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

Bio Products Laboratory

Status and phase: Completed; Phase 3

Conditions: Factor X Deficiency

Treatments: Biological: FACTOR X

Study type: Interventional

Funder types: Other

Identifiers

NCT01721681

Ten 02

2012-003093-98 (EudraCT Number)

Details and patient eligibility

About

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).

Enrollment: 9 patients

Sex: All

Ages: Under 11 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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9 participants in 1 patient group

FACTOR X

Experimental group

Description:

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.

Treatment:

Biological: FACTOR X