NCT02314325

Brief Summary

This trial is designed to assess if there is evidence of subclinical joint bleeding on MRI/X-Ray in adults with severe Haemophilia A while on standard and/or pharmacokinetically tailored prophylaxis regimens. Participants with severe Haemophilia A will have longitudinal MRI and XRay imaging of their elbows, ankles and knees at 0, 6 and 18 months while on standard ( 0-6 months) and then pharmacokinetically tailored (7-18 months) recombinant Factor VIII prophylaxis.

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
20

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2014

Typical duration for phase_4

Geographic Reach
1 country

1 active site

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

Study Start

First participant enrolled

April 1, 2014

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

September 3, 2014

Completed
3 months until next milestone

First Posted

Study publicly available on registry

December 11, 2014

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 1, 2016

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2016

Completed
Last Updated

December 11, 2014

Status Verified

December 1, 2014

Enrollment Period

2.1 years

First QC Date

September 3, 2014

Last Update Submit

December 10, 2014

Conditions

Severe Haemophilia A

Keywords

Factor VIII deficiencyArthropathyMRIXRayJointHJHS

Outcome Measures

Primary Outcomes (1)

  • Number of subclinical haemarthroses

    The number of subclinical haemarthroses identifed on serial MRI scans of elbow, knee and ankle joints

    18 months

Secondary Outcomes (8)

  • EQ5D QoL score

    18 months

  • Percentage of prescribed doses of prophylaxis taken

    18 months

  • IPAQ score

    18 months

  • Haemophilia joint health score (HJHS)

    18 months

  • Amount of FVIII usage (units)

    18 months

  • +3 more secondary outcomes

Study Arms (2)

Standard prophylaxis

ACTIVE COMPARATOR

Advate \[Antihemophilic Factor(Recombinant)\] 20-40 IU/kg 5-7 infusions per 14days

Drug: ADVATE [Antihemophilic Factor (Recombinant)]

Pharmacokinetic tailored prophylaxis

EXPERIMENTAL

Advate \[Antihemophilic Factor(Recombinant)\] dose determined by individual patient pharmacokinetics and infusions administerd on alternate days

Drug: ADVATE [Antihemophilic Factor (Recombinant)]

Interventions

ADVATE [Antihemophilic Factor (Recombinant)]DRUG

In Arm 1 prior patients will be dosed as per body weight 20-40 IU/kg 5-7 infusions per fortnight

Standard prophylaxis

Eligibility Criteria

Age18 Years+
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Male patients with severe Haemophilia A (baseline Factor VIII level of \<0.01 IU/mL)
  • Age 18 years and above
  • Patients taking any regular prophylactic regimen (defined as regular factor VIII infusions, at least 5 times a fortnight, with the aim of minimising haemarthroses and other clinically significant bleeds).
  • Low titre inhibitors, past history of an inhibitor, abnormal liver function, drugs that interfere with haemostasis and low Cluster of Differentiation 4 (CD4) counts are allowed.

You may not qualify if:

  • Presence of a target joint on prophylaxis (defined as 3 bleeds into one joint, during a 6 month period, during the last year).
  • The occurrence of more than 3 haemarthroses in the last year that required more than 2 infusions to resolve.
  • Patients with a learning disability or dementia
  • Prisoners
  • Adults who are unconscious/unable to give informed consent
  • Participants with a pacemaker or implanted medical devices which are unsuitable to have a MRI will be excluded from the MRI scans during the trial but may proceed with other components.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. James's Hospital

Dublin, Dublin, D8, Ireland

RECRUITING

MeSH Terms

Conditions

Hemophilia AJoint Diseases

Interventions

Factor VIII

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Niamh M O'Connell, PhD, FRCPath

    St. James's Hospital, Ireland

    PRINCIPAL INVESTIGATOR

  • James O'Donnell, PhD, FRCPath

    St. James's Hospital, Ireland

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Michelle M Lavin, FRCPath

CONTACT

+35314162142mlavin@stjames.ie

Niamh M O'Connell, FRCPath

CONTACT

+35314162142

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
DIAGNOSTIC
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Consultant Haematologist

Study Record Dates

First Submitted

September 3, 2014

First Posted

December 11, 2014

Study Start

April 1, 2014

Primary Completion

May 1, 2016

Study Completion

October 1, 2016

Last Updated

December 11, 2014

Record last verified: 2014-12

Locations

IE(1)