Trial Outcomes & Findings for BAY14-2222 Prophylaxis and Joint Function Improvement (Adults) (NCT NCT00586521)
NCT ID: NCT00586521
Last Updated: 2014-11-03
Results Overview
Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
20 participants
Primary outcome timeframe
Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)
Results posted on
2014-11-03
Participant Flow
Patients were recruited at Haemophilia treatment centers
All subjects who met eligibility criteria on screening were enrolled into the study and began the on-demand treatment period.
Participant milestones
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
On Demand Treatment (Months 1-6)
STARTED
|
20
|
|
On Demand Treatment (Months 1-6)
COMPLETED
|
19
|
|
On Demand Treatment (Months 1-6)
NOT COMPLETED
|
1
|
|
Prophylactic Treatment (Months 8-13)
STARTED
|
19
|
|
Prophylactic Treatment (Months 8-13)
COMPLETED
|
19
|
|
Prophylactic Treatment (Months 8-13)
NOT COMPLETED
|
0
|
Reasons for withdrawal
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
On Demand Treatment (Months 1-6)
Withdrawal by Subject
|
1
|
Baseline Characteristics
BAY14-2222 Prophylaxis and Joint Function Improvement (Adults)
Baseline characteristics by cohort
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 Participants
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
20 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Age, Continuous
|
36.4 years
STANDARD_DEVIATION 3.5 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=5 Participants
|
|
Target joints presence
Number participants with target joints presence
|
16 participants
n=5 Participants
|
|
Target joints presence
Number participants without target joints presence
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)Population: intent-to-treat population
Number of joint bleeds during Months 8-13 compared to number of joint bleeds during Months 1-6
Outcome measures
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 Participants
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Number of Joint Bleeds
Months 1-6 (on-demand)
|
18.5 Number of joint bleeds
Standard Deviation 11.6
|
|
Number of Joint Bleeds
Months 8-13 (prophylactic)
|
1.5 Number of joint bleeds
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: Months 1-6 (on-demand treatment) and 8-13 (prophylactic treatment)Population: intention-to-treat
Mean number of all bleeds during Months 8-13 (prophylactic) compared to mean number of all bleeds during Months 1-6 (on-demand)
Outcome measures
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 Participants
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Number of All Bleeds
Months 1-6 (on-demand)
|
23.7 All bleeds
Standard Deviation 13.3
|
|
Number of All Bleeds
Month 8-13 (prophylactic)
|
1.9 All bleeds
Standard Deviation 3.3
|
SECONDARY outcome
Timeframe: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)Population: intent-to-treat
Total score with a range of 0-100, evaluating ankle, knee and elbow, 0 indicates normal function, higher values indicate joint damage
Outcome measures
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 Participants
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score
Month 6 (on-demand)
|
25.3 Gilbert score (0-100)
Standard Deviation 11.7
|
|
Physical Assessment Compared to On-demand Treatment as Determined by the Gilbert Score
Month 13 (prophylactic)
|
19.8 Gilbert score (0-100)
Standard Deviation 11.7
|
SECONDARY outcome
Timeframe: Month 13 (end of prophylactic treatment) and Month 6 (end of on-demand treatment)Population: intent-to-treat-population
Total transformed score with a range of 0-100, higher values indicate better outcome. 41 items in 6 domains: physical functioning; role functioning; worry; consequences; positive affect; treatment concern.
Outcome measures
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 Participants
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire
Month 6 (on-demand)
|
73.4 Transformed score
Standard Deviation 17.4
|
|
Quality of Life Compared to On-demand Treatment as Measured by the Haemo-QoL A Questionnaire
Month 13 (prophylactic)
|
75.9 Transformed score
Standard Deviation 16.1
|
Adverse Events
rFVIII-FS (Octocog-alfa), (Kogenate FS)
Serious events: 2 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 participants at risk
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Injury, poisoning and procedural complications
Carbon monoxide poisoning
|
5.0%
1/20
|
|
Injury, poisoning and procedural complications
Femur fracture
|
5.0%
1/20
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
5.0%
1/20
|
|
Metabolism and nutrition disorders
Dehydration
|
5.0%
1/20
|
|
Social circumstances
Alcohol use
|
5.0%
1/20
|
Other adverse events
| Measure |
rFVIII-FS (Octocog-alfa), (Kogenate FS)
n=20 participants at risk
On-demand treatment was to follow the same treatment pattern the subject was using before entering the study. While on prophylactic treatment, all subjects were to be treated at a dose of 20-40 IU/kg, 3 times per week at a stable dose.
|
|---|---|
|
Infections and infestations
Lower respiratory tract infection
|
15.0%
3/20
|
|
Infections and infestations
Influenza
|
10.0%
2/20
|
|
Infections and infestations
Bronchitis
|
5.0%
1/20
|
|
Infections and infestations
Gastroenteritis
|
5.0%
1/20
|
|
Infections and infestations
Gingival infection
|
5.0%
1/20
|
|
Infections and infestations
Nasopharyngitis
|
5.0%
1/20
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
5.0%
1/20
|
|
Injury, poisoning and procedural complications
Traumatic haemorrhage
|
10.0%
2/20
|
|
Injury, poisoning and procedural complications
Limb injury
|
5.0%
1/20
|
|
Injury, poisoning and procedural complications
Meniscus lesion
|
5.0%
1/20
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
10.0%
2/20
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
5.0%
1/20
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
5.0%
1/20
|
|
Musculoskeletal and connective tissue disorders
Synovitis
|
5.0%
1/20
|
|
Nervous system disorders
Dysgeusia
|
5.0%
1/20
|
|
Nervous system disorders
Headache
|
5.0%
1/20
|
|
Nervous system disorders
Sinus headache
|
5.0%
1/20
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.0%
1/20
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
5.0%
1/20
|
|
Skin and subcutaneous tissue disorders
Dermatitis
|
5.0%
1/20
|
|
Skin and subcutaneous tissue disorders
Hyperhidrosis
|
5.0%
1/20
|
|
Ear and labyrinth disorders
Ear discomfort
|
5.0%
1/20
|
|
Gastrointestinal disorders
Diarrhoea
|
5.0%
1/20
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20
|
|
General disorders
Chest pain
|
5.0%
1/20
|
|
Psychiatric disorders
Anxiety
|
5.0%
1/20
|
|
Renal and urinary disorders
Renal colic
|
5.0%
1/20
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60