A Phase 3 interventional study of FACTOR X in Factor X Deficiency, sponsored by Bio Products Laboratory. Completed at 3 sites in United Kingdom. Open to participants aged Up to 11 Years. Per ClinicalTrials.gov, last updated 2018-04-02.
Sponsored by Bio Products Laboratory · Phase 3, Interventional, and Prevention
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:
Bio Products Laboratory is the lead sponsor of 27 studies on the registry; none are open to participants now.
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Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Biological: FACTOR X
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.
Time frame: 6 months
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.
Time frame: 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.
Time frame: Baseline Visit and End of Study Visit, 30 minutes post-dose
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.
| Milestone | Overall Study |
|---|---|
| Started | 9 |
| Completed | 9 |
| Not completed | 0 |
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.
| Participants | Overall Study |
|---|---|
| The Number of Participants With Excellent Reduction in Bleeding When Given FACTOR X as Routine Prophylaxis Over 6 Months | 9 |
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.
| Participants | Overall Study |
|---|---|
| Safety of FACTOR X: Number of Participants Experiencing Adverse Events | 8 |
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.
| IU/dL | Overall Study |
|---|---|
| Pharmacokinetics: FX:C Incremental Recovery | 1.74 (1.66 to 1.82) |
Collected over 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.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Human Coagulation FACTOR X | — | 1/9 (11.1%) | 3/9 (33.3%) |
| Event | Human Coagulation FACTOR X |
|---|---|
| Lower Respiratory Tract InfectionInfections and infestations | 1/9 |
| Influenza AInfections and infestations | 1/9 |
| Event | Human Coagulation FACTOR X |
|---|---|
| AneamiaBlood and lymphatic system disorders | 3/9 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/9 |
| High Temperature/FeverGeneral disorders | 3/9 |
| ColdInfections and infestations | 3/9 |
| HeadacheNervous system disorders | 2/9 |
| Viral InfectionInfections and infestations | 2/9 |
| CoryzalInfections and infestations | 2/9 |
| Parvovirus IgM, no symptomsInvestigations | 1/9 |
| Pain In Back Of LegMusculoskeletal and connective tissue disorders | 1/9 |
| Intermittent Low Grade FeverGeneral disorders | 1/9 |
| Age, Categorical(Participants) | Human Coagulation FACTOR X |
|---|---|
| <=18 years | 9 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Human Coagulation FACTOR X |
|---|---|
| Female | 5 |
| Male | 4 |
| Region of Enrollment(participants) | Human Coagulation FACTOR X |
|---|---|
| United Kingdom | 9 |
The record gives a start of Apr 2015, a primary completion of Oct 2016 and a completion of Oct 2016. It does not say whether these dates are actual or estimated.
Results were first posted in Mar 2018, 1 year 5 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Mar 2018. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Factor X
Factor X Deficiency
Bio Products Laboratory