NCT01721681

Brief Summary

The primary objective of the study is to assess the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months. The secondary objectives of the study are:

  1. 1.To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
  2. 2.To assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks).

Trial Health

87
On Track

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Apr 2015

Geographic Reach
1 country

3 active sites

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

October 25, 2012

Completed
12 days until next milestone

First Posted

Study publicly available on registry

November 6, 2012

Completed
2.4 years until next milestone

Study Start

First participant enrolled

April 1, 2015

Completed
1.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2016

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

March 7, 2018

Completed
Last Updated

April 2, 2018

Status Verified

March 1, 2018

Enrollment Period

1.5 years

First QC Date

October 25, 2012

Results QC Date

October 11, 2017

Last Update Submit

March 7, 2018

Conditions

Factor X Deficiency

Outcome Measures

Primary Outcomes (1)

  • The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months

    The Investigator's assessment of the efficacy of FACTOR X in reduction/prevention of bleeding when given as routine prophylaxis over 6 months. The efficacy was assessed according to tabulated criteria; Excellent, good, poor, unassessable.

    6 months

Secondary Outcomes (2)

  • Safety of FACTOR X: Number of Participants Experiencing Adverse Events

    6 months

  • Pharmacokinetics: FX:C Incremental Recovery

    Baseline Visit and End of Study Visit, 30 minutes post-dose

Study Arms (1)

FACTOR X

EXPERIMENTAL

At the Baseline Visit, eligible children will receive a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children will be treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week is recommended, but is not mandatory. Each dose of FACTOR X must not exceed 60 IU/kg.

Biological: FACTOR X

Interventions

FACTOR XBIOLOGICAL
FACTOR X

Eligibility Criteria

AgeUp to 11 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Children with hereditary severe or moderate FX deficiency (FX:C \<5 IU/dL), based on their lowest reliable FX:C recorded.
  • Children under 12 years old, whose parent/guardian has given informed consent.
  • Children with a history of severe bleeding e.g.: intracranial haemorrhage, before starting prophylactic therapy, OR a mutation in the F10 gene causing a documented severe bleeding phenotype.

You may not qualify if:

  • Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia
  • Children must have no history or suspicion of inhibitors to factor X.
  • Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
  • Children with a history of unreliability or non-cooperation.
  • Children who are participating or have taken part in another trial within the last 30 days.
  • Children planning more than 4 weeks' continuous absence from the locality of the investigational site, between the Screening Visit and the End of Study Visit at approximately 6 months (26 weeks) post-Baseline.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (3)

Addenbrookes Hospital

Cambridge, CB2 0QQ, United Kingdom

Location

Great Ormond Street Hospital

London, WC1N 3JH, United Kingdom

Location

Sheffield Children's Hospital

Sheffield, S10 2TH, United Kingdom

Location

MeSH Terms

Conditions

Factor X Deficiency

Interventions

Factor X

Condition Hierarchy (Ancestors)

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

Intervention Hierarchy (Ancestors)

Enzyme PrecursorsEnzymes and CoenzymesBlood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Limitations and Caveats

2 subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the Per-Protocol analysis. 9 unique subjects were enrolled even though there were 11 treatment cycles

Results Point of Contact

Title
Head of Medical Affairs
Organization
Bio Products Laboratory Ltd
medinfo@bpl.co.uk
+44 20 8957 2200

Study Officials

  • Ri Liesner, Dr

    Great Ormond Street Hospital

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
GT60
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
PREVENTION
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 25, 2012

First Posted

November 6, 2012

Study Start

April 1, 2015

Primary Completion

October 1, 2016

Study Completion

October 1, 2016

Last Updated

April 2, 2018

Results First Posted

March 7, 2018

Record last verified: 2018-03

Locations

GB(3)