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Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

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ClinicalTrials.gov Identifier: NCT01685216
Recruitment Status : Completed
First Posted : September 14, 2012
Results First Posted : October 30, 2015
Last Update Posted : October 30, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gaucher Disease, Type 3
Intervention Biological: velaglucerase alfa
Enrollment 7
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 7
Completed 6
Not Completed 1
Reason Not Completed
Did not receive study drug             1
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants
5.17  (4.446)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants
2 to 4 years 4
5 to 17 years 2
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
1
  16.7%
Male
5
  83.3%
1.Primary Outcome
Title Change From Baseline to 12 Months (Week 53) in Hemoglobin Concentration
Hide Description 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.
Time Frame Baseline, Week 53 or end of study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: g/dL
2.15  (1.213)
2.Secondary Outcome
Title Change From Baseline to 12 Months (Week 53) in Platelet Count
Hide Description 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.
Time Frame Baseline, Week 53
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: platelets (x10^9)/L
136.6  (51.48)
3.Secondary Outcome
Title Percent Change From Baseline to 12 Months (Week 51) in Normalized Liver Volume Measured Using Magnetic Resonance Imaging (MRI)
Hide Description 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.
Time Frame Baseline, Week 51 or end of study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percent change
-30.12  (10.366)
4.Secondary Outcome
Title Percent Change From Baseline to 12 Months (Week 51) in Normalized Spleen Volume Measured Using Magnetic Resonance Imaging (MRI)
Hide Description 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.
Time Frame Baseline, Week 51
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percent change
-62.27  (19.991)
5.Secondary Outcome
Title Number of Participants With Abnormal Neurological Status During The Study
Hide Description 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.
Time Frame Baseline, Weeks 13, 25, 37, and 53 or end of study
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
6
6.Secondary Outcome
Title Number of Participants Who Experienced a Treatment-Emergent Adverse Event
Hide Description 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.
Time Frame 57 weeks
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Safety Analysis population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
Any TEAE 6
Serious TEAE 1
Infusion-related TEAE 1
7.Secondary Outcome
Title Number of Participants Who Developed Anti-Velaglucerase Alfa Antibodies During The Study
Hide Description 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).
Time Frame Baseline, Weeks 13, 25, 37 and 53
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The Intent-to-Treat population, defined as all participants who received at least 1 study drug infusion (full or partial).
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
Baseline 0
Week 13 1
Week 25 1
Week 37 1
Week 53 1
Time Frame [Not Specified]
Adverse Event Reporting Description 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).
 
Arm/Group Title Velaglucerase Alfa
Hide Arm/Group Description 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.
All-Cause Mortality
Velaglucerase Alfa
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Velaglucerase Alfa
Affected / at Risk (%) # Events
Total   1/6 (16.67%)    
Gastrointestinal disorders   
Inguinal hernia  1  1/6 (16.67%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (14.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Velaglucerase Alfa
Affected / at Risk (%) # Events
Total   6/6 (100.00%)    
Blood and lymphatic system disorders   
Lymphadenopathy  1  2/6 (33.33%)  2
Anaemia  1  1/6 (16.67%)  1
Iron deficiency anaemia  1  1/6 (16.67%)  1
Neutropenia  1  1/6 (16.67%)  1
Thrombocytopenia  1  1/6 (16.67%)  1
Eye disorders   
Conjunctivitis  1  1/6 (16.67%)  1
Strabismus  1  1/6 (16.67%)  1
Gastrointestinal disorders   
Diarrhoea  1  2/6 (33.33%)  5
Gingival bleeding  1  1/6 (16.67%)  2
Inguinal hernia  1  1/6 (16.67%)  1
Dyspepsia  1  1/6 (16.67%)  1
Vomiting  1  1/6 (16.67%)  1
General disorders   
Pyrexia  1  4/6 (66.67%)  17
Gait disturbance  1  1/6 (16.67%)  2
Influenza like illness  1  1/6 (16.67%)  2
Asthenia  1  1/6 (16.67%)  1
Infections and infestations   
Bronchitis  1  2/6 (33.33%)  4
Acute tonsillitis  1  1/6 (16.67%)  1
Gastroenteritis  1  1/6 (16.67%)  1
Hordeolum  1  1/6 (16.67%)  1
Nasopharyngitis  1  1/6 (16.67%)  1
Oral herpes  1  1/6 (16.67%)  1
Pharyngitis  1  1/6 (16.67%)  1
Postprocedural cellulitis  1  1/6 (16.67%)  1
Rhinitis  1  1/6 (16.67%)  1
Injury, poisoning and procedural complications   
Excoriation  1  1/6 (16.67%)  2
Fall  1  1/6 (16.67%)  2
Chilblains  1  1/6 (16.67%)  1
Procedural pain  1  1/6 (16.67%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/6 (16.67%)  6
Bone pain  1  1/6 (16.67%)  6
Myalgia  1  1/6 (16.67%)  2
Musculoskeletal pain  1  1/6 (16.67%)  1
Pain in extremity  1  1/6 (16.67%)  1
Synovial cyst  1  1/6 (16.67%)  1
Nervous system disorders   
Headache  1  1/6 (16.67%)  8
Convulsion  1  1/6 (16.67%)  2
Fine motor delay  1  1/6 (16.67%)  1
Gross motor delay  1  1/6 (16.67%)  1
Muscle spasticity  1  1/6 (16.67%)  1
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/6 (33.33%)  3
Asthma  1  1/6 (16.67%)  2
Productive cough  1  1/6 (16.67%)  2
Rhinorrhoea  1  1/6 (16.67%)  1
Wheezing  1  1/6 (16.67%)  1
Skin and subcutaneous tissue disorders   
Dermatitis allergic  1  1/6 (16.67%)  1
Heat rash  1  1/6 (16.67%)  1
Rash papular  1  1/6 (16.67%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (14.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Study Physician
Organization: Shire Development LLC
Phone: +1 866 842 5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01685216     History of Changes
Other Study ID Numbers: HGT-GCB-068
First Submitted: September 10, 2012
First Posted: September 14, 2012
Results First Submitted: October 1, 2015
Results First Posted: October 30, 2015
Last Update Posted: October 30, 2015