Drugs / PYRIDOXAL PHOSPHATE ANHYDROUS
PYRIDOXAL PHOSPHATE ANHYDROUS
Trials 10
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT03738943 | Feb 2019 → Jun 2021 | — | Colorado State University | Completed | No outcome recorded |
| Phase 11 trial | ||||||
| Phase 1 | NCT05366933 | Aug 2022 → Jul 2027 expected | optic nerve disorder | National Aeronautics and Space Administration (NASA) | Recruiting | No outcome recorded |
| Phase 24 trials | ||||||
| Phase 2 | NCT01281878 | Dec 2010 → Sep 2012 | primary hyperoxaluria type 1 | University of Cologne | Completed | No outcome recorded |
| Phase 2 | NCT00917293 | May 2009 → Aug 2012 | tardive dyskinesia | Medicure | Terminated | No outcome recorded |
| Phase 2 | NCT00157729 | Aug 2004 → Jul 2005 | hypertensive disorder, metabolic syndrome X, type 2 diabetes mellitus | Medicure | Completed | No outcome recorded |
| Phase 2 | NCT00157716 | Apr 2004 → Oct 2005 | coronary artery disorder, myocardial ischemia | Medicure | Completed | No outcome recorded |
| Phase 32 trials · 1 missed | ||||||
| Phase 3 | NCT04706013 | Feb 2024 → Nov 2026 expected | inborn disorder of pyridoxine metabolism | Medicure | Recruiting | No outcome recorded |
| Phase 3 | NCT00402506 | Nov 2006 → Sep 2007 | myocardial ischemia | Medicure | Completed | Missed primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT04762316 | Jan 2019 → Dec 2025 | uterine corpus leiomyoma | Trieu, Nguyen Thi, M.D. | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT07469462 | Mar → Sep 2026 | anxiety, depressive disorder | University of Reading | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2025-09-23 | Medicure | Results MEDICURE ANNOUNCES UPDATE ON ENROLLMENT IN ITS MEND-PNPO PHASE 3 CLINICAL TRIAL AND PRESENTATION OF THE TRIAL DESIGN AND BASELINE DATA AT THE 2025 CHILD NEUROLOGY SOCIETY CONFERENCE medicure.com ↗
Based on enrolled patients, baseline P5P treatment regimens ranged between 14 mg/kg/day to 58 mg/kg/day, and baseline steady state P5P levels were also variable between subjects ranging from 4.84 ng/mL to 136 ng/mL. |
| 2024-04-23 | Medicure | Regulatory MEDICURE RECEIVES US FDA FAST TRACK DESIGNATION FOR MC-1 FOR PNPO DEFICIENCY medicure.com ↗ |
| 2023-11-23 | Medicure | Regulatory MEDICURE ANNOUNCES FDA PROVIDES COMPLETE APPROVAL TO ENROLL PATIENTS IN ITS PIVOTAL PHASE 3 TRIAL FOR TREATMENT OF RARE PEDIATRIC DISEASE medicure.com ↗
today announced that the FDA provides complete approval to enroll patients in its pivotal Phase 3 clinical trial to evaluate the use of its investigational product MC-1 for treatment of a rare pediatric disease called pyridox(am)ine 5'-phosphate oxidase (PNPO) deficiency. |
| 2021-01-07 | Medicure | Regulatory Medicure Announces Pivotal Phase 3 Trial IND Filing with FDA for Treatment of Seizures Associated with Pyridox(am)ine 5'-phosphate oxidase (PNPO) Deficiency medicure.com ↗
The FDA and the European Medicines Agency ("EMA") have both granted a Rare Pediatric Disease Designation to MC-1 for the treatment of seizures associated with PNPO deficiency. |
All press releases naming this drug 4 releases
Evidence & citations 13 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | PYRIDOXAL PHOSPHATE ANHYDROUS | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | MC-1 | “(MC-1) Pyridoxal-5'-phosphate” NCT00157716 ↗ |
| Known as | NSC-82388 | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | Pyridoxal 5'-Phosphate | “Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency” NCT04706013 ↗ |
| Known as | Pyridoxal 5-phosphate monohydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | PYRIDOXAL CALCIUM PHOSPHATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | Pyridoxal phosphate hydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | Pyridoxal phosphate monohydrate | ChEMBL registry synonym — accepted as the source's own label CHEMBL82202 ↗ |
| Known as | pyridoxal-phosphate | “Oral solution of pyridoxal phosphate start with 5mg per kg body weight per day in two dosages over 6 weeks, increase stepwise by 5mg/kg body weight every 6 weeks up to 20 mg/kg...” NCT01281878 ↗4“Treatment with pyridoxal-phosphate in increasing dosages every six weeks starting with 5mg/kg body weight up to 20 mg/kg body weight.” NCT01281878 ↗ “Vitamin B-Complex Supplement containing: 5-methyltetrahydrofolate, riboflavin 5 phosphate, pyridoxal phosphate, and methylcobalamin” NCT05366933 ↗ “Pregnenolone 60mg & Pyridoxal Phosphate 20mg & Dydrogesterone 10mg & Impatien balsamina extract 100 mg” NCT04762316 ↗ |
| Action | Inhibit | “the purpose of this study is to determine whether Vitamin B6 or PLP can inhibit vasodilation” NCT03738943 ↗ |
| Modality | Other / unclassified | “daily nutraceutical supplement containing bioactive B vitamins” NCT05366933 ↗ |
| Modality | Small molecule | — CHEMBL82202 ↗ |
| Route | Oral | “Pyridoxal 5'-Phosphate 500mgs po bid for 12 weeks.” NCT00917293 ↗ |