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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified November 2012 by Bio Products Laboratory
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory
ClinicalTrials.gov Identifier:
NCT01721681
First received: October 25, 2012
Last updated: November 13, 2012
Last verified: November 2012
  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 12 months.

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 primary efficacy variable is the investigator's assessment of the efficacy of FACTOR X in the prevention of bleeding when given as routine prophylaxis over 12 months [ Time Frame: 1 year ] [ Designated as safety issue: No ]
    The efficacy criteria will be 'excellent', 'good', 'poor' or 'unassessable' and will be based on the number of major and minor breakthrough bleeds, and the number of excessive bleeding episodes following injury.


Secondary Outcome Measures:
  • Area Under Curve (AUC) [ Time Frame: predose, 30 mins, 4 hours, 24 hours, 48 hours and 72 hours ] [ Designated as safety issue: Yes ]
    For children equal to or greater than 1 year old.

  • Area Under Curve (AUC) [ Time Frame: At two post-dose timepoints : 4 hours and 72 hours, or 30 mins and 48 hours, or 4 hours and 24 hours or 30 mins and 72 hours ] [ Designated as safety issue: Yes ]
    For children < 1 year old for for children equal to or > 1 year old whose bodyweight is less than 10.5kg


Other Outcome Measures:
  • Safety Outcomes [ Time Frame: Up to 28 days days after the final dose of FACTOR X. ] [ Designated as safety issue: Yes ]

    Adverse events, haematology, serum biochemistry, PT and APTT, viral serology, FX inhibitor screens, vital signs, physical examination, infusion site observations, genotype analysis (optional) and number of exposure days.

    Archive samples will be collected before first dose of FACTOR X and at the End-of-Study Visit.



Estimated Enrollment: 16
Study Start Date: February 2013
Estimated Study Completion Date: May 2015
Estimated Primary Completion Date: May 2015 (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 12 months (52 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
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Children with hereditary severe FX deficiency (FX:C <1 IU/dL, based on their lowest reliable FX:C recorded).
  2. Children under 12 years old, whose parent/guardian has given written informed consent.
  3. Assent of child (for children aged 6-11 years).
  4. Children with a history of severe bleeding (a minimum of one bleed with a bleed score of 3 or 4, Appendix XI) or a mutation in the F10 gene causing a documented severe bleeding phenotype.

Exclusion Criteria:

The presence of any one of these criteria makes the child ineligible:

  1. Children with a history of inhibitor development to FX or a positive result at the Screening Visit (quantitative result of >0.6 BU).
  2. Children who have thrombocytopenia (platelets <50 x 109/L).
  3. Children who have clinically significant renal disease (serum creatinine >200 umol/L).
  4. Children who have clinically significant liver disease (serum ALT levels greater than three times the upper normal limit).
  5. Children known to have other coagulopathy or thrombophilia.
  6. Children who have known or suspected hypersensitivity to the investigational medicinal product or its excipients.
  7. Children with a history of unreliability or non-cooperation.
  8. Children who are participating or have taken part in another trial within the last 30 days.
  9. Children who are planning more than 4 weeks' continuous absence from the locality of the investigational site, between the Screening Visit and the 12 Month Visit.

    .

  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: Miranda Norton, Dr +44 (0)208 957 2661 miranda.norton@bpl.co.uk
Contact: Kate Gillanders +44 (0)208 957 2562 kate.gillanders@bpl.co.uk

Locations
United Kingdom
Great Ormond Street Hospital Not yet recruiting
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
Bio Products Laboratory
Investigators
Principal Investigator: Ri Liesner, Dr Great Ormond Street Hospital
  More Information

No publications provided

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: November 13, 2012
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 September 22, 2014