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.
Verified November 2012 by Bio Products Laboratory
Sponsor:
Bio Products Laboratory
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
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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:
- To assess the pharmacokinetics of FACTOR X after a single dose of 50 IU/kg.
- 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 |
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:
- Children with hereditary severe FX deficiency (FX:C <1 IU/dL, based on their lowest reliable FX:C recorded).
- Children under 12 years old, whose parent/guardian has given written informed consent.
- Assent of child (for children aged 6-11 years).
- 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:
- Children with a history of inhibitor development to FX or a positive result at the Screening Visit (quantitative result of >0.6 BU).
- Children who have thrombocytopenia (platelets <50 x 109/L).
- Children who have clinically significant renal disease (serum creatinine >200 umol/L).
- Children who have clinically significant liver disease (serum ALT levels greater than three times the upper normal limit).
- Children known to have other coagulopathy or thrombophilia.
- 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 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
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 Hemorrhage Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn Pathologic Processes |
ClinicalTrials.gov processed this record on May 16, 2013