A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

• Sponsor: Pfizer (Industry)
• Overall Status: Completed
• CT.gov ID: NCT01422187
• Collaborator: (none)
• Enrollment: 19
• Arms: 2
• Duration (Months): 37

Study Details

Study Description

Brief Summary

This is a multi-center trial to further extend the assessment of the safety and efficacy of taliglucerase alfa in adult subjects (≥18 years old) with Gaucher disease who have enrolled in Protocol PB-06-003. Subjects will continue to receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The duration of treatment will be a maximum of 21 months or until taliglucerase alfa is commercially available to the subject at the discretion of the Sponsor.

Condition or Disease	Intervention/Treatment	Phase
Gaucher Disease	Drug: Taliglucerase alfa	Phase 3

Study Design

• Study Type: Interventional
• Actual Enrollment: 19 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease
• Study Start Date: Aug 1, 2011
• Actual Primary Completion Date: May 1, 2014
• Actual Study Completion Date: Sep 1, 2014

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Taliglucerase alfa 30 units/kg Subjects randomized to receive 30 units/kg	Drug: Taliglucerase alfa; Taliglucerase infusion every two weeks for 21 months
Experimental: Taliglucerase alfa 60 units/kg Subjects randomized to 60 units/kg	Drug: Taliglucerase alfa; Taliglucerase infusion every two weeks for 21 months

Outcome Measures

Primary Outcome Measures

1. Spleen Volume [60 months]

   Spleen volume measured by MRI

Secondary Outcome Measures

1. Liver Volume [60 months]

   Liver volume by MRI

2. Platelet Count [60 months]

   Platelet count measure annually

3. Hemoglobin [60 months]

   Hemoglobin measure yearly

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• Successfully completed Protocol PB-06-001 and enrolled in Protocol PB-06-003

• The subject signs an informed consent

Exclusion Criteria:

• Currently taking another investigational drug for any condition.

• Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

• Pfizer

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Pfizer
• ClinicalTrials.gov Identifier: NCT01422187
• Other Study ID Numbers: PB-06-007
• First Posted: Aug 23, 2011
• Last Update Posted: Oct 5, 2018
• Last Verified: Sep 1, 2018

• Keywords provided by Pfizer: enzyme replacement therapy
• Additional relevant MeSH terms: Gaucher Disease

Study Results

Participant Flow

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
Period Title: Overall Study
STARTED	8	9	2
COMPLETED	7	8	2
NOT COMPLETED	1	1	0

Baseline Characteristics

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted	Total
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Total of all reporting groups
Overall Participants	8	9	2	19
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	44.8 (13.4)	39.9 (12.0)	38.5 (0.7)	41.8 (11.8)
Sex: Female, Male (Count of Participants)
Female	4 50%	3 33.3%	1 50%	8 42.1%
Male	4 50%	6 66.7%	1 50%	11 57.9%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	0 0%	0 0%	0 0%	0 0%
Asian	0 0%	0 0%	0 0%	0 0%
Native Hawaiian or Other Pacific Islander	0 0%	0 0%	0 0%	0 0%
Black or African American	0 0%	0 0%	0 0%	0 0%
White	8 100%	9 100%	2 100%	19 100%
More than one race	0 0%	0 0%	0 0%	0 0%
Unknown or Not Reported	0 0%	0 0%	0 0%	0 0%
Region of Enrollment (participants) [Number]
Israel	2 25%	2 22.2%	0 0%	4 21.1%
South Africa	0 0%	0 0%	1 50%	1 5.3%
Italy	0 0%	1 11.1%	0 0%	1 5.3%
Serbia	2 25%	2 22.2%	0 0%	4 21.1%
Mexico	3 37.5%	2 22.2%	0 0%	5 26.3%
Chile	0 0%	1 11.1%	1 50%	2 10.5%
Spain	1 12.5%	1 11.1%	0 0%	2 10.5%

Outcome Measures

1. Primary Outcome

Title	Spleen Volume
Description	Spleen volume measured by MRI
Time Frame	60 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
Measure Participants	8	9	2
Baseline	2032.0 (454.9)	1960.0 (451.7)	2321.7 (679.6)
Month 12	1522.6 (429.6)	1063.7 (280.7)	1677.9 (609.3)
Month 24	1193.0 (320.2)	744.9 (130.6)	1536.3 (506.7)
Month 36	1068.7 (264.6)	634.0 (71.0)	1256.6 (385.0)
Month 48	1137.4 (314.6)	526.8 (48.5)	1070.0 (280.6)
Month 60	1019.2 (285.3)	516.1 (54.5)	919.9 (180.9)

2. Secondary Outcome

Title	Liver Volume
Description	Liver volume by MRI
Time Frame	60 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
Measure Participants	8	9	2
Baseline	3103.4 (324.7)	2536.1 (170.0)	2488.6 (206.2)
Month 12	2619.3 (266.4)	2155.2 (104.8)	2048.0 (87.8)
Month 24	2367.9 (212.8)	1939.5 (101.5)	2064.0 (202.5)
Month 36	2429.6 (229.6)	2010.6 (143.5)	1959.0 (140.5)
Month 48	2429.1 (283.2)	1971.2 (179.3)	1792.2 (143.6)
Month 60	2290.9 (233.6)	2067.7 (188.9)	1820.8 (196.5)

3. Secondary Outcome

Title	Platelet Count
Description	Platelet count measure annually
Time Frame	60 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
Measure Participants	8	9	2
Baseline	70975 (8700.6)	70067 (7695.5)	69500 (42500)
Month 12	86613 (11894)	137556 (18358)	88000 (65000)
Month 24	103250 (17008)	168778 (23717)	101000 (68000)
Month 36	106775 (13517)	155667 (16060)	94500 (64500)
Month 48	100643 (15314)	184000 (16688)	103000 (53000)
Month 60	104986 (18961)	180625 (17062)	103500 (60500)

4. Secondary Outcome

Title	Hemoglobin
Description	Hemoglobin measure yearly
Time Frame	60 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Taliglucerase Alfa 30 Units/kg	Taliglucerase Alfa 60 Units/kg	Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months	Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
Measure Participants	8	9	2
Baseline	12.3 (0.7)	11.6 (0.9)	12.5 (0.9)
Month 12	13.9 (0.7)	14.2 (0.7)	14.3 (0.9)
Month 24	13.5 (0.8)	14.1 (0.7)	14.2 (0.2)
Month 36	14.0 (0.5)	14.2 (0.8)	14.4 (0.1)
Month 48	13.7 (0.6)	14.0 (0.8)	14.5 (0.1)
Month 60	14.1 (0.9)	13.9 (0.6)	14.3 (0.1)

Adverse Events

Time Frame	21 Months
Arm/Group Title	Taliglucerase Alfa 30 Units/kg		Taliglucerase Alfa 60 Units/kg		Dose Adjusted
Arm/Group Description	Subjects randomized to receive 30 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months		Subjects randomized to 60 units/kg Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months		Subjects randomized to 30 units/kg but with dose increased Taliglucerase alfa: Taliglucerase infusion every two weeks for 21 months
All Cause Mortality
	Taliglucerase Alfa 30 Units/kg		Taliglucerase Alfa 60 Units/kg		Dose Adjusted
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	/ (NaN)		/ (NaN)		/ (NaN)
Serious Adverse Events
	Taliglucerase Alfa 30 Units/kg		Taliglucerase Alfa 60 Units/kg		Dose Adjusted
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/8 (0%)		2/9 (22.2%)		0/2 (0%)
Metabolism and nutrition disorders
Thyroid Carcinoma	0/8 (0%)	0	1/9 (11.1%)	1	0/2 (0%)	0
Musculoskeletal and connective tissue disorders
Osteoarthritis	0/8 (0%)	0	1/9 (11.1%)	1	0/2 (0%)	0
Other (Not Including Serious) Adverse Events
	Taliglucerase Alfa 30 Units/kg		Taliglucerase Alfa 60 Units/kg		Dose Adjusted
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	6/8 (75%)		8/9 (88.9%)		1/2 (50%)
Gastrointestinal disorders
DYSPEPSIA	1/8 (12.5%)	1	1/9 (11.1%)	1	0/2 (0%)	0
TOOTHACHE	1/8 (12.5%)	1	1/9 (11.1%)	1	0/2 (0%)	0
Injury, poisoning and procedural complications
CONTUSION	0/8 (0%)	0	2/9 (22.2%)	2	0/2 (0%)	0
Musculoskeletal and connective tissue disorders
ARTHRALGIA	0/8 (0%)	0	3/9 (33.3%)	3	0/2 (0%)	0
BONE PAIN	0/8 (0%)	0	1/9 (11.1%)	1	1/2 (50%)	1
Nervous system disorders
HYPOAESTHESIA	2/8 (25%)	2	0/9 (0%)	0	0/2 (0%)	0
Respiratory, thoracic and mediastinal disorders
NASOPHARYNGITIS	2/8 (25%)	2	2/9 (22.2%)	2	0/2 (0%)	0
PHARYNGITIS	2/8 (25%)	2	0/9 (0%)	0	0/2 (0%)	0

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT 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.