porphobilinogen has been researched along with Pain in 8 studies
Pain: An unpleasant sensation induced by noxious stimuli which are detected by NERVE ENDINGS of NOCICEPTIVE NEURONS.
| Excerpt | Relevance | Reference |
|---|---|---|
| ") Key secondary end points were levels of ALA and porphobilinogen and the annualized attack rate among patients with acute hepatic porphyria, along with hemin use and daily worst pain scores in patients with acute intermittent porphyria." | 5.34 | Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria. ( Anderson, KE; Balwani, M; Bissell, DM; Bonkovsky, HL; Chen, J; Garg, P; Gouya, L; Harper, P; Horie, Y; Ivanova, A; Kauppinen, R; Keel, SB; Kim, JB; Ko, JJ; Langendonk, JG; Liu, S; Minder, E; Parker, C; Peiró, PA; Penz, C; Phillips, J; Rees, DC; Sardh, E; Silver, SM; Simon, AR; Stein, PE; Stölzel, U; Sweetser, MT; Vaishnaw, A; Vassiliou, D; Ventura, P; Wang, B; Wang, JD; Windyga, J, 2020) |
| " A provisional diagnosis of acute intermittent porphyria was made and was confirmed by the increased levels of urinary delta-aminolevulinic acid (ALA) and porphobilinogen (PBG)." | 3.68 | Acute intermittent porphyria. A perplexing case. ( Arena, A; Russo, P; Scuderi, D, 1992) |
| "Acute hepatic porphyria (AHP) is a family of four rare genetic diseases, each involving deficiency in a hepatic heme biosynthetic enzyme." | 3.11 | Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study. ( Balwani, M; Cassiman, D; Kubisch, I; Liu, S; Sweetser, MT; Takase, KI; Thapar, M; Ventura, P; Wang, B, 2022) |
| "Acute intermittent porphyria is one of eight disorders arising from disturbances in heme biosynthesis where the precursors, 5-aminolevulinate and porphobilinogen, are elevated in plasma and urine." | 1.72 | Peritoneal dialysis resulting in discontinuance of recurring attacks of acute intermittent porphyria: A case report. ( Erlandsen, EJ; Randers, E; Thomsen, HH; Østergaard, MGØ, 2022) |
| Timeframe | Studies, this research(%) | All Research% |
|---|---|---|
| pre-1990 | 4 (50.00) | 18.7374 |
| 1990's | 1 (12.50) | 18.2507 |
| 2000's | 0 (0.00) | 29.6817 |
| 2010's | 0 (0.00) | 24.3611 |
| 2020's | 3 (37.50) | 2.80 |
| Authors | Studies |
|---|---|
| Østergaard, MGØ | 1 |
| Erlandsen, EJ | 1 |
| Thomsen, HH | 1 |
| Randers, E | 1 |
| Wang, B | 2 |
| Ventura, P | 2 |
| Takase, KI | 1 |
| Thapar, M | 1 |
| Cassiman, D | 1 |
| Kubisch, I | 1 |
| Liu, S | 2 |
| Sweetser, MT | 2 |
| Balwani, M | 2 |
| Sardh, E | 1 |
| Peiró, PA | 1 |
| Rees, DC | 1 |
| Stölzel, U | 1 |
| Bissell, DM | 1 |
| Bonkovsky, HL | 1 |
| Windyga, J | 1 |
| Anderson, KE | 1 |
| Parker, C | 1 |
| Silver, SM | 1 |
| Keel, SB | 1 |
| Wang, JD | 1 |
| Stein, PE | 1 |
| Harper, P | 1 |
| Vassiliou, D | 1 |
| Phillips, J | 1 |
| Ivanova, A | 1 |
| Langendonk, JG | 1 |
| Kauppinen, R | 1 |
| Minder, E | 1 |
| Horie, Y | 1 |
| Penz, C | 1 |
| Chen, J | 1 |
| Ko, JJ | 1 |
| Garg, P | 1 |
| Vaishnaw, A | 1 |
| Kim, JB | 1 |
| Simon, AR | 1 |
| Gouya, L | 1 |
| Wechsler, HL | 1 |
| Arena, A | 1 |
| Russo, P | 1 |
| Scuderi, D | 1 |
| Potter, HP | 2 |
| Barclay, N | 1 |
| Trial | Phase | Enrollment | Study Type | Start Date | Status | ||
|---|---|---|---|---|---|---|---|
| ENVISION: A Phase 3 Randomized, Double-blind, Placebo-Controlled Multicenter Study With an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients With Acute Hepatic Porphyrias[NCT03338816] | Phase 3 | 94 participants (Actual) | Interventional | 2017-11-16 | Completed | ||
| [information is prepared from clinicaltrials.gov, extracted Sep-2024] | |||||||
Annualized rate of hemin doses was evaluated as annualized days of hemin use. (NCT03338816)
Timeframe: 6 months
| Intervention | annualized rate of use (Mean) |
|---|---|
| Placebo | 29.71 |
| Givosiran 2.5 mg/kg | 6.77 |
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home. (NCT03338816)
Timeframe: 6 months
| Intervention | annualized attack rate (Mean) |
|---|---|
| Placebo | 12.52 |
| Givosiran 2.5 mg/kg | 3.22 |
Porphyria attacks were defined as meeting all of the following criteria: an acute episode of neurovisceral pain in the abdomen, back, chest, extremities and/or limbs, no other medically determined cause, and required treatment with intravenous (IV) dextrose or hemin, carbohydrates, or analgesics, or other medications such as antiemetics at a dose or frequency beyond the participant's usual daily porphyria management. The annualized rate of porphyria attacks is a composite endpoint which included porphyria attacks requiring hospitalization, urgent healthcare visit, or IV hemin administration at home. (NCT03338816)
Timeframe: 6 months
| Intervention | annualized attack rate (Mean) |
|---|---|
| Placebo | 12.26 |
| Givosiran 2.5 mg/kg | 3.35 |
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale*week (Median) |
|---|---|
| Placebo | 5.286 |
| Givosiran 2.5 mg/kg | -11.514 |
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Placebo | -4.011 |
| Givosiran 2.5 mg/kg | 1.481 |
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the post baseline weekly mean score minus the baseline score. Lower scores indicate an improvement. The 6-month AUC was calculated based on change from baseline in weekly mean scores. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale*week (Least Squares Mean) |
|---|---|
| Placebo | -4.208 |
| Givosiran 2.5 mg/kg | -11.148 |
Participants rated worst daily nausea score in an eDiary using an 11-point NRS, in which 0=no nausea and 10=worst nausea. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Placebo | -0.181 |
| Givosiran 2.5 mg/kg | 0.067 |
Participants rated daily worst fatigue score in an eDiary using the 11-point BFI-SF NRS, in which 0=no fatigue and 10=worst fatigue. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Placebo | -0.182 |
| Givosiran 2.5 mg/kg | -0.502 |
Participants rated worst daily pain score in an eDiary using the 11-point BPI-SF NRS, in which 0=no pain and 10=worst pain. Daily eDiary entries were averaged into a weekly (i.e. 7 day) score. The change from baseline in weekly mean scores is defined as the postbaseline weekly mean score minus the baseline score. Lower scores indicate an improvement. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale (Median) |
|---|---|
| Placebo | 0.245 |
| Givosiran 2.5 mg/kg | -0.506 |
The SF-12 is a survey designed for use in patients with multiple chronic conditions. This 12-item scale can be used to assess the physical and mental health of respondents. 10 of the 12 questions are answered on a 5 point likert scale and 2 are answered on a 3 point likert scale. The questions are then scored and weighted into 2 subscales, physical health and mental health. Respondents can have a score that ranges from 0-100 with 100 being the best score and indicating high physical or mental health. A 3 point change in SF-12 score reflects a meaningful difference. A higher score indicates improvement. (NCT03338816)
Timeframe: Baseline and 6 months
| Intervention | score on a scale (Least Squares Mean) |
|---|---|
| Placebo | 1.431 |
| Givosiran 2.5 mg/kg | 5.369 |
The PD effect of givosiran was evaluated by spot urine PBG levels normalized to spot urine creatinine levels. (NCT03338816)
Timeframe: 6 months
| Intervention | mmol/mol Cr (Least Squares Mean) |
|---|---|
| Placebo | 49.110 |
| Givosiran 2.5 mg/kg | 12.906 |
The PD effect of givosiran was evaluated by spot urine ALA levels normalized to spot urine creatinine levels. (NCT03338816)
Timeframe: 3 and 6 months
| Intervention | mmol/mol creatinine (Cr) (Least Squares Mean) | |
|---|---|---|
| Month 3 | Month 6 | |
| Givosiran 2.5 mg/kg | 1.756 | 4.013 |
| Placebo | 19.965 | 23.150 |
2 trials available for porphobilinogen and Pain
| Article | Year |
|---|---|
Disease burden in patients with acute hepatic porphyria: experience from the phase 3 ENVISION study.
Topics: Cost of Illness; Hemin; Humans; Pain; Porphobilinogen; Porphobilinogen Synthase; Porphyria, Acute In | 2022 |
Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria.
Topics: Acetylgalactosamine; Adult; Aminolevulinic Acid; Double-Blind Method; Fatigue; Female; Humans; Injec | 2020 |
6 other studies available for porphobilinogen and Pain
| Article | Year |
|---|---|
Peritoneal dialysis resulting in discontinuance of recurring attacks of acute intermittent porphyria: A case report.
Topics: Aged; Heme; Humans; Male; Pain; Peritoneal Dialysis; Porphobilinogen; Porphyria, Acute Intermittent; | 2022 |
Letter: Variegate porphyria.
Topics: Abdomen; Adult; Cholestyramine Resin; Coproporphyrins; Feces; Female; Humans; Pain; Plasmapheresis; | 1975 |
Acute intermittent porphyria. A perplexing case.
Topics: Acute Disease; Aminolevulinic Acid; Female; Humans; Middle Aged; Nervous System Diseases; Pain; Porp | 1992 |
Letter: Toxicity of dimethylformamide.
Topics: Abdomen; Dimethylformamide; Environmental Exposure; Humans; Pain; Porphobilinogen | 1974 |
Dimethylformamide-induced abdominal pain and liver injury.
Topics: Abdomen; Abdomen, Acute; Accidents, Occupational; Chemical and Drug Induced Liver Injury; Dimethylfo | 1973 |
Acute intermittent porphyria in childhood. A neglected diagnosis?
Topics: Abdomen; Acute Disease; Child; Color; Humans; Hypertension; Hyponatremia; Male; Pain; Porphobilinoge | 1974 |