Skip to main content

Table 6 Quantitative GL3 scoring in kidney peri-tubular capillaries (PTC)

From: Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies

 

Visit

Reader 1

Reader 2

Average

FAB CL-2021

    

2-01022

Baseline

2.3

2.3

2.3

 

Week 12

5.7

6.6

6.2

2-0103

Baseline

4.4

7.4

5.9

 

Week 12

0.3

0.3

0.3

2-0104

Baseline

2.3

3.5

2.9

 

Week 48

0.9

1.4

1.2

2-0202

Baseline

0.5

0.2

0.3

 

Week 12

0.8

0.3

0.5

 

Week 48

0.3

0.3

0.3

FAB CL-2031

    

3-03012

Baseline

0.7

2.0

1.4

 

Week 24

3.5

-

3.5

 

Week 48

0

-

0

3-0302

Baseline

2.4

2.6

2.6

 

Week 24

3.5

5.4

4.5

 

Week 48

4.4

4.2

4.3

3-0303

Baseline

3.5

2.2

2.9

 

Week 24

1.0

1.2

1.1

 

Week 48

2.6

3

2.8

3-RF013

Baseline

0.4

0.1

0.3

 

Week 24

0.1

0.0

0.1

 

Week 48

-

-

-

3-RF034

Baseline

-

-

-

 

Week 24

0.0

0.1

0.1

 

Week 48

0.0

0.0

0.1

  1. 1 Study number.
  2. 2 Subject number.
  3. 3 Week 48 biopsy not evaluable.
  4. 4 Baseline biopsy not evaluable.
  5. Average number of GL-3 inclusions per cell as measured in biopsy samples at different time points (BLISS).