Trial Outcomes & Findings for A Study to Investigate Bio Product Laboratory Ltd (BPL's) Factor X in the Prophylaxis of Bleeding in Children <12 Years (NCT NCT01721681)

Last Updated: 2018-04-02

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

9 participants

Primary outcome timeframe

6 months

Results posted on

2018-04-02

Participant Flow

Two subjects completed less than 26 weeks in the study. The subjects were re-enrolled and data from their first treatment cycle was excluded from the per-protocol analysis. 9 unique subjects were enrolled event though there were 11 treatment cycles.

Participant milestones

Participant milestones
Measure	Overall Study At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Overall Study STARTED	9
Overall Study COMPLETED	9
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Human Coagulation FACTOR X n=9 Participants At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Age, Categorical <=18 years	9 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United Kingdom	9 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Outcome measures

Outcome measures
Measure	Overall Study n=9 Participants At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months	9 Participants

SECONDARY outcome

Timeframe: 6 months

Population: The safety evaluation examined the summaries for any trends. No formal hypothesis was carried out.

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.

Outcome measures

Outcome measures
Measure	Overall Study n=9 Participants At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Safety of FACTOR X: Number of Participants Experiencing Adverse Events	8 Participants

SECONDARY outcome

Timeframe: Baseline Visit and End of Study Visit, 30 minutes post-dose

Population: Plasma concentrations were obtained for FX:C for all 9 subjects at 30 minutes post dose at Visit 1 and Visit 5.

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.

Outcome measures

Outcome measures
Measure	Overall Study n=9 Participants At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Pharmacokinetics: FX:C Incremental Recovery	1.74 IU/dL Interval 1.66 to 1.82

Adverse Events

Human Coagulation FACTOR X

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Human Coagulation FACTOR X n=9 participants at risk At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Infections and infestations Influenza A	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Lower Respiratory Tract Infection	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.

Other adverse events

Other adverse events
Measure	Human Coagulation FACTOR X n=9 participants at risk At the Baseline Visit, eligible children received a bolus dose of 50 IU/kg FACTOR X. After the Baseline Visit, children were treated with FACTOR X prophylactically for a period of 6 months (26 weeks). A dosing regimen of 40-50 IU/kg twice a week was recommended, but was not mandatory. Each dose of FACTOR X was not to not exceed 60 IU/kg.
Investigations Parvovirus IgM, no symptoms	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Musculoskeletal and connective tissue disorders Pain In Back Of Leg	11.1% 1/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Blood and lymphatic system disorders Aneamia	33.3% 3/9 • Number of events 3 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
General disorders Intermittent Low Grade Fever	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Respiratory, thoracic and mediastinal disorders Cough	33.3% 3/9 • Number of events 4 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
General disorders High Temperature/Fever	33.3% 3/9 • Number of events 5 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Nervous system disorders Headache	22.2% 2/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Skin and subcutaneous tissue disorders Vitiligo	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Cold	33.3% 3/9 • Number of events 4 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Investigations Increased Temperature	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Viral Infection	22.2% 2/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Chest Infection	11.1% 1/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Respiratory, thoracic and mediastinal disorders Wheeze	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Metabolism and nutrition disorders Decreased Appetite	11.1% 1/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Positive MRSA Swab To Groin	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Musculoskeletal and connective tissue disorders Pain In Left Arm	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Influenza A	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Infections and infestations Coryzal	22.2% 2/9 • Number of events 2 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Nervous system disorders Lethargy	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.
Reproductive system and breast disorders Dysmenorrhoea	11.1% 1/9 • Number of events 1 • From signing of informed consent form until 28 days post-last dose. For subjects who had received FACTOR X on compassionate use prior to study entry, retrospective adverse events data was collected as part of medical history.

Additional Information

Head of Medical Affairs

Bio Products Laboratory Ltd

Phone: +44 20 8957 2200

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60