Trial Outcomes & Findings for Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease (NCT NCT01614574)
NCT ID: NCT01614574
Last Updated: 2021-06-28
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
6 participants
Primary outcome timeframe
Baseline to week 51
Results posted on
2021-06-28
Participant Flow
Participant milestones
| Measure |
VPRIV® (15-60 U/kg)
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease
Baseline characteristics by cohort
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Age, Continuous
|
20 Years
STANDARD_DEVIATION 10.62 • n=5 Participants
|
|
Age, Customized
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Customized
Between 18 and 65 years
|
2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
JAPAN
|
6 Participants
n=5 Participants
|
|
Hemoglobin Concentration
|
13.77 (g/dL)
STANDARD_DEVIATION 0.954 • n=5 Participants
|
|
Platelet Count
|
182.9 (x 10^9 platelets/L)
STANDARD_DEVIATION 32.05 • n=5 Participants
|
|
Normalized liver volume
|
1.92 (% of Body Weight)
STANDARD_DEVIATION 0.342 • n=5 Participants
|
|
Normalized spleen volume
|
0.39 (% of Body Weight)
STANDARD_DEVIATION 0.067 • n=5 Participants
|
|
Plasma chitotriosidase
|
1243.7 (nmol/mL/h)
STANDARD_DEVIATION 344.46 • n=5 Participants
|
|
CCL18 levels
|
134.8 (ng/mL)
STANDARD_DEVIATION 132.26 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Number of Severe Adverse Events (SAE)
|
1 events
|
PRIMARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Number of Treatment Emergent Adverse Events (TEAE)
|
31 events
|
PRIMARY outcome
Timeframe: Baseline to week51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Development of Anti-velaglucerase Alfa Antibody
Positive (Baseline)
|
0 participants
|
|
Development of Anti-velaglucerase Alfa Antibody
Negative (Baseline)
|
6 participants
|
|
Development of Anti-velaglucerase Alfa Antibody
Positive (Week 51)
|
0 participants
|
|
Development of Anti-velaglucerase Alfa Antibody
Negative (Week 51)
|
6 participants
|
PRIMARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Number of Infusion- Related Adverse Events
|
2 events
|
PRIMARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Number of Patients With Concomitant Medication
|
6 participants
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in Hemoglobin Concentration
|
0.05 (g/dL)
Standard Deviation 0.729
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in Platelet Count
|
13.8 (x 10^9 platelets/L)
Standard Deviation 35.75
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in Liver Volume, Normalized to Body Weight
|
0.05 (% of Body Weight)
Standard Deviation 0.148
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in Spleen Volume, Normalized to Body Weight
|
0.39 (% of Body Weight)
Standard Deviation 0.067
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in Plasma Chitotriosidase Levels
|
-47.3 (nmol/mL/h)
Standard Deviation 64.44
|
SECONDARY outcome
Timeframe: Baseline to week 51Outcome measures
| Measure |
VPRIV® (15-60 U/kg)
n=6 Participants
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Change From Baseline in CCL18 Levels
|
5.2 (ng/mL)
Standard Deviation 10.59
|
Adverse Events
VPRIV® (15-60 U/kg)
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
VPRIV® (15-60 U/kg)
n=6 participants at risk
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Eye disorders
RETINAL DETACHMENT
|
16.7%
1/6 • Number of events 1
|
Other adverse events
| Measure |
VPRIV® (15-60 U/kg)
n=6 participants at risk
Administered as an intravenous (IV) infusion over a 60 minute period.
|
|---|---|
|
Eye disorders
RETINOPATHY PROLIFERATIVE
|
16.7%
1/6 • Number of events 1
|
|
Eye disorders
VISUAL ACUITY REDUCED
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
CHEILITIS
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
ENTERITIS
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
NAUSEA
|
16.7%
1/6 • Number of events 1
|
|
Gastrointestinal disorders
VOMITING
|
16.7%
1/6 • Number of events 1
|
|
General disorders
OEDEMA PERIPHERAL
|
16.7%
1/6 • Number of events 1
|
|
General disorders
SUBMANDIBULAR MASS
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
GASTROENTERITIS
|
16.7%
1/6 • Number of events 2
|
|
Infections and infestations
HAND-FOOT-AND-MOUTH DISEASE
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
HORDEOLUM
|
16.7%
1/6 • Number of events 1
|
|
Infections and infestations
NASOPHARYNGITIS
|
100.0%
6/6 • Number of events 9
|
|
Infections and infestations
OTITIS MEDIA
|
16.7%
1/6 • Number of events 1
|
|
Injury, poisoning and procedural complications
CONTUSION
|
50.0%
3/6 • Number of events 3
|
|
Injury, poisoning and procedural complications
JOINT SPRAIN
|
16.7%
1/6 • Number of events 1
|
|
Injury, poisoning and procedural complications
LIMB INJURY
|
16.7%
1/6 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
33.3%
2/6 • Number of events 2
|
|
Nervous system disorders
HEADACHE
|
16.7%
1/6 • Number of events 1
|
|
Nervous system disorders
LOSS OF CONSCIOUSNESS
|
16.7%
1/6 • Number of events 1
|
|
Nervous system disorders
MYOCLONUS
|
16.7%
1/6 • Number of events 1
|
|
Psychiatric disorders
ADJUSTMENT DISORDER
|
16.7%
1/6 • Number of events 1
|
|
Reproductive system and breast disorders
MENSTRUATION IRREGULAR
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
|
16.7%
1/6 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
PHARYNGOLARYNGEAL PAIN
|
16.7%
1/6 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
TACHYPNOEA
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
ACNE
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
ALOPECIA
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
ECZEMA
|
16.7%
1/6 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
URTICARIA
|
16.7%
1/6 • Number of events 1
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Shire's agreements with investigators vary. All agreements provide Shire the right to embargo communications regarding trial results prior to public release for a period ≤180 days from the time submitted to Shire for review. Shire does not prohibit publication, but can require the removal of confidential information (excluding trial results) and can request postponement of a single-center publication until after disclosure of the trial's multi-center publication
- Publication restrictions are in place
Restriction type: OTHER