Factor X DeficiencyPhase 3NCT01721681

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

A Phase 3 study testing FACTOR X in people with Factor X Deficiency, run by Bio Products Laboratory at 3 sites in United Kingdom.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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. Read more about this study

Who is it for?: Ages Up to 11 years
Conditions the study is for: Factor X Deficiency
What is being tested?: FACTOR X
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 9 participants
Where: 3 sitesUnited Kingdom
Study dates: Apr 2015to Oct 2016
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Up to 11 years
All sexes
No healthy volunteers
Child

Who may be able to join

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.

Who cannot join

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.

Trial locations and contacts

3 sites in 1 country.

United Kingdom3 sites

Addenbrookes Hospital
Cambridge, CB2 0QQ
Great Ormond Street Hospital
London, WC1N 3JH
Sheffield Children's Hospital
Sheffield, S10 2TH

Other trials in United Kingdom

About this study

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

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 6 months (26 weeks).

Conditions: Factor X Deficiency
Being tested: FACTOR X
Medical subject headings: Factor X Deficiency

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

FACTOR XBiological

Study groups in NCT01721681
Study group	Role	What they receive
FACTOR X	Experimental	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. Receives: Biological: FACTOR X

What is being measured

The primary outcome is the main question the study is designed to answer.

The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 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.
Measured: 6 months

Secondary outcomes (2)

Safety of FACTOR X: Number of Participants Experiencing Adverse Events
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.
Measured: 6 months
Pharmacokinetics: FX:C Incremental Recovery
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.
Measured: Baseline Visit and End of Study Visit, 30 minutes post-dose

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Apr 2015Oct 2016

First posted6 Nov 2012 (estimated)
Study startApr 2015
Primary completionOct 2016
Study completionOct 2016

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Factor X DeficiencyStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 9 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01721681
Sponsor's protocol ID: Ten 02
Other IDs: 2012-003093-98
Status last verified: Mar 2018
First submitted: 25 Oct 2012
Last updated: 2 Apr 2018
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
A Database For The Collection Of Clinical, Laboratory, And Treatment Data From Patients With Factor X Deficiency To Define The Natural History Of The Disease And Improve Patient Management
Factor X Deficiency
Active, not recruitingObservational study
X-linked Hypophosphatemia Disease Monitoring Program
X-linked Hypophosphatemia, Hypophosphatemic Rickets
RecruitingObservational study
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Hematologic Disorder, Bleeding Disorder
RecruitingPhase 1
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
Healthy Participants, Hemophilia A
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

All Factor X Deficiency trials

Study at a glance

From the registry record, last updated by the sponsor on 2 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.

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
ClinicalTrials.gov ID: NCT01721681
Acronym / sponsor ID: Ten 02
Conditions: Factor X Deficiency
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 9 participants (actual)
Lead sponsor: Bio Products LaboratoryUniversity, hospital or other
Study start: Apr 2015
Primary completion: Oct 2016 (actual)
Study completion: Oct 2016 (actual)
First posted: 6 Nov 2012 (estimated)
Results: Posted 7 Mar 2018

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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 6 months (26 weeks).

Study design

Primary purpose: Prevention
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
FACTOR X	Experimental	Biological: FACTOR XAt 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.

Who may qualify

Ages: Up to 11 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (3 listed)

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.

Key exclusion criteria (6 listed)

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.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
FACTOR X	Biological	FACTOR X

Primary outcome

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.

2 secondary outcomes

Safety of FACTOR X: Number of Participants Experiencing Adverse Events
Time frame: 6 months
Pharmacokinetics: FX:C Incremental Recovery
Time frame: Baseline Visit and End of Study Visit, 30 minutes post-dose

Results and safety

Results posted to the registry on 7 Mar 2018, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Overall Study	9	9

Primary outcome: The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months

Time frame: 6 months. Count of participants. Unit: Participants

Measure	Overall Study n = 9
Value	9

2 secondary outcomes are reported in the full results.

Adverse events

Time frame: 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 reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Human Coagulation FACTOR X	1 of 9 (11.1%)	3 of 9 (33.3%)	Not reported

Limitations and caveats (from the sponsor): 2 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 even though there were 11 treatment cycles

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2018.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.

Related trials for Factor X Deficiency

RecruitingObservational study
A Database For The Collection Of Clinical, Laboratory, And Treatment Data From Patients With Factor X Deficiency To Define The Natural History Of The Disease And Improve Patient Management
Factor X Deficiency
Active, not recruitingObservational study
X-linked Hypophosphatemia Disease Monitoring Program
X-linked Hypophosphatemia, Hypophosphatemic Rickets
RecruitingObservational study
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Hematologic Disorder, Bleeding Disorder
RecruitingPhase 1
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
Healthy Participants, Hemophilia A
Active, not recruitingObservational study
Early Check: Expanded Screening in Newborns
Spinal Muscular Atrophy, Fragile X Syndrome

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Ri Liesner, Dr principal investigator

Great Ormond Street Hospital

3 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing FACTOR X in people with Factor X Deficiency, run by Bio Products Laboratory at 3 sites in United Kingdom.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 2 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 7 Mar 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months (time frame: 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01721681.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Apr 2018.