Phase 3
N=9
A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years
Factor X Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT01721681 ↗Enrolled (actual)
9
Serious AEs
11.1%
Results posted
Mar 2018
Primary outcome: Primary: The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months — 9 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- FACTOR X (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Bio Products Laboratory
- Primary completion
- Oct 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months |
9 | — |
| SECONDARY Safety of FACTOR X: Number of Participants Experiencing Adverse Events |
8 | — |
| SECONDARY Pharmacokinetics: FX:C Incremental Recovery |
1.74 | — |
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).
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT01721681). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.