Trial Outcomes & Findings for A Study Investigating Treatment Factor X in People With Factor X Deficiency (NCT NCT00930176)
NCT ID: NCT00930176
Last Updated: 2014-12-12
Results Overview
Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg). Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
16 participants
Primary outcome timeframe
At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)
Results posted on
2014-12-12
Participant Flow
Participant milestones
| Measure |
Human Coagulation FACTOR X
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
Overall Study
STARTED
|
16
|
|
Overall Study
COMPLETED
|
15
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Human Coagulation FACTOR X
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
Overall Study
Death
|
1
|
Baseline Characteristics
A Study Investigating Treatment Factor X in People With Factor X Deficiency
Baseline characteristics by cohort
| Measure |
Human Coagulation FACTOR X
n=16 Participants
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
Age, Categorical
<=18 years
|
6 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
10 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
4 participants
n=5 Participants
|
|
Region of Enrollment
Turkey
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Germany
|
1 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At Baseline (during first 60 minutes post-dose) and at 6 months post-Baseline (during first 60 minutes post-dose)Incremental recovery is defined as the peak rise in plasma FX levels (IU/dL), as measured at 15, 30 and 60 minutes post-dose, divided by the dose (IU/kg). Value given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Outcome measures
| Measure |
Human Coagulation FACTOR X
n=16 Participants
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
FX:C Incremental Recovery
|
2.07 IU/dL per IU/kg
Geometric Coefficient of Variation 21.01
|
PRIMARY outcome
Timeframe: At Baseline and at 6 months post-BaselineValue given is the mean of 31 results: 16 for Baseline Visit + 15 for Repeat PK assessment
Outcome measures
| Measure |
Human Coagulation FACTOR X
n=16 Participants
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
FX:C Half-life
|
29.36 hours
Geometric Coefficient of Variation 22.89
|
Adverse Events
Human Coagulation FACTOR X
Serious events: 6 serious events
Other events: 15 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Human Coagulation FACTOR X
n=16 participants at risk
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
2/16 • Number of events 4 • From signing of informed consent form until 28 days post-last dose.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Reproductive system and breast disorders
Menorrhagia
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Muscle haemorrhage
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Pneumonia
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Nosocomial infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Tooth abscess
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Gastritis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Gastric ulcer helicobacter
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Nervous system disorders
Syncope
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
Other adverse events
| Measure |
Human Coagulation FACTOR X
n=16 participants at risk
Human Coagulation FACTOR X: Standard dose 25IU/kg to be administered at the Baseline Visit (1st PK assessment) and at the repeat PK assessment. Also administered to treat a bleed or to prevent a bleed.
|
|---|---|
|
Vascular disorders
Haematoma
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Vascular disorders
Hypotension
|
25.0%
4/16 • Number of events 4 • From signing of informed consent form until 28 days post-last dose.
|
|
Immune system disorders
Urticaria
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Fatigue
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Infusion site erytema
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Infusion site pain
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Malaise
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Non-cardiac chest pain
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Oedema peripheral
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Pyrexia
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Swelling
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Ulcer
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
General disorders
Vessel puncture site haematoma
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Psychiatric disorders
Insomnia
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Injury, poisoning and procedural complications
Contusion
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Injury, poisoning and procedural complications
Head injury
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Injury, poisoning and procedural complications
Joint injury
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Blood and lymphatic system disorders
Anaemia
|
12.5%
2/16 • Number of events 4 • From signing of informed consent form until 28 days post-last dose.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnoea
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Nervous system disorders
Dizziness
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Nervous system disorders
Headache
|
50.0%
8/16 • Number of events 14 • From signing of informed consent form until 28 days post-last dose.
|
|
Nervous system disorders
Migraine
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Constipation
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Diarrhoea
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Gingivitis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Nausea
|
12.5%
2/16 • Number of events 6 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Odynophagia
|
6.2%
1/16 • Number of events 3 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Toothache
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Gastrointestinal disorders
Vomiting
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Renal and urinary disorders
Nephrolithiasis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Skin and subcutaneous tissue disorders
Acne
|
6.2%
1/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Skin and subcutaneous tissue disorders
Pruritis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
31.2%
5/16 • Number of events 14 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
37.5%
6/16 • Number of events 10 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Groin pain
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
18.8%
3/16 • Number of events 3 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
6.2%
1/16 • Number of events 4 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Osteoarthritis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
37.5%
6/16 • Number of events 8 • From signing of informed consent form until 28 days post-last dose.
|
|
Metabolism and nutrition disorders
Fluid overload
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Bronchitis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Cystitis
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Fungal infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Nasopharyngitis
|
43.8%
7/16 • Number of events 11 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Oral infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Osteomyelitis
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Otitis media
|
12.5%
2/16 • Number of events 2 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Respiratory tract infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Tooth infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
4/16 • Number of events 9 • From signing of informed consent form until 28 days post-last dose.
|
|
Infections and infestations
Urinary tract infection
|
6.2%
1/16 • Number of events 1 • From signing of informed consent form until 28 days post-last dose.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60