Trial Outcomes & Findings for A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (NCT NCT04002830)

NCT ID: NCT04002830

Last Updated: 2024-12-17

Results Overview

Percent change from baseline

• Recruitment status: COMPLETED
• Study phase: PHASE4
• Target enrollment: 14 participants
• Primary outcome timeframe: from baseline to month 12
• Results posted on: 2024-12-17

Participant Flow

If a patient did not meet the inclusion criteria or meet any of the exclusion criteria listed in the protocol, the patient was excluded at screening visits and was not enrolled.

Participant milestones

Measure	Taliglucerase Alfa The intervention: Taliglucerase alfa (60 units/kg, intravenously, every 2 weeks for 12 months). The target population: Treatment-naïve subjects with Type 3 Gaucher Disease. Any subgroup considerations: All participants will receive the investigational product.
Overall Study STARTED	14
Overall Study COMPLETED	14
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

Baseline characteristics by cohort

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Age, Categorical <=18 years	14 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	3.2 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	13 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	1 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment Israel	1 participants n=5 Participants
Region of Enrollment India	13 participants n=5 Participants

PRIMARY outcome

Timeframe: from baseline to month 12

Percent change from baseline

Outcome measures

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Percent Change From Baseline in Spleen Volume Measured by MRI	-51.33 Percent change Standard Error 8.67

SECONDARY outcome

Timeframe: from baseline to month 12

Percent change from baseline

Outcome measures

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Percent Change From Baseline in Liver Volume Measured by MRI	-33.91 Percent change Standard Error 3.81

SECONDARY outcome

Timeframe: from baseline to Months 3, 6, 9, and 12

Percent change from baseline

Outcome measures

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Percent Change in Hemoglobin 3 months	26.43 percentage change in hemoglobin (%) Standard Error 7.92
Percent Change in Hemoglobin 6 months	28.97 percentage change in hemoglobin (%) Standard Error 6.43
Percent Change in Hemoglobin 9 months	29.93 percentage change in hemoglobin (%) Standard Error 6.50
Percent Change in Hemoglobin 12 months	35.81 percentage change in hemoglobin (%) Standard Error 8.88

SECONDARY outcome

Timeframe: from baseline to Months 3, 6, 9, and 12

Percent change from baseline

Outcome measures

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Percent Change in Platelet Count 3 months	2.50 percentage change in platelet count (%) Standard Error 66.09
Percent Change in Platelet Count 6 months	53.33 percentage change in platelet count (%) Standard Error 56.20
Percent Change in Platelet Count 9 months	94.44 percentage change in platelet count (%) Standard Error 54.20
Percent Change in Platelet Count 12 months	122.44 percentage change in platelet count (%) Standard Error 79.82

SECONDARY outcome

Timeframe: from baseline to Months 3, 6, 9, and 12

Percent change from baseline

Outcome measures

Measure	Taliglucerase Alfa n=14 Participants Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Percent Change in Lyso-GB1 3 months	-29.16 percentage change in Lyso-Gb1 (%) Standard Error 3.99
Percent Change in Lyso-GB1 6 months	-46.57 percentage change in Lyso-Gb1 (%) Standard Error 7.95
Percent Change in Lyso-GB1 9 months	-60.00 percentage change in Lyso-Gb1 (%) Standard Error 4.16
Percent Change in Lyso-GB1 12 months	-74.32 percentage change in Lyso-Gb1 (%) Standard Error 5.60

Adverse Events

Taliglucerase Alfa

Serious events: 1 serious events

Other events: 9 other events

Deaths: 0 deaths

Serious adverse events

Measure

Taliglucerase Alfa n=14 participants at risk Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.

Respiratory, thoracic and mediastinal disorders Shortness of breath

7.1% 1/14 • Number of events 1 • 12 months

Other adverse events

Measure	Taliglucerase Alfa n=14 participants at risk Intravenous infusion of Taligluucerase alfa (Elelyso) in treatment-naive patients with type 3 Gaucher disease Elelyso: Taliglucerase alfa is currently an approved therapy in the United States and many other countries for adults and children with a confirmed diagnosis of Type 1 GD ,and is also approved for use in Type 3 GD in a small number of countries.
Respiratory, thoracic and mediastinal disorders Cough	21.4% 3/14 • Number of events 4 • 12 months
Blood and lymphatic system disorders Hematoma	14.3% 2/14 • Number of events 2 • 12 months
General disorders Fever	14.3% 2/14 • Number of events 3 • 12 months
Skin and subcutaneous tissue disorders Urticaria	21.4% 3/14 • Number of events 5 • 12 months
Respiratory, thoracic and mediastinal disorders Upper respiratory infection	28.6% 4/14 • Number of events 4 • 12 months
Gastrointestinal disorders Vomitting	7.1% 1/14 • Number of events 2 • 12 months
Respiratory, thoracic and mediastinal disorders Pneumonia	7.1% 1/14 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Labored breathing	7.1% 1/14 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Snoring	7.1% 1/14 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders Suffocation	7.1% 1/14 • Number of events 1 • 12 months
General disorders Sweating	7.1% 1/14 • Number of events 1 • 12 months
Injury, poisoning and procedural complications Mosquito bite	7.1% 1/14 • Number of events 1 • 12 months
Nervous system disorders Hypotonus	7.1% 1/14 • Number of events 1 • 12 months
Musculoskeletal and connective tissue disorders Kyphosis	7.1% 1/14 • Number of events 1 • 12 months
Musculoskeletal and connective tissue disorders Pectus carinatum	7.1% 1/14 • Number of events 1 • 12 months

Additional Information

Majdolen Istaiti

Shaare Zedek Medical Center

Phone: 972526659995

Email: joleenist@szmc.org.il

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place