A Study to Investigate BPL's Factor X in the Prophylaxis of Bleeding in Children <12 Years

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2016 by Bio Products Laboratory
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory
ClinicalTrials.gov Identifier:
NCT01721681
First received: October 25, 2012
Last updated: March 4, 2016
Last verified: March 2016
  Purpose

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

Condition Intervention Phase
Factor X Deficiency
Biological: FACTOR X
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Prevention
Official Title: A Phase III Open, Multicentre Study to Investigate the Safety, Pharmacokinetics and Efficacy of BPL's High Purity Factor X in the Prophylaxis of Bleeding in Factor X Deficient Children Under the Age of 12 Years

Resource links provided by NLM:


Further study details as provided by Bio Products Laboratory:

Primary Outcome Measures:
  • The investigator's assessment of the efficacy of FACTOR X in the reduction/prevention of bleeding when given as routine prophylaxis over 6 months. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
    This will take into account the child's risk of breakthrough bleeding (due to bleeding history, treatment history and genetic mutation) and other relevant factors eg compliance with the protocol, attainment of required factor X activity trough levels. Retrospective data will be included in the analysis as supportive data.


Secondary Outcome Measures:
  • Number of bleeds per month including severity, duration, location and cause [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Investigator's and parent's/guardian's assessment of efficacy in treating a bleed for all bleeds (parent/guardian assessment) and for bleeds treated at hospital (Investigator's assessment). [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Other Outcome Measures:
  • Factor X activity trough levels at all scheduled study visits and at all Bleed Assessment and Trough Measurement unscheduled visits. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Factor X activity 30 minute post-dose incremental recovery at the Baseline Visit and the End of Study Visit [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Factor X activity incremental recovery and trough levels following any change in dose regimen required for clinical reasons/insufficient trough levels. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Dose to treat a bleed (IU/kg factor X activity) (including initial dose for new bleeds and any repeated doses for ongoing bleeds), number of infusions to treat a bleed and dose per infusion; all analysed on a per-bleed and a per-subject basis. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Total dose in IU/kg of factor X (factor X activity), total number of infusions and average dose per infusion for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
  • Average monthly dose in IU/kg of factor X (factor X activity), and average monthly number of infusions for: prophylactic use, to treat a bleed, any additional preventative use, any surgical use and overall use; all analysed on a per subject basis. [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 9
Study Start Date: April 2015
Estimated Study Completion Date: July 2016
Estimated Primary Completion Date: July 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: FACTOR X

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

  Eligibility

Ages Eligible for Study:   up to 11 Years   (Child)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria

  1. Children with hereditary severe or moderate FX deficiency (FX:C <5 IU/dL), based on their lowest reliable FX:C recorded.
  2. Children under 12 years old, whose parent/guardian has given informed consent.
  3. 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.

Exclusion Criteria

  1. Children must not suffer from clinically significant liver disease, renal disease, or other coagulopathy or thrombophilia
  2. Children must have no history or suspicion of inhibitors to factor X.
  3. Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
  4. Children with a history of unreliability or non-cooperation.
  5. Children who are participating or have taken part in another trial within the last 30 days.
  6. 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01721681

Contacts
Contact: Head of Medical Affairs +44 20 8957 2200 medinfo@bpl.co.uk

Locations
United Kingdom
Addenbrookes Hospital Completed
Cambridge, United Kingdom, CB2 0QQ
Great Ormond Street Hospital Recruiting
London, United Kingdom, WC1N 3JH
Contact: Ri Liesner    0207 829 8846    Ri.Liesner@gosh.nhs.uk   
Sheffield Children's Hospital Completed
Sheffield, United Kingdom, S10 2TH
Sponsors and Collaborators
Bio Products Laboratory
Investigators
Principal Investigator: Ri Liesner, Dr Great Ormond Street Hospital
  More Information

Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01721681     History of Changes
Other Study ID Numbers: Ten 02  2012-003093-98 
Study First Received: October 25, 2012
Last Updated: March 4, 2016
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Spain: Agencia Española de Medicamentos y Productos Sanitarios

Additional relevant MeSH terms:
Factor X Deficiency
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on August 25, 2016