NCT04583930

Brief Summary

Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. With the development of recombinant factor VIII Fc fusion protein (rFVIIIFc) the conventional routine prophylaxis regime is complemented by an extended half-life (EHL) factor replacement prophylaxis with the potential of improved bleed prevention and reduced injection frequency at similar factor consumption. Aim of this longitudinal multicentre study is to evaluate the influence of an EHL factor replacement regime with rFVIIIFc on haemophilic specific parameters (annual bleeding rate, bleeding localisation), joint status, pain, functional parameters, treatment adherence and health-related quality of life in PwH A.

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
48

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Feb 2021

Typical duration for all trials

Geographic Reach
1 country

2 active sites

Status
unknown

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

July 28, 2020

Completed
3 months until next milestone

First Posted

Study publicly available on registry

October 12, 2020

Completed
4 months until next milestone

Study Start

First participant enrolled

February 1, 2021

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2023

Completed
20 days until next milestone

Study Completion

Last participant's last visit for all outcomes

April 20, 2023

Completed
Last Updated

May 11, 2021

Status Verified

May 1, 2021

Enrollment Period

2.2 years

First QC Date

July 28, 2020

Last Update Submit

May 6, 2021

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (3)

  • total annual bleeding rate

    number of total bleedings

    one year

  • orthopaedic joint status (Haemophilia health joint score)

    The clinical joint status will be examined in all patients with haemophilia by the World Federation Joint Examination Score and Haemophilia Joint Health Score. Higher score points imply an increased deficit in the functional and structural joint status as a sign of more pronounced haemophilic arthropathy, with a maximum possible value of 124 (no deficits = 0).

    one year

  • pressure pain thresholds

    physiological Parameter in Newton

    one year

Secondary Outcomes (4)

  • spontaneous joint ABR

    one year

  • subjective quality of life (SF-36)

    two years

  • subjective physical performance (HEP-Test-Q)

    two years

  • self-perceived functional abilities (Haemophilia Activities List)

    two years

Study Arms (1)

Patients with Haemophilia A

* Patients suffering from moderate to severe haemophilia A * Age ≥ 18-years * Treatment with FVIII prophylaxis * Submitted written informed consent

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Patients with moderate or severe Haemophilia A will be included in this study consecutively, who will be switched from FVIII prophylaxis on rFVIIIFc prophylaxis.

You may qualify if:

  • Patients suffering from moderate to severe haemophilia A
  • Age ≥ 18-years
  • Treatment with FVIII prophylaxis
  • Submitted written informed consent

You may not qualify if:

  • Patients suffering from other bleeding diseases
  • Patients with inhibitors
  • Patients without written informed consent
  • Age \< 18-year-old
  • Any surgeries up to 6 months before the examination date
  • Suffering from different rheumatologic diseases like M. Bechterew, Psoriasis or other local or generalized joint infections (Borreliosis, septic arthritis)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Department of Sports Medicine, University of Wuppertal

Wuppertal, North Rhine-Westphalia, 42117, Germany

RECRUITING

Department of Sports Medicine

Wuppertal, Northwest, 42117, Germany

RECRUITING

MeSH Terms

Conditions

Hemophilia A

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 Officials

  • Thomas Hilberg, Prof.

    Head of Department

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Thomas Hilberg, Prof.

CONTACT

004920237320812hilberg@uni-wuppertal.de

Jamil Hmida

CONTACT

0049202439jamil.hmida@uni-wuppertal.de

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Head of Department

Study Record Dates

First Submitted

July 28, 2020

First Posted

October 12, 2020

Study Start

February 1, 2021

Primary Completion

March 31, 2023

Study Completion

April 20, 2023

Last Updated

May 11, 2021

Record last verified: 2021-05

Data Sharing

IPD Sharing
Will not share

Locations

DE(2)