Trial Outcomes & Findings for Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease (NCT NCT01685216)
NCT ID: NCT01685216
Last Updated: 2021-06-11
Results Overview
Hemoglobin concentration was measured as part of the hematology panel or measured separately when the hematology panel was not scheduled. Samples were measured by a central laboratory. Baseline is the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1/Day 1. A positive change from baseline indicates that hemoglobin concentration increased.
COMPLETED
PHASE1/PHASE2
7 participants
Baseline, Week 53 or end of study
2021-06-11
Participant Flow
Participant milestones
| Measure |
Velaglucerase Alfa
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Overall Study
STARTED
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7
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Overall Study
COMPLETED
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6
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Overall Study
NOT COMPLETED
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1
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Reasons for withdrawal
| Measure |
Velaglucerase Alfa
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Overall Study
Did not receive study drug
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1
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Baseline Characteristics
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
Baseline characteristics by cohort
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Age, Continuous
|
5.17 years
STANDARD_DEVIATION 4.446 • n=5 Participants
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Age, Customized
2 to 4 years
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4 Participants
n=5 Participants
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Age, Customized
5 to 17 years
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2 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
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1 Participants
n=5 Participants
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Sex: Female, Male
Male
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5 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Baseline, Week 53 or end of studyPopulation: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Hemoglobin concentration was measured as part of the hematology panel or measured separately when the hematology panel was not scheduled. Samples were measured by a central laboratory. Baseline is the modified baseline hemoglobin concentration, the average of the values from screening, baseline, and Week 1/Day 1. A positive change from baseline indicates that hemoglobin concentration increased.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration
|
2.15 g/dL
Standard Deviation 1.213
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SECONDARY outcome
Timeframe: Baseline, Week 53Population: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Platelet count was measured at a central laboratory as part of the hematology panel. Baseline is the modified baseline platelet count, the average of the values from screening, baseline and Week 1/Day 1. A positive change from baseline indicates that platelet count increased.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Change From Baseline to 12 Months (Week 53) in Platelet Count
|
136.6 platelets (x10^9)/L
Standard Deviation 51.48
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SECONDARY outcome
Timeframe: Baseline, Week 51 or end of studyPopulation: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Quantitative abdominal MRI was used to measure liver volume. If sedation was necessary to perform an MRI and the investigator deemed that this would be an unwarranted risk to the participant, liver volume could have been measured by ultrasound. Organ volume was measured by a single independent reviewer who was blinded to the participant identification and time point. The liver size relative to body weight was determined using the corresponding body weight measured at the same visit. Change in liver volume is presented as the normalized percentage of body weight. A negative change from baseline indicates that liver volume decreased.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI)
|
-30.12 percent change
Standard Deviation 10.366
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SECONDARY outcome
Timeframe: Baseline, Week 51Population: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Quantitative abdominal MRI was used to measure spleen volume. If sedation was necessary to perform an MRI and the investigator deemed that this would be an unwarranted risk to the participant, spleen volume could have been measured by ultrasound. Organ volume was measured by a single independent reviewer who was blinded to the participant identification and time point. The spleen size relative to body weight was determined using the corresponding body weight measured at the same visit. Change in spleen volume is presented as the normalized percentage of body weight. A negative change from baseline indicates that spleen volume decreased.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI)
|
-62.27 percent change
Standard Deviation 19.991
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SECONDARY outcome
Timeframe: Baseline, Weeks 13, 25, 37, and 53 or end of studyPopulation: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Neurological symptoms were evaluated at regular intervals during the study and assessed on an individualized basis by a limited, age- and developmental stage-appropriate neurological examination adapted to suit the status of each participant. It was preferred that each neurological examination be performed by a neurologist with experience in assessment of neurological symptoms in patients with Gaucher disease and, if possible, the same neurologist (or designee) who evaluated a given participant at baseline performed the neurological examinations scheduled for that participant during the treatment phase and at the end of study visit.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Number of Participants With Abnormal Neurological Status During The Study
|
6 participants
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SECONDARY outcome
Timeframe: 57 weeksPopulation: The Safety Analysis population, defined as all participants who received at least 1 study drug infusion (full or partial).
Adverse events (AEs) were monitored continuously throughout the study from the time the participant or participants parent/legal guardian signed the informed consent/assent (if applicable) until 30 days after the participant's last dose of study drug or at the end of study visit and/or until the event resolved or stabilized, or an outcome had been reached, whichever came first. Treatment-emergent adverse events (TEAEs) were defined as AEs which occurred on or after the time of the first infusion until 30 days after the participant's last study infusion. An infusion-related reaction is defined as an AE that 1) began either during or within 12 hours after the start of the infusion, and 2) was judged as possibly or probably related to study medication.
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Number of Participants Who Experienced a Treatment-Emergent Adverse Event
Any TEAE
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6 participants
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Number of Participants Who Experienced a Treatment-Emergent Adverse Event
Serious TEAE
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1 participants
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Number of Participants Who Experienced a Treatment-Emergent Adverse Event
Infusion-related TEAE
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1 participants
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SECONDARY outcome
Timeframe: Baseline, Weeks 13, 25, 37 and 53Population: The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Participants provided blood samples for measurement of anti-velaglucerase alfa antibodies in serum at baseline and approximately every 12 weeks during the treatment phase. Blood samples collected during the treatment phase were to be drawn prior to infusions. Analysis of anti-velaglucerase antibodies used a validated 3-tier immunoassay method (screening, confirmatory, and titer).
Outcome measures
| Measure |
Velaglucerase Alfa
n=6 Participants
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Week 13
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1 participants
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Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Week 25
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1 participants
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Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Week 37
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1 participants
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Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Baseline
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0 participants
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Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Week 53
|
1 participants
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Adverse Events
Velaglucerase Alfa
Serious adverse events
| Measure |
Velaglucerase Alfa
n=6 participants at risk
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Gastrointestinal disorders
Inguinal hernia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Other adverse events
| Measure |
Velaglucerase Alfa
n=6 participants at risk
Participants received an intravenous (IV) infusion of velaglucerase alfa at 60 U/kg, every other week for 1 year, then were followed for 1 month.
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|---|---|
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Blood and lymphatic system disorders
Lymphadenopathy
|
33.3%
2/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Blood and lymphatic system disorders
Anaemia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Blood and lymphatic system disorders
Iron deficiency anaemia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Blood and lymphatic system disorders
Neutropenia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Blood and lymphatic system disorders
Thrombocytopenia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Eye disorders
Conjunctivitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Eye disorders
Strabismus
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Gastrointestinal disorders
Diarrhoea
|
33.3%
2/6 • Number of events 5
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Gastrointestinal disorders
Gingival bleeding
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16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Gastrointestinal disorders
Inguinal hernia
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Gastrointestinal disorders
Dyspepsia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Gastrointestinal disorders
Vomiting
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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General disorders
Pyrexia
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66.7%
4/6 • Number of events 17
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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General disorders
Gait disturbance
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16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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General disorders
Influenza like illness
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16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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General disorders
Asthenia
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Bronchitis
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33.3%
2/6 • Number of events 4
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Acute tonsillitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Gastroenteritis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Hordeolum
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Nasopharyngitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Oral herpes
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Pharyngitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Postprocedural cellulitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Infections and infestations
Rhinitis
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Injury, poisoning and procedural complications
Excoriation
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16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Musculoskeletal and connective tissue disorders
Arthralgia
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16.7%
1/6 • Number of events 6
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Musculoskeletal and connective tissue disorders
Bone pain
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16.7%
1/6 • Number of events 6
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Musculoskeletal and connective tissue disorders
Myalgia
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16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Musculoskeletal and connective tissue disorders
Pain in extremity
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16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Musculoskeletal and connective tissue disorders
Synovial cyst
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Nervous system disorders
Headache
|
16.7%
1/6 • Number of events 8
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Nervous system disorders
Convulsion
|
16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Nervous system disorders
Fine motor delay
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Nervous system disorders
Gross motor delay
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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|
Nervous system disorders
Muscle spasticity
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • Number of events 3
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Skin and subcutaneous tissue disorders
Heat rash
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Injury, poisoning and procedural complications
Fall
|
16.7%
1/6 • Number of events 2
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Injury, poisoning and procedural complications
Chilblains
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
|
|
Injury, poisoning and procedural complications
Procedural pain
|
16.7%
1/6 • Number of events 1
Treatment-emergent adverse events are reported for the Safety population, defined as all participants who received at least 1 study drug infusion (full or partial).
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Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER