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Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

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ClinicalTrials.gov Identifier: NCT00478647
Recruitment Status : Completed
First Posted : May 25, 2007
Results First Posted : September 2, 2010
Last Update Posted : April 9, 2015
Sponsor:
Collaborator:
Shire Human Genetic Therapies, Inc.
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gaucher Disease, Type 1
Intervention Biological: GA-GCB (velaglucerase alfa)
Enrollment 40

Recruitment Details The first participant was enrolled on 25 July 2007 and the last participant completed on 26 June 2009. Participants received the same dose of velaglucerase alfa (GA-GCB) as their previous dose of imiglucerase (range- </= 60 Unit per kilogram (U/kg) - >/=15 U/kg) every other week via intravenous infusion.
Pre-assignment Details Participant at least 2 years old with documented diagnosis of type 1 Gaucher disease.Consistent treatment(every other week at a dose ≤/= 60 U/kg and ≥/= 15 U/kg) with imiglucerase for a minimum of 30 consecutive months;same dose during the 6 months prior to study enrollment.Minor dosing interval variance was allowed per standard clinical practice.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description 15-60 U/kg, every other week via intravenous infusion
Period Title: Overall Study
Started 40
Completed 38 [1]
Not Completed 2
Reason Not Completed
Adverse Event             1
Withdrawal by Subject             1
[1]
Intent-to-Treat (ITT)=participants who have received at least 1 full or partial dose of study drug.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description 15-60 U/kg, every other week via intravenous infusion
Overall Number of Baseline Participants 40
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
<=18 years
9
  22.5%
Between 18 and 65 years
27
  67.5%
>=65 years
4
  10.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 40 participants
35.6  (18.37)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 40 participants
Female
22
  55.0%
Male
18
  45.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 40 participants
United States 22
Spain 1
Poland 5
Israel 9
United Kingdom 3
Baseline hemoglobin concentration  
Median (Full Range)
Unit of measure:  Gram per deciliter (g/dL)
Number Analyzed 40 participants
13.775
(10.40 to 16.45)
Baseline liver volume  
Median (Full Range)
Unit of measure:  Percent (%) body weight
Number Analyzed 40 participants
1.90
(1.4 to 3.9)
Baseline platelet count  
Median (Full Range)
Unit of measure:  10^9 per liter (10^9/L)
Number Analyzed 40 participants
162.00
(29.0 to 399.0)
Baseline spleen volume  
Median (Full Range)
Unit of measure:  Percent (%) body weight
Number Analyzed 40 participants
0.50
(0.2 to 3.2)
1.Primary Outcome
Title Participants Who Experienced at Least One Adverse Event
Hide Description

Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies.

Refer to Adverse event section for further details.

Time Frame Week 53
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Safety population included subjects who have received at least 1 full or partial dose of study drug.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description:
15-60 U/kg, every other week via intravenous infusion
Overall Number of Participants Analyzed 40
Measure Type: Number
Unit of Measure: participants
34
2.Secondary Outcome
Title Change From Baseline to Week 53 in Hemoglobin Concentration
Hide Description [Not Specified]
Time Frame Week 53
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description:
15-60 U/kg, every other week via intravenous infusion
Overall Number of Participants Analyzed 40
Mean (90% Confidence Interval)
Unit of Measure: g/dL
-0.101
(-0.272 to 0.070)
3.Secondary Outcome
Title Percent Change From Baseline to Week 53 in Platelet Count
Hide Description [Not Specified]
Time Frame Week 53
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description:
15-60 U/kg, every other week via intravenous infusion
Overall Number of Participants Analyzed 40
Mean (90% Confidence Interval)
Unit of Measure: percent (%) change
7.04
(0.54 to 13.53)
4.Secondary Outcome
Title Percent Change From Baseline to Week 51 in Normalized Liver Volume
Hide Description Liver volume has been normalized for percentage (%) of body weight. Liver size relative to body weight= (Liver volume [cc]/Body weight [kg])*100
Time Frame Week 51
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description:
15-60 U/kg, every other week via intravenous infusion
Overall Number of Participants Analyzed 40
Mean (90% Confidence Interval)
Unit of Measure: Percent (%) change
-0.03
(-2.62 to 2.56)
5.Secondary Outcome
Title Percent Change From Baseline to Week 51 in Normalized Spleen Volume
Hide Description Spleen volume has been normalized for percentage (%) of body weight. Spleen size relative to body weight= (Spleen volume [cc]/Body weight [kg])*100
Time Frame Week 51
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
ITT population. Four splenectomized participants were excluded.
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description:
15-60 U/kg, every other week via intravenous infusion
Overall Number of Participants Analyzed 36
Mean (90% Confidence Interval)
Unit of Measure: Percent (%) change
-5.56
(-10.77 to -0.35)
Time Frame Time of informed consent until 30 days after the last dose and/or until the event was resolved/stabilized or outcome was reached, whichever came first. Participants who discontinued/withdrew prior to Week 53, and followed up to 30 days after last dose.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title GA-GCB (Velaglucerase Alfa)
Hide Arm/Group Description 15-60 U/kg, every other week via intravenous infusion
All-Cause Mortality
GA-GCB (Velaglucerase Alfa)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
GA-GCB (Velaglucerase Alfa)
Affected / at Risk (%) # Events
Total   4/40 (10.00%)    
Immune system disorders   
Anaphylactoid reaction  1  1/40 (2.50%)  1
Drug hypersensitivity  1  1/40 (2.50%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/40 (2.50%)  1
Skin and subcutaneous tissue disorders   
Urticaria  1  1/40 (2.50%)  1
Swelling face  1  1/40 (2.50%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
GA-GCB (Velaglucerase Alfa)
Affected / at Risk (%) # Events
Total   31/40 (77.50%)    
Ear and labyrinth disorders   
Ear pain  1  3/40 (7.50%)  3
Eye disorders   
Conjunctivitis  1  2/40 (5.00%)  2
Gastrointestinal disorders   
Abdominal pain upper  1  5/40 (12.50%)  11
Constipation  1  2/40 (5.00%)  2
Diarrhoea  1  4/40 (10.00%)  4
Nausea  1  4/40 (10.00%)  4
Toothache  1  2/40 (5.00%)  3
Vomiting  1  2/40 (5.00%)  2
General disorders   
Fatigue  1  5/40 (12.50%)  13
Influenza like illness  1  4/40 (10.00%)  5
Malaise  1  2/40 (5.00%)  33
Non-Cardiac chest pain  1  2/40 (5.00%)  3
Pyrexia  1  5/40 (12.50%)  7
Infections and infestations   
Bronchitis  1  3/40 (7.50%)  4
Influenza  1  4/40 (10.00%)  4
Nasopharyngitis  1  8/40 (20.00%)  10
Pharyngitis  1  2/40 (5.00%)  2
Rhinovirus infection  1  2/40 (5.00%)  2
Upper respiratory tract infection  1  5/40 (12.50%)  7
Urinary tract infection  1  3/40 (7.50%)  3
Injury, poisoning and procedural complications   
Fall  1  2/40 (5.00%)  2
Procedural pain  1  2/40 (5.00%)  2
Investigations   
Activated partial thromboplastin time prolonged  1  2/40 (5.00%)  2
Blood glucose increased  1  3/40 (7.50%)  3
Musculoskeletal and connective tissue disorders   
Arthralgia  1  8/40 (20.00%)  14
Back pain  1  7/40 (17.50%)  10
Muscle spasms  1  2/40 (5.00%)  2
Myalgia  1  6/40 (15.00%)  7
Pain in extremity  1  5/40 (12.50%)  6
Rotator cuff syndrome  1  2/40 (5.00%)  3
Nervous system disorders   
Dizziness  1  3/40 (7.50%)  3
Headache  1  12/40 (30.00%)  29
Paraesthesia  1  3/40 (7.50%)  3
Psychiatric disorders   
Anxiety  1  2/40 (5.00%)  2
Renal and urinary disorders   
Proteinuria  1  2/40 (5.00%)  2
Respiratory, thoracic and mediastinal disorders   
Cough  1  6/40 (15.00%)  6
Epistaxis  1  3/40 (7.50%)  7
Nasal congestion  1  3/40 (7.50%)  6
Pharyngolaryngeal pain  1  6/40 (15.00%)  8
Sinus congestion  1  2/40 (5.00%)  2
Vascular disorders   
Hypertension  1  3/40 (7.50%)  15
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (9.0)
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.
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.
Results Point of Contact
Name/Title: Tiffany Crump
Organization: Shire HGT
Phone: 484-595-8850
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT00478647     History of Changes
Other Study ID Numbers: TKT034
2006-006304-11 ( EudraCT Number )
First Submitted: May 23, 2007
First Posted: May 25, 2007
Results First Submitted: August 4, 2010
Results First Posted: September 2, 2010
Last Update Posted: April 9, 2015