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NCT Number: NCT01721681

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

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).

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Key information

Conditions

Factor X Deficiency Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

Up to 11 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Addenbrookes Hospital, Cambridge, United Kingdom

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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.

Treatment and study plan

FACTOR X

Biological

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

Bio Products Laboratory

Other

Registry information

Official study 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

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Nov 6, 2012
Registry last updated
Apr 2, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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