glycolic acid has been researched along with Oxaluria, Primary in 22 studies
glycolic acid: RN given refers to parent cpd
glycolic acid : A 2-hydroxy monocarboxylic acid that is acetic acid where the methyl group has been hydroxylated.
| Excerpt | Relevance | Reference |
|---|---|---|
| " In part B, patients with primary hyperoxaluria type 1 had a mean maximal reduction from baseline of 75% across dosing cohorts in 24-hour urinary oxalate excretion." | 3.01 | Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial. ( Cochat, P; Deschênes, G; Erbe, DV; Frishberg, Y; Garg, PP; Groothoff, JW; Habtemariam, BA; Harambat, J; Haslett, P; Hulton, SA; Lieske, JC; Lorch, U; Lu, J; Magen, D; McGregor, TL; Milliner, DS; Talamudupula, S; Vaishnaw, AK; Van't Hoff, WG, 2021) |
| "In primary hyperoxaluria type 1 excessive endogenous production of oxalate and glycolate leads to increased urinary excretion of these metabolites." | 1.91 | Simple, fast and inexpensive quantification of glycolate in the urine of patients with primary hyperoxaluria type 1. ( Boehm, T; Friesser, E; Hoppe, B; Hubmann, H; Jilma, B; Klavins, K; Kovacevic, K; Macheroux, P; Martin-Higueras, C; Walli, A; Wallner, S; Zitta, C, 2023) |
| "Primary hyperoxaluria type 1 (PH1) is an inborn error of glyoxylate metabolism, characterized by increased endogenous oxalate production." | 1.62 | Endogenous Oxalate Production in Primary Hyperoxaluria Type 1 Patients. ( Garrelfs, SF; Groothoff, JW; Oosterveld, MJS; Peters-Sengers, H; Schierbeek, H; van den Akker, CHP; van Goudoever, JB; van Harskamp, D; Wanders, RJA; Wijburg, FA, 2021) |
| "The major clinical manifestation of the Primary Hyperoxalurias (PH) is increased production of oxalate, as a consequence of genetic mutations that lead to aberrant glyoxylate and hydroxyproline metabolism." | 1.56 | The effects of the inactivation of Hydroxyproline dehydrogenase on urinary oxalate and glycolate excretion in mouse models of primary hyperoxaluria. ( Buchalski, B; Challa, A; Fargue, S; Holmes, RP; Knight, J; Lowther, WT; Wood, KD, 2020) |
| "This may reveal underdiagnosed primary hyperoxaluria, even in older patients." | 1.46 | Late diagnosis of primary hyperoxaluria type III. ( Acquaviva, C; Blouin, JM; Bouchet, S; de la Faille, R; Harambat, J; Llanas, B; Richard, E, 2017) |
| "The primary hyperoxalurias are inherited disorders of glyoxylate metabolism, which cause over-production of oxalate leading to urolithiasis and subsequent renal failure." | 1.43 | Simultaneous analysis of urinary metabolites for preliminary identification of primary hyperoxaluria. ( Clifford-Mobley, O; Hewitt, L; Rumsby, G, 2016) |
| "The hereditary kidney stone disease primary hyperoxaluria type 1 (PH1) is caused by a functional deficiency of the liver-specific, peroxisomal, pyridoxal-phosphate-dependent enzyme, alanine:glyoxylate aminotransferase (AGT)." | 1.43 | Effects of alanine:glyoxylate aminotransferase variants and pyridoxine sensitivity on oxalate metabolism in a cell-based cytotoxicity assay. ( Danpure, CJ; Fargue, S; Holmes, RP; Knight, J; Rumsby, G, 2016) |
| "Type I primary hyperoxaluria (PH I) results from a deficiency of alanine:glyoxylate aminotransferase, whereas type II disease has been traced to a deficiency of D-glycerate dehydrogenase." | 1.30 | Extraction of glyceric and glycolic acids from urine with tetrahydrofuran: utility in detection of primary hyperoxaluria. ( Dietzen, DJ; Kenagy, DN; Landt, M; Milliner, DS; Smith, CH; Wilhite, TR, 1997) |
| "Calcium oxalate was estimated to represent 0." | 1.29 | Bony content of oxalate in patients with primary hyperoxaluria or oxalosis-unrelated renal failure. ( Barbos, MP; Cadario, A; Cerelli, E; Linari, F; Marangella, M; Petrarulo, M; Tricerri, A; Vitale, C, 1995) |
| "The detection of type I primary hyperoxaluria is based on the finding of exceedingly high oxalate excretion which is associated with increased glycolate excretion." | 1.28 | Glycolate determination detects type I primary hyperoxaluria in dialysis patients. ( Bianco, O; Finocchiaro, P; Linari, F; Marangella, M; Petrarulo, M; Vitale, C, 1991) |
| "Primary oxalosis is a rare inborn error of oxalate metabolism." | 1.28 | Primary oxalosis mimicking hyperparathyroidism diagnosed after long-term hemodialysis. ( Cadario, A; Canavese, C; Marangella, M; Massara, C; Pavan, I; Petrarulo, M; Portigliatti Barbos, M; Rotolo, U; Salomone, M, 1990) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 1 (4.55) | 18.7374 |
| 1990's | 9 (40.91) | 18.2507 |
| 2000's | 1 (4.55) | 29.6817 |
| 2010's | 4 (18.18) | 24.3611 |
| 2020's | 7 (31.82) | 2.80 |
| Authors | Studies |
|---|---|
| Garrelfs, SF | 3 |
| van Harskamp, D | 2 |
| Peters-Sengers, H | 2 |
| van den Akker, CHP | 1 |
| Wanders, RJA | 1 |
| Wijburg, FA | 1 |
| van Goudoever, JB | 2 |
| Groothoff, JW | 4 |
| Schierbeek, H | 2 |
| Oosterveld, MJS | 3 |
| Metry, EL | 1 |
| Vaz, FM | 1 |
| Bijlsma, JA | 1 |
| Neradova, A | 1 |
| Boehm, T | 1 |
| Martin-Higueras, C | 1 |
| Friesser, E | 1 |
| Zitta, C | 1 |
| Wallner, S | 1 |
| Walli, A | 1 |
| Kovacevic, K | 1 |
| Hubmann, H | 1 |
| Klavins, K | 1 |
| Macheroux, P | 1 |
| Hoppe, B | 1 |
| Jilma, B | 1 |
| Buchalski, B | 1 |
| Wood, KD | 1 |
| Challa, A | 1 |
| Fargue, S | 2 |
| Holmes, RP | 2 |
| Lowther, WT | 1 |
| Knight, J | 2 |
| McGregor, TL | 2 |
| Hunt, KA | 1 |
| Yee, E | 1 |
| Mason, D | 1 |
| Nioi, P | 1 |
| Ticau, S | 1 |
| Pelosi, M | 1 |
| Loken, PR | 1 |
| Finer, S | 1 |
| Lawlor, DA | 1 |
| Fauman, EB | 1 |
| Huang, QQ | 1 |
| Griffiths, CJ | 1 |
| MacArthur, DG | 1 |
| Trembath, RC | 1 |
| Oglesbee, D | 1 |
| Lieske, JC | 2 |
| Erbe, DV | 2 |
| Wright, J | 1 |
| van Heel, DA | 1 |
| Frishberg, Y | 1 |
| Deschênes, G | 1 |
| Hulton, SA | 1 |
| Magen, D | 1 |
| Harambat, J | 2 |
| Van't Hoff, WG | 1 |
| Lorch, U | 1 |
| Milliner, DS | 2 |
| Haslett, P | 1 |
| Garg, PP | 1 |
| Vaishnaw, AK | 1 |
| Talamudupula, S | 1 |
| Lu, J | 1 |
| Habtemariam, BA | 1 |
| Cochat, P | 2 |
| Clifford-Mobley, O | 2 |
| Rumsby, G | 3 |
| Kanodia, S | 1 |
| Didi, M | 1 |
| Holt, R | 1 |
| Senniappan, S | 1 |
| Hewitt, L | 1 |
| Danpure, CJ | 1 |
| Richard, E | 1 |
| Blouin, JM | 1 |
| Llanas, B | 1 |
| Bouchet, S | 1 |
| Acquaviva, C | 1 |
| de la Faille, R | 1 |
| HOCKADAY, TD | 1 |
| FREDERICK, EW | 1 |
| CLAYTON, JE | 1 |
| SMITH, LH | 1 |
| Wong, PN | 1 |
| Law, EL | 1 |
| Tong, GM | 1 |
| Mak, SK | 1 |
| Lo, KY | 1 |
| Wong, AK | 1 |
| Marangella, M | 5 |
| Vitale, C | 2 |
| Petrarulo, M | 5 |
| Tricerri, A | 1 |
| Cerelli, E | 1 |
| Cadario, A | 2 |
| Barbos, MP | 1 |
| Linari, F | 4 |
| Dietzen, DJ | 1 |
| Wilhite, TR | 1 |
| Kenagy, DN | 1 |
| Smith, CH | 1 |
| Landt, M | 1 |
| Ogawa, Y | 1 |
| Hatano, T | 1 |
| Rinaldi, S | 1 |
| Rizzoni, G | 1 |
| Bianco, O | 2 |
| Finocchiaro, P | 1 |
| Cosseddu, D | 1 |
| Pellegrino, S | 1 |
| Canavese, C | 1 |
| Salomone, M | 1 |
| Massara, C | 1 |
| Portigliatti Barbos, M | 1 |
| Pavan, I | 1 |
| Rotolo, U | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| A Phase 1/2, Single-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Safety, Tolerability, Pharmacokinetic and Pharmacodynamics Study of Subcutaneously Administered ALN-GO1 in Healthy Adult Subjects, and Patients With Primary Hyperoxaluria Ty[NCT02706886] | Phase 1/Phase 2 | 52 participants (Actual) | Interventional | 2016-03-08 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
The endpoint was only measured during the initial 85 days in Part B. (NCT02706886)
Timeframe: Part B (MAD): Baseline
| Intervention | mg/g (Mean) |
|---|---|
| Part B: MAD: Placebo | 193 |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 241 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 289 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 281 |
(NCT02706886)
Timeframe: Part B (MAD): Baseline
| Intervention | mL/min/1.73 m^2 (Mean) |
|---|---|
| Part B: MAD: Placebo | 64.389 |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 108.149 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 86.268 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 88.251 |
The endpoint was only measured in Part B. (NCT02706886)
Timeframe: Part B (MAD): Baseline
| Intervention | mmol/24h/1.73m^2 (Mean) |
|---|---|
| Part B: MAD: Placebo | 1.96 |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 1.73 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 1.84 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 1.30 |
The pharmacodynamic (PD) outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. (NCT02706886)
Timeframe: Part A (SAD): Baseline, Part B (MAD): Baseline
| Intervention | umol/L (Mean) |
|---|---|
| Part A: SAD: Placebo | 5.1 |
| Part A: SAD: Lumasiran 0.3 mg/kg | 5.3 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 5.7 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 6.2 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 4.8 |
The endpoint was only measured in Part A. (NCT02706886)
Timeframe: Part A (SAD): Baseline
| Intervention | mg/g (Mean) |
|---|---|
| Part A: SAD: Placebo | 12.4 |
| Part A: SAD: Lumasiran 0.3 mg/kg | 15.7 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 15.7 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 13.0 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 14.8 |
An AE is any untoward medical occurrence in a clinical investigational subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. (NCT02706886)
Timeframe: Part A (SAD): Up to 405 days; Part B (MAD): Up to 546 days
| Intervention | Participants (Count of Participants) |
|---|---|
| Part A: SAD: Placebo | 5 |
| Part A: SAD: Lumasiran 0.3 mg/kg | 6 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 2 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 6 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 6 |
| Part B: MAD: Placebo | 2 |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 8 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 7 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 4 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | h*ng/mL (Mean) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 293.5232 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 1899.8119 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 7211.5890 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 16778.0579 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | h*ng/mL (Mean) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 1428.0412 | 1608.1457 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 7400.2181 | 7959.7873 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | h*ng/mL (Mean) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 6337.9082 | 5136.3462 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | percentage fractional excretion (Mean) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 17.4219 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 19.0713 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 21.0472 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 25.7931 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | percentage fractional excretion (Mean) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 11.0895 | 9.4698 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 11.1877 | 12.4604 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | percentage fractional excretion (Mean) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 7.1691 | 13.6938 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | ng/mL (Mean) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 39.7940 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 204.3748 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 533.4527 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 1176.1302 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | ng/mL (Mean) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 324.1386 | 147.6780 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 582.4515 | 701.1708 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 minutes (min), 1 hour (h), 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | ng/mL (Mean) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 432.2798 | 411.5613 |
The PD outcome measure of plasma glycolate concentration could only be calculated for Part A. Due to an issue with the plasma glycolate assay at the testing laboratory the data for Part B could not be calculated. (NCT02706886)
Timeframe: Part A (SAD): Days 15, 29, 57 and 85; Part B (MAD): Days 15, 29, 57, 85
| Intervention | percentage change from baseline (Mean) | |||
|---|---|---|---|---|
| Day 15 | Day 29 | Day 57 | Day 85 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 58.3 | 32.9 | 66.3 | 15.6 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 48.5 | 70.6 | 109.8 | 40.7 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 56.4 | 146.4 | 230.1 | 196.2 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 59.5 | 390.1 | 730.4 | 731.3 |
| Part A: SAD: Placebo | 18.3 | 22.4 | 126.7 | 31.2 |
The endpoint was only measured in Part A. (NCT02706886)
Timeframe: Part A (SAD): Days 29 and 57
| Intervention | percentage change from baseline (Mean) | |
|---|---|---|
| Day 29 | Day 57 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 32.5 | 38.0 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 82.9 | 47.8 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 109.1 | 215.0 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 210.5 | 310.7 |
| Part A: SAD: Placebo | 8.1 | 73.8 |
The endpoint was only measured during the initial 85 days in Part B. (NCT02706886)
Timeframe: Part B (MAD): 24 hour urine collections on Days 29, 57 and 85
| Intervention | percentage change from baseline (Mean) | ||
|---|---|---|---|
| Day 29 | Day 57 | Day 85 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 53.1 | 82.3 | 71.0 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 33.0 | 81.8 | 42.0 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 31.4 | 42.3 | 43.7 |
| Part B: MAD: Placebo | -15.1 | -13.8 | -23.0 |
The endpoint was only measured in Part B. (NCT02706886)
Timeframe: Part B (MAD): 24 hour urine collections on Days 29, 57, 85, 113, 141, 169, 197
| Intervention | percentage change from baseline (Mean) | ||
|---|---|---|---|
| Day 29 | Day 57 | Day 85 | |
| Part B: MAD: Placebo | -2.4 | -27.8 | 9.1 |
The endpoint was only measured in Part B. (NCT02706886)
Timeframe: Part B (MAD): 24 hour urine collections on Days 29, 57, 85, 113, 141, 169, 197
| Intervention | percentage change from baseline (Mean) | ||||||
|---|---|---|---|---|---|---|---|
| Day 29 | Day 57 | Day 85 | Day 113 | Day 141 | Day 169 | Day 197 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | -41.1 | -49.7 | -65.6 | -61.4 | -64.6 | -61.6 | -63.8 |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | -49.2 | -49.1 | -53.3 | -59.1 | -68.4 | -48.7 | -52.7 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | -57.5 | -72.5 | -68.4 | -78.1 | -73.5 | -69.3 | -71.2 |
(NCT02706886)
Timeframe: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
| Intervention | percentage change from baseline (Mean) | ||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 29 | Day 57 | Day 85 | Day 113 | Day 141 | Day 169 | Day 197 | Day 225 | Day 253 | Day 281 | Day 309 | Day 337 | Day 365 | Day 393 | Day 421 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | -6.672 | -8.426 | -13.536 | -14.424 | -8.804 | -19.796 | -8.546 | -18.221 | -5.140 | -14.283 | -0.403 | -5.168 | 2.268 | -11.942 | 7.610 |
(NCT02706886)
Timeframe: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
| Intervention | percentage change from baseline (Mean) | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Day 29 | Day 57 | Day 85 | Day 113 | Day 141 | Day 169 | Day 197 | Day 225 | Day 253 | Day 281 | Day 309 | Day 337 | Day 365 | Day 393 | Day 421 | Day 449 | |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | -0.624 | 4.505 | -3.720 | 3.444 | 9.315 | -8.544 | -13.513 | 29.013 | 10.232 | 5.841 | 8.441 | 8.688 | 9.201 | -4.484 | -6.498 | -15.093 |
(NCT02706886)
Timeframe: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
| Intervention | percentage change from baseline (Mean) | ||
|---|---|---|---|
| Day 29 | Day 57 | Day 85 | |
| Part B: MAD: Placebo | 9.019 | 20.184 | -5.945 |
(NCT02706886)
Timeframe: Part B (MAD): Days 29, 57, 85, 113, 141, 169, 197, 225, 253, 281, 309, 337, 365, 393, 421, 449
| Intervention | percentage change from baseline (Mean) | |||||||
|---|---|---|---|---|---|---|---|---|
| Day 29 | Day 57 | Day 85 | Day 113 | Day 141 | Day 169 | Day 197 | Day 225 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 2.748 | 37.030 | -6.113 | 7.570 | -30.691 | 20.210 | 28.857 | -14.964 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | L/h (Mean) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 8.7817 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 5.4906 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 5.8211 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 6.3417 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | L/h (Mean) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 2.2612 | 1.9610 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 2.3818 | 2.5150 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: pooled urine 0-4 h, 4-8 h and 8-24 h; Part B (MAD): Part B (MAD phase): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: pooled urine 0-4 h, 4-8 h, 8-12 h and 12-24 h
| Intervention | L/h (Mean) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 2.0564 | 3.3663 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Mean) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 1.0 mg/kg | 7.0655 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 5.9798 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 3.4683 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Mean) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 3.2670 | 7.8090 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 5.4574 | 5.8356 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Mean) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 7.8028 | 4.6694 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Median) |
|---|---|
| Day 1 | |
| Part A: SAD: Lumasiran 0.3 mg/kg | 5.0167 |
| Part A: SAD: Lumasiran 1.0 mg/kg | 1.5000 |
| Part A: SAD: Lumasiran 3.0 mg/kg | 3.0000 |
| Part A: SAD: Lumasiran 6.0 mg/kg | 7.0000 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Median) | |
|---|---|---|
| Day 1 | Day 57 | |
| Part B: MAD: Lumasiran 1.0 mg/kg qM | 3.9917 | 3.0417 |
| Part B: MAD: Lumasiran 3.0 mg/kg qM | 4.9917 | 2.9833 |
Samples for Part A were collected only on Day 1; Part B on Days 1 and 57 for qM and on Days 1 and 85 for q3M arm groups. (NCT02706886)
Timeframe: Part A (SAD): Day 1: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h and 24 h; Part B (MAD): Days 1 and 57 for qM dosing and Days 1 and 85 for q3M dosing: predose, 30 min, 1 h, 2 h, 4 h, 6 h, 8 h, 12 h, 24 h and 48 h
| Intervention | hours (Median) | |
|---|---|---|
| Day 1 | Day 85 | |
| Part B: MAD: Lumasiran 3.0 mg/kg q3M | 9.0000 | 5.9833 |
2 reviews available for glycolic acid and Oxaluria, Primary
| Article | Year |
|---|---|
[Primary hyperoxaluria type 1 (PH1)].
Topics: Biomarkers; Glycolates; Humans; Hyperoxaluria, Primary; Minisatellite Repeats; Mutation; Oxalates; O | 1998 |
Primary hyperoxaluria type 1.
Topics: Child; Female; Glycolates; Humans; Hyperoxaluria, Primary; Kidney Failure, Chronic; Kidney Transplan | 1999 |
1 trial available for glycolic acid and Oxaluria, Primary
| Article | Year |
|---|---|
Phase 1/2 Study of Lumasiran for Treatment of Primary Hyperoxaluria Type 1: A Placebo-Controlled Randomized Clinical Trial.
Topics: Adolescent; Adult; Child; Female; Glycolates; Humans; Hyperoxaluria, Primary; Male; Oxalates; Renal | 2021 |
19 other studies available for glycolic acid and Oxaluria, Primary
| Article | Year |
|---|---|
Endogenous Oxalate Production in Primary Hyperoxaluria Type 1 Patients.
Topics: Glycine; Glycolates; Glyoxylates; Humans; Hyperoxaluria; Hyperoxaluria, Primary; Oxalates; Pyridoxin | 2021 |
Plasma oxalate and glycolate concentrations in dialysis patients with and without primary hyperoxaluria type 1.
Topics: Glycolates; Humans; Hyperoxaluria; Hyperoxaluria, Primary; Oxalates; Renal Dialysis | 2023 |
Simple, fast and inexpensive quantification of glycolate in the urine of patients with primary hyperoxaluria type 1.
Topics: Animals; Glycine; Glycolates; Glyoxylates; Hyperoxaluria; Hyperoxaluria, Primary; Mice; Oxalates | 2023 |
The effects of the inactivation of Hydroxyproline dehydrogenase on urinary oxalate and glycolate excretion in mouse models of primary hyperoxaluria.
Topics: Amino Acid Sequence; Animals; Base Sequence; Calcium; Disease Models, Animal; Female; Glycolates; Hu | 2020 |
Development and Validation of a New Gas Chromatography-Tandem Mass Spectrometry Method for the Measurement of Enrichment of Glyoxylate Metabolism Analytes in Hyperoxaluria Patients Using a Stable Isotope Procedure.
Topics: Acetamides; Carbon Isotopes; Fluoroacetates; Gas Chromatography-Mass Spectrometry; Glycolates; Glyox | 2020 |
Characterising a healthy adult with a rare HAO1 knockout to support a therapeutic strategy for primary hyperoxaluria.
Topics: Adult; Alcohol Oxidoreductases; Animals; CHO Cells; Cricetulus; Female; Glycolates; Humans; Hyperoxa | 2020 |
Glycolate oxidase deficiency in a patient with congenital hyperinsulinism and unexplained hyperoxaluria.
Topics: Alcohol Oxidoreductases; Congenital Hyperinsulinism; Diagnosis, Differential; Female; Glycolates; Hu | 2017 |
Simultaneous analysis of urinary metabolites for preliminary identification of primary hyperoxaluria.
Topics: Calibration; Female; Glutarates; Glyceric Acids; Glycolates; Humans; Hyperoxaluria, Primary; Limit o | 2016 |
Effects of alanine:glyoxylate aminotransferase variants and pyridoxine sensitivity on oxalate metabolism in a cell-based cytotoxicity assay.
Topics: Animals; Cell Survival; CHO Cells; Cricetulus; Glycolates; Humans; Hyperoxaluria, Primary; Mutation; | 2016 |
Late diagnosis of primary hyperoxaluria type III.
Topics: Aged; Carcinoma, Renal Cell; Delayed Diagnosis; Gene Expression; Glyceric Acids; Glycolates; Humans; | 2017 |
STUDIES ON PRIMARY HYPEROXALURIA. II. URINARY OXALATE, GLYCOLATE, AND GLYOXYLATE MEASUREMENT BY ISOTOPE DILUTION METHODS.
Topics: Acetates; Body Fluids; Carbon Isotopes; Child; Glycolates; Glyoxylates; Humans; Hyperoxaluria, Prima | 1965 |
Diagnosis of primary hyperoxaluria type 1 by determination of peritoneal dialysate glycolic acid using standard organic-acids analysis method.
Topics: Aged; Female; Glycolates; Hemodialysis Solutions; Humans; Hyperoxaluria, Primary; Male; Middle Aged; | 2003 |
Bony content of oxalate in patients with primary hyperoxaluria or oxalosis-unrelated renal failure.
Topics: Adolescent; Adult; Biopsy; Bone and Bones; Calcium; Calcium Oxalate; Child; Chronic Kidney Disease-M | 1995 |
Extraction of glyceric and glycolic acids from urine with tetrahydrofuran: utility in detection of primary hyperoxaluria.
Topics: Adolescent; Child; Child, Preschool; Chromatography, Gas; Creatinine; Furans; Glyceric Acids; Glycol | 1997 |
Clinical quiz. Primary hyperoxaluria (PH) type 1.
Topics: Acute Kidney Injury; Calcium Oxalate; Creatinine; Diagnosis, Differential; Glycolates; Humans; Hyper | 1991 |
Glycolate determination detects type I primary hyperoxaluria in dialysis patients.
Topics: Adolescent; Adult; Chromatography, High Pressure Liquid; Glycolates; Humans; Hyperoxaluria, Primary; | 1991 |
High-performance liquid chromatographic determination of plasma glycolic acid in healthy subjects and in cases of hyperoxaluria syndromes.
Topics: Chromatography, High Pressure Liquid; Glycolates; Humans; Hydrazones; Hyperoxaluria, Primary; Kidney | 1991 |
Improved high-performance liquid chromatographic determination of urinary glycolic acid.
Topics: Adult; Alcohol Oxidoreductases; Chromatography, High Pressure Liquid; Glycolates; Humans; Hyperoxalu | 1990 |
Primary oxalosis mimicking hyperparathyroidism diagnosed after long-term hemodialysis.
Topics: Adult; Biopsy; Bone and Bones; Diagnosis, Differential; Glycolates; Humans; Hyperoxaluria, Primary; | 1990 |