Table 2.
Summary of efficacy results for the only patient who tested positive for both IgG and neutralizing antibodies
| Efficacy endpoint | Baseline | Change from baseline to
|
||||
|---|---|---|---|---|---|---|
| Week 13 | Week 25 | Week 37 | Week 49 | Week 55 | ||
| HRV | ||||||
| SDNN (ms) | 76.0 | −1.0 | −6.0 | −12.0 | – | 17.0 |
| r-MSSD (ms) | 63.0 | −12.0 | −15.0 | −17.0 | – | 3.0 |
| pNN50 (%) | 26.7 | −9.5 | −10.8 | −13.7 | – | 0.6 |
| Cardiac structure | ||||||
| LVMI (g/m2.7) | 48.2 | −16.8 | −2.2 | −16.6 | −12.4 | −1.0 |
| MFS (%) | 15.9 | 4.0 | 1.5 | 3.8 | 2.8 | 3.9 |
| Renal function | ||||||
| eGFR (mL/min/1.73 m2) | 104.9 | 0.0 | 28.4 | 3.4 | 33.8 | 6.0 |
| Pharmacodynamics | ||||||
| Plasma Gb3 (nmol/mL) | 24.2 | −10.7 | −10.2 | −10.5 | −11.6 | −6.9 |
| Urinary Gb3 (nmol/g creatinine) | NA | |||||
| Clinical outcomes | ||||||
| BPI-worst pain | 9 | −9 | −9 | 1 | −5 | −4 |
| BPI-pain severity | 4.75 | −4.75 | −4.75 | −0.50 | −2.75 | −3.00 |
| Overall HUI2 | 0.7 | 0.2 | 0.3 | – | 0.0 | 0.1 |
| Overall HUI3 | 0.6 | 0.3 | 0.3 | – | 0.2 | 0.0 |
| CHQ-global health | 30 | 0 | 30 | 30 | 0 | 30 |
Notes: pNN50 represents the percentage of differences between adjacent filtered RR intervals that are >50 ms for the whole analysis. r-MSSD represents the square root of the sum of squares of difference between adjacent filtered RR intervals over the length of the analysis. SDNN represents the standard deviation of all filtered RR intervals for the length of the analysis.
Abbreviations: BPI, Brief Pain Inventory; CHQ, Children’s Health Questionnaire; eGFR, estimated glomerular filtration rate; Gb3, globotriaosylceramide; HRV, heart rate variability; HUI, Health Utilities Index; LVMI, left ventricular mass index; MFS, midwall fractional shortening; NA, not available.