Table 1.
Baseline characteristics of study population
| OC3 (N = 21) | Placebo (N = 15) | Total (N = 36) | |
|---|---|---|---|
| Age at screening, years | |||
| n | 21 | 15 | 36 |
| Mean (SD) | 16.2 (12.72) | 22.6 (16.55) | 18.8 (14.57) |
| Median | 12.6 | 17.1 | 15.0 |
| Min, Max | 4, 54 | 7, 63 | 15, 63 |
| p valuea | 0.10 | ||
| Age distribution at screening, n (%) | |||
| Children (<12 years) | 10 (47.6) | 3 (20.0) | 13 (36.1) |
| Adolescents (≥12, <18 years) | 6 (28.6) | 6 (40.0) | 12 (33.3) |
| Adults (≥18 years) | 5 (23.8) | 6 (40.0) | 11 (30.6) |
| p valuea | 0.117 | ||
| Ethnicity, n (%) | |||
| Caucasian n (%) | 20 (95.2) | 15 (100) | 35 (97.2) |
| Other n (%) | 1 (4.8) | 0 (0) | 1 (2.8) |
| p valuea | 0.40 | ||
| Sex, n (%) | |||
| Male | 10 (47.6) | 9 (60.0) | 19 (52.8) |
| Female | 11 (52.4) | 6 (40.0) | 17 (47.2) |
| p valuea | 0.47 | ||
| Primary hyperoxaluria type, n (%) | |||
| Type I | 18 (85.7) | 13 (86.7) | 31 (86.1) |
| Type II | 3 (14.3) | 2 (13.3) | 5 (13.9) |
| p valuea | 0.936 | ||
| Time since diagnosis, yearsb | |||
| n | 21 | 14 | 35 |
| Mean (SD) | 5.50 (6.08) | 15.44 (11.96) | 9.47 (10.04) |
| Median | 3.30 | 13.10 | 5.70 |
| Min, Max | −0.1b–23.1 | 4.8–41.6 | −0.1–41.6 |
| p valuea | <0.001 | ||
| Age at diagnosis, years | |||
| n | 21 | 14 | 35 |
| Mean (SD) | 10.7 (12.98) | 8.1 (11.51) | 9.7 (12.31) |
| Median | 5.0 | 5.0 | 5.0 |
| Min, Max | 0–53 | 2–47 | 0–53 |
| p valuea | 0.56 | ||
| Frequency of stone events during past year, n (%) | |||
| 0 event | 16 (76.2) | 8 (53.3) | 24 (66.7) |
| 1 event | 4 (19.0) | 2 (13.3) | 6 (16.7) |
| 2 event | 0 (0.0) | 4 (26.7) | 4 (11.1) |
| ≥3 event | 1 (4.8) | 1 (6.7) | 2 (5.6) |
| p valuea | 0.08 | ||
| Stone events during past three years | |||
| n | 21 | 15 | 36 |
| Mean (SD) | 2.0 (3.53) | 1.7 (2.05) | 1.9 (2.97) |
| Median | 1.0 | 1.0 | 1.0 |
| Min, Max | 0–16 | 0–7 | 0–16 |
| p valuea | 0.92 | ||
| Estimated glomerular filtration rate, mL/min/1.73 m2 | |||
| n | 21 | 15 | 36 |
| Mean (SD) | 111.6 (48.60) | 109.6 (40.26) | 110.8 (44.71) |
| Median | 121.0 | 103.0 | 107.0 |
| Min, Max | 36–188 | 44–203 | 36–203 |
| p valuea | 0.822 | ||
| Renal function during the past year, n (%) | |||
| Stable | 20 (95.2) | 15 (100) | 35 (97.2) |
| Worsening | 1 (4.8) | 0 (0.0) | 1 (2.8) |
| p valuea | 0.40 | ||
| CKD stage, n (%) | |||
| Stage I (eGFR ≥ 90) | 14 (66.7) | 11 (73.3) | 25 (69.4) |
| Stage II (eGFR ≥ 60, <90) | 3 (14.3) | 3 (20.0) | 6 (16.7) |
| Stage III (eGFR ≥ 30, <60) | 4 (19.0) | 1 (6.7) | 5 (13.9) |
| p valuea | 0.44 | ||
| Urinary oxalate excretion (mmol/24 h/1.73 m2) at baseline | |||
| n | 19 | 12 | – |
| Mean (SD) | 1.88 (0.50) | 1.62 (0.52) | – |
| Median | 1.74 | 1.56 | – |
| Min, Max | 1.1–2.8 | 1.1–2.5 | – |
| p valuea | 0.32 | ||
eGFR estimated glomerular filtration rate, SD standard deviation
a p value obtained in post hoc analyses
b Time since diagnosis was the date of screening minus the date of diagnosis; one patient was diagnosed after screening and hence, there was one negative value