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A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years

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ClinicalTrials.gov Identifier: NCT01721681
Recruitment Status : Completed
First Posted : November 6, 2012
Results First Posted : March 7, 2018
Last Update Posted : April 2, 2018
Sponsor:
Information provided by (Responsible Party):
Bio Products Laboratory

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. 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 or disease Intervention/treatment Phase
Factor X Deficiency Biological: FACTOR X Phase 3

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 9 participants
Intervention Model: Single Group Assignment
Masking: None (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
Study Start Date : April 2015
Actual Primary Completion Date : October 2016
Actual Study Completion Date : October 2016

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding
U.S. FDA Resources

Arm Intervention/treatment
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



Primary Outcome Measures :
  1. The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months [ Time Frame: 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.



Secondary Outcome Measures :
  1. Safety of FACTOR X: Number of Participants Experiencing Adverse Events [ Time Frame: 6 months ]
    One of the secondary objectives was to assess the safety of FACTOR X when given as routine prophylaxis over 6 months (26 weeks). The general strategy of the safety evaluation was to examine the summaries for any trends. No formal hypothesis was carried out. The number of participants who experienced Adverse Events is provided.

  2. Pharmacokinetics: FX:C Incremental Recovery [ Time Frame: Baseline Visit and End of Study Visit, 30 minutes post-dose ]
    One of the secondary objectives was to assess the pharmacokinetics (FX:C incremental recovery 30 minute post-dose at the Visit 1 (Baseline) and the End of Study Visit after a single dose of 50 IU/kg). The overall mean IR calculated for both visits is presented in the outcome measure table.



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Ages Eligible for Study:   up to 11 Years   (Child)
Sexes Eligible for Study:   All
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01721681


Locations
United Kingdom
Addenbrookes Hospital
Cambridge, United Kingdom, CB2 0QQ
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Sheffield Children's Hospital
Sheffield, United Kingdom, S10 2TH
Sponsors and Collaborators
Bio Products Laboratory
Investigators
Principal Investigator: Ri Liesner, Dr Great Ormond Street Hospital

Responsible Party: Bio Products Laboratory
ClinicalTrials.gov Identifier: NCT01721681     History of Changes
Other Study ID Numbers: Ten 02
2012-003093-98 ( EudraCT Number )
First Posted: November 6, 2012    Key Record Dates
Results First Posted: March 7, 2018
Last Update Posted: April 2, 2018
Last Verified: March 2018

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