3 - Month 6 - Month 12

  • Post-traumatic bleedings

    Month 1 - Month 3 - Month 6 - Month 12

  • Post-traumatic bleedings - localisation

    Month 1 - Month 3 - Month 6 - Month 12

  • Administration of efmorococtog alfa for correction of bleedings

    Month 1 - Month 3 - Month 6 - Month 12

  • +13 more secondary outcomes

Study Arms (1)

Patients receiving Efmoroctocog alfa

EXPERIMENTAL

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]

Interventions

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).

Patients receiving Efmoroctocog alfa

Eligibility Criteria

Age12 Years - 25 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

League of Clinical Research (LeagueCRR)

Moscow, 119590, Russia

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

factor VIII-Fc fusion protein

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Model Details: A 12-month prospective open-label, single-arm multicentre study.
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 20, 2021

First Posted

January 28, 2021

Study Start

April 15, 2021

Primary Completion

February 20, 2023

Study Completion

February 20, 2023

Last Updated

December 12, 2023

Record last verified: 2023-12

Data Sharing

IPD Sharing
Will not share

Locations

RU(1)