An Open-Label Extension Study of GA-GCB ERT in Patients W... | NCT00635427

ID	Type	Description	Link
2008-001965-27	EudraCT Number

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	Experimental	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)	Biological: VPRIV®
VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039)	Experimental	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044	Biological: VPRIV®
VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034)	Experimental	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034	Biological: VPRIV®

Interventions

Name	Type	Description	Arm Group Labels	Other Names
VPRIV®	Biological	Intravenous infusion, every other week (EOW)	VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034) VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039)

Measure	Description	Time Frame
Overall Summary of Treatment Emergent Adverse Events	Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.	Baseline to termination of study

Measure	Description	Time Frame
Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group		Baseline to 24 months
Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group		Baseline to 24 months

Eligibility Module

Name	Affiliation	Role
Study Director	Takeda	Study Director

Facility	Status	City	State	ZIP	Country	Contacts
Los Angeles Medical Center		Los Angeles	California	90027	United States
Children's Hospital Oakland

PubMed Identifier	Type	Citation	Retractions
27839979	Derived	Zimran A, Elstein D, Gonzalez DE, Lukina EA, Qin Y, Dinh Q, Turkia HB. Treatment-naive Gaucher disease patients achieve therapeutic goals and normalization with velaglucerase alfa by 4years in phase 3 trials. Blood Cells Mol Dis. 2018 Feb;68:153-159. doi: 10.1016/j.bcmd.2016.10.007. Epub 2016 Oct 21.
26043810	Derived

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg) -TKT032)	VPRIV 45 U/kg, IV, every other week (EOW) for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044
FG001	VPRIV 60 U/kg (Parent Study- VPRIV (60 U/kg)-TKT032)

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Age at the time the informed consent was obtained in the core study.

Mean

Standard Deviation

Years

Title

Denominators

Categories

Title	Measurements
	BG00032.5± 16.75 BG00122± 11.08 BG00232.9± 16.14 BG003

Age, Customized

Age at the time the informed consent was obtained in the core study.

Count of Participants

Participants

Title

Denominators

Categories

At least 18 years

Title	Measurements
	BG0002 BG0013 BG002

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG0007 BG0016 BG002

Splenectomy status

Count of Participants

Participants

Title

Denominators

Categories

Yes

Title	Measurements
	BG0000 BG0010 BG002

Baseline hemoglobin concentration per treatment group

Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039).

Mean

Full Range

gram per deciliter

Title

Denominators

Categories

Title	Measurements
	BG00010.68(8.5 to 12.9) BG001

Baseline platelet counts per treatment group

Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039).

Mean

Full Range

x10^9/L

Title

Denominators

Categories

Title	Measurements
	BG00069.3(13 to 146) BG00179.4

Baseline liver volume per treatment group

Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HGT-GCB-039). Normal liver volume was defined as 2.5 percent of body weight.

Mean

Full Range

Percent (%) body weight

Title

Denominators

Categories

Title	Measurements
	BG0001.64(1.1 to 2.9) BG001

Baseline Spleen volume per treatment group

Baseline was defined as data collected prior to the first dose in the core study (TKT032, TKT034, and HT-GCB-039). Normal spleen volume is defined as 0.2 percentage of body weight.

Mean

Full Range

Multiple of Normal

Title

Denominators

Categories

Title	Measurements
	BG00023.08(4.8 to 65.1) BG001

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Overall Summary of Treatment Emergent Adverse Events

Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.

All participants who received at least 1 infusion (full or partial) of study drug were evaluated for safety (ie, were included in the safety population). There were 95 participants in the safety population.

Posted

Number

Participants

Baseline to termination of study

ID	Title	Description
OG000	VPRIV 60 U/kg(Parent Study VPRIV(45 or 60 U/kg) TKT032,GCB039)	This is the overall velaglucerase alfa group consisting of the following population: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
OG001	VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg)HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched to 60 U/kg VPRIV in HGT-GCB-044
OG002	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044

Units	Counts
Participants	OG00041 OG00116 OG00238

Title

Denominators

Categories

Experienced no AEs

Title	Measurements
	OG0003 OG0011 OG0023

Experienced at least 1 AE

Secondary

Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group

Posted

Mean

95% Confidence Interval

(g/dL)

Baseline to 24 months

ID	Title	Description
OG000	VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes participants from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
OG001	VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched to 60 U/kg VPRIV in HGT-GCB-044
OG002	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044

Units	Counts

Secondary

Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group

Posted

Mean

95% Confidence Interval

(10^9/L)

Baseline to 24 months

ID	Title	Description
OG000	VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes participants from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
OG001	VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched to 60 U/kg VPRIV in HGT-GCB-044
OG002	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044

Units	Counts

Secondary

Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group

Posted

Mean

95% Confidence Interval

% Body weight

Baseline to 24 months

ID	Title	Description
OG000	VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes participants from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
OG001	VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched to 60 U/kg VPRIV in HGT-GCB-044
OG002	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044

Units	Counts

Secondary

Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group

Posted

Mean

95% Confidence Interval

Precent (%) change

Baseline to 24 months

ID	Title	Description
OG000	VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes participants from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
OG001	VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched to 60 U/kg VPRIV in HGT-GCB-044
OG002	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044

Units

Serious Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Splenomegaly	Blood and lymphatic system disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Abdominal hernia	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Umbilical hernia	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Non-cardiac chest pain	General disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk
Bronchopneumonia	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Pyelonephritis acute	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Respiratory tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Urinary tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0002 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Lower limb fracture	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Post procedural haematoma	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0012 events1 affected41 at risk EG0020 events0 affected16 at risk
Lumbar spinal stenosis	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Osteonecrosis	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0012 events2 affected41 at risk EG0023 events2 affected16 at risk
Benign renal neoplasm	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Convulsion	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Syncope	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Abortion	Pregnancy, puerperium and perinatal conditions	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Oligohydramnios	Pregnancy, puerperium and perinatal conditions	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Renal colic	Renal and urinary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Phlebitis	Vascular disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Abdominal pain	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0013 events3 affected41 at risk EG0021 events1 affected16 at risk
Abdominal pain upper	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0004 events3 affected38 at risk EG0016 events4 affected41 at risk EG0022 events2 affected16 at risk
Diarrhoea	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0013 events2 affected41 at risk EG0021 events1 affected16 at risk
Dyspepsia	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0004 events2 affected38 at risk EG0013 events3 affected41 at risk EG0020 events0 affected16 at risk
Toothache	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0003 events2 affected38 at risk EG0017 events6 affected41 at risk EG0022 events2 affected16 at risk
Fatigue	General disorders	MedDRA (9.0)	Systematic Assessment	EG0008 events7 affected38 at risk EG0013 events3 affected41 at risk EG0020 events0 affected16 at risk
Pyrexia	General disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG00110 events6 affected41 at risk EG0021 events1 affected16 at risk
Bronchitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG00113 events5 affected41 at risk EG0021 events1 affected16 at risk
Bronchitis acute	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0015 events5 affected41 at risk EG0021 events1 affected16 at risk
Gastroenteritis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG00110 events7 affected41 at risk EG0021 events1 affected16 at risk
Influenza	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0006 events4 affected38 at risk EG00110 events6 affected41 at risk EG00212 events4 affected16 at risk
Nasopharyngitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG00031 events16 affected38 at risk EG00120 events11 affected41 at risk EG0024 events3 affected16 at risk
Pharyngitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG00011 events5 affected38 at risk EG0012 events2 affected41 at risk EG0021 events1 affected16 at risk
Tinea versicolour	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG00113 events4 affected41 at risk EG0022 events1 affected16 at risk
Tonsillitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0014 events4 affected41 at risk EG0020 events0 affected16 at risk
Upper respiratory tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0009 events9 affected38 at risk EG0017 events4 affected41 at risk EG00214 events6 affected16 at risk
Urinary tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0005 events3 affected38 at risk EG0015 events3 affected41 at risk EG0020 events0 affected16 at risk
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG00013 events9 affected38 at risk EG00133 events14 affected41 at risk EG00234 events2 affected16 at risk
Back pain	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0007 events4 affected38 at risk EG0016 events5 affected41 at risk EG0021 events1 affected16 at risk
Bone pain	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG00012 events7 affected38 at risk EG00120 events7 affected41 at risk EG0027 events2 affected16 at risk
Myalgia	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0003 events2 affected38 at risk EG0013 events3 affected41 at risk EG0020 events0 affected16 at risk
Pain in extremity	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0006 events4 affected38 at risk EG0016 events5 affected41 at risk EG0021 events1 affected16 at risk
Shoulder pain	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0004 events3 affected38 at risk EG0014 events2 affected41 at risk EG0022 events1 affected16 at risk
Headache	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG00010 events6 affected38 at risk EG00116 events7 affected41 at risk EG0026 events4 affected16 at risk
Paraesthesia	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0012 events2 affected41 at risk EG0023 events1 affected16 at risk
Cough	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0006 events5 affected38 at risk EG0010 events0 affected41 at risk EG0024 events3 affected16 at risk
Pharyngolaryngeal pain	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0008 events5 affected38 at risk EG0014 events3 affected41 at risk EG0020 events0 affected16 at risk
Hypertension	Vascular disorders	MedDRA (9.0)	Systematic Assessment	EG0006 events4 affected38 at risk EG0016 events5 affected41 at risk EG0020 events0 affected16 at risk
Rectal haemorrhage	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Vomiting	Gastrointestinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Adverse drug reaction	General disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Chest discomfort	General disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Gait disturbance	General disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Influenza like illness	General disorders	MedDRA (9.0)	Systematic Assessment	EG0003 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Dental caries	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk
Diarrhoea infectious	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Hepatitis a	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Hordeolum	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0013 events2 affected41 at risk EG0021 events1 affected16 at risk
Lower respiratory tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Pyelonephritis acute	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Respiratory tract infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0002 events1 affected38 at risk EG0010 events0 affected41 at risk EG0023 events2 affected16 at risk
Rhinitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0013 events3 affected41 at risk EG0021 events1 affected16 at risk
Sinusitis	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0005 events3 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Staphylococcal infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Vaginal infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0003 events3 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Varicella	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Vulvovaginal mycotic infection	Infections and infestations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Arthritis	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0022 events2 affected16 at risk
Muscle spasms	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Osteoarthritis	Musculoskeletal and connective tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0003 events3 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Hyperprolactinaemia	Endocrine disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Convulsion	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Dizziness	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0013 events3 affected41 at risk EG0023 events1 affected16 at risk
Drop attacks	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Hypoaesthesia	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Sciatica	Nervous system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Anaemia	Blood and lymphatic system disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0022 events1 affected16 at risk
Leukocytosis	Blood and lymphatic system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Lymphadenitis	Blood and lymphatic system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Splenomegaly	Blood and lymphatic system disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0013 events3 affected41 at risk EG0020 events0 affected16 at risk
Epistaxis	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Dysphonia	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Dyspnoea	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events1 affected38 at risk EG0010 events0 affected41 at risk EG0023 events1 affected16 at risk
Postnasal drip	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Productive cough	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0015 events4 affected41 at risk EG0020 events0 affected16 at risk
Rhinitis allergic	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0023 events1 affected16 at risk
Sinus congestion	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0003 events3 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Tachypnoea	Respiratory, thoracic and mediastinal disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0022 events1 affected16 at risk
Haematoma	Vascular disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0011 events1 affected41 at risk EG0020 events0 affected16 at risk
Lentigo	Neoplasms benign, malignant and unspecified (incl cysts and polyps)	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Galactorrhoea	Reproductive system and breast disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Anaemia postoperative	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk
Burns first degree	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0015 events3 affected41 at risk EG0020 events0 affected16 at risk
Contusion	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0002 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Injury	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0012 events2 affected41 at risk EG0021 events1 affected16 at risk
Excoriation	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Joint injury	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Muscle strain	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Procedural pain	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0006 events3 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Skin laceration	Injury, poisoning and procedural complications	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Alanine aminotransferase increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Aspartate aminotransferase Increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Blood creatine phosphokinase Increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0013 events1 affected41 at risk EG0020 events0 affected16 at risk
Blood urine present	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk
Haemoglobin decreased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Mean cell volume increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Red blood cell count decreased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Neutrophil count increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Red blood cells urine positive	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk
White blood cell count increased	Investigations	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
White blood cells urine positive	Investigations	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Dermatitis allergic	Skin and subcutaneous tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Dermatitis contact	Skin and subcutaneous tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Rash vesicular	Skin and subcutaneous tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0027 events1 affected16 at risk
Erythema	Skin and subcutaneous tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Pruritus generalised	Skin and subcutaneous tissue disorders	MedDRA (9.0)	Systematic Assessment	EG0001 events1 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Biliary colic	Hepatobiliary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0022 events1 affected16 at risk
Cytolytic hepatitis	Hepatobiliary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0016 events3 affected41 at risk EG0020 events0 affected16 at risk
Cholelithiasis	Hepatobiliary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0022 events2 affected16 at risk
Liver disorder	Hepatobiliary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Nephrolithiasis	Renal and urinary disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0021 events1 affected16 at risk
Conjunctivitis allergic	Eye disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0010 events0 affected41 at risk EG0022 events1 affected16 at risk
Dry eye	Eye disorders	MedDRA (9.0)	Systematic Assessment	EG0002 events2 affected38 at risk EG0010 events0 affected41 at risk EG0020 events0 affected16 at risk
Visual acuity reduced	Eye disorders	MedDRA (9.0)	Systematic Assessment	EG0000 events0 affected38 at risk EG0011 events1 affected41 at risk EG0021 events1 affected16 at risk

ID	Title	Description
BG000	VPRIV 60 U/kg (Parent Study VPRIV (45 U/kg)-TKT032)	VPRIV 45 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044.
BG001	VPRIV 60 U/kg (Parent Study VPRIV (60 U/kg)-TKT032)	VPRIV 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625).
BG002	VPRIV 60 U/kg (Parent Study VPRIV (60U/kg) HGT-GCB-039)	VPRIV 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631).
BG003	VPRIV 60 U/kg (Parent Study-imiglucerase (60 U/kg) HGT-GCB-039	Imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631) and switched 60 U/kg VPRIV in HGT-GCB-044
BG004	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044
BG005	Total	Total of all reporting groups

ID	Term
D013106	Sphingolipidoses
D020140	Lysosomal Storage Diseases, Nervous System
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases
D002493	Central Nervous System Diseases
D009422	Nervous System Diseases
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D008064	Lipidoses
D008052	Lipid Metabolism, Inborn Errors
D016464	Lysosomal Storage Diseases
D008659	Metabolic Diseases
D009750	Nutritional and Metabolic Diseases
D052439	Lipid Metabolism Disorders

ID	Term
D005959	Glucosidases
D006026	Glycoside Hydrolases
D006867	Hydrolases
D004798	Enzymes
D045762	Enzymes and Coenzymes

ID	Title	Description	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	VPRIV 15-60 U/kg (Parent Study VPRIV (15-60 U/kg) TKT034)	VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647). Participants continued to receive VPRIV at a dose of 15-60 U/kg throughout participation in HGT-GCB-044	6	38	35	38
EG001	VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)	This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes participants from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)	6	41	35	41
EG002	VPRIV 60 U/kg (Parent Study-imiglucerase(60 U/kg) HGT-GCB-039)	imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631) and switched 60 U/kg VPRIV in HGT-GCB-044	4	16	15	16