Drugs / Betaine
Betaine
also known as
Betaine Hydrochloride · trimethylglycine · Abromine · Acidol pepsin · Amversio · anhydrous betaine HCl · Betaine anhydrous · betaine HCl · +6 more
Small molecule targets PEX1 via restoration
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Cystadane | — | 2007-02-14 | europa.eu ↗ |
| Approved | US (FDA) | CYSTADANE | — | 1996-10-25 | fda.gov ↗ |
Trials 15 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 01 trial | ||||||
| Phase 0 | NCT00882193 | May 2009 → Jan 2011 | chronic hepatitis C virus infection | University of Nebraska | Terminated | No outcome recorded |
| Phase 13 trials | ||||||
| Phase 1 | NCT02122211 | Apr 2014 → Jul 2016 | male infertility | University of North Carolina, Chapel Hill | Completed | No outcome recorded |
| Phase 1 | NCT01759875 | Jan 2013 → Jan 2014 | — | University of California, San Francisco | Completed | No outcome recorded |
| Phase 1 | NCT01398046 | Aug 2011 → Feb 2014 | — | University of California, San Francisco | Completed | No outcome recorded |
| Phase 24 trials | ||||||
| Phase 2 | NCT07276204 | Mar 2026 → Dec 2029 expected | metabolic dysfunction-associated steatohepatitis | Southern California Institute for Research and Education | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT06308367 | Mar 2024 → Mar 2027 expected | syringomyelia | Xuanwu Hospital, Beijing | Recruiting | No outcome recorded |
| Phase 2 | NCT02404337 | Jul 2015 → Dec 2017 | homocystinuria | Assistance Publique - Hôpitaux de Paris | Completed | No outcome recorded |
| Phase 2 | NCT00283387 | Feb 2007 → Sep 2011 | primary hyperoxaluria type 1 | Mayo Clinic | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT00310336 | Aug 2006 → Aug 2009 | hepatitis C virus infection | University Hospital, Basel, Switzerland | Completed | No outcome recorded |
| Phase 32 trials | ||||||
| Phase 3 | NCT01838941 | Mar 2013 → Jan 2015 | peroxisome biogenesis disorder | McGill University Health Centre/Research Institute of the McGill University Health Centre | Completed | No outcome recorded |
| Phase 3 | NCT01371357 | May → Dec 2011 | hyperhomocysteinemia | Center for Health Sciences, Serbia | Completed | No outcome recorded |
| Phase 42 trials | ||||||
| Phase 4 | NCT01749982 | Dec 2012 → Mar 2013 | — | Columbia University | Completed | No outcome recorded |
| Phase 4 | NCT00226356 | Dec 2004 → Jun 2007 | major depressive disorder | Cambridge Health Alliance | Completed | No outcome recorded |
| Phase not applicable2 trials | ||||||
| — | NCT05585476 | Oct 2025 → Jun 2026 overdue | female breast carcinoma, female reproductive system disorder | Instituto de Investigación Sanitaria de la Fundación Jiménez Díaz | Not yet recruiting | No outcome recorded |
| — | NCT00348933 | Jul 2006 → Feb 2010 | Angelman syndrome | University of California, San Diego | Completed | No outcome recorded |
Also used as a comparator or background therapy in 3 trials
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 22 trials | ||||||
| Phase 2 | NCT01950039 Comparator | Jan 2014 → Dec 2017 | glucose metabolism disease, obesity disorder | Joslin Diabetes Center | Completed | No outcome recorded |
| Phase 2 | NCT00586911 Comparator | Jan 2003 → Jun 2006 | metabolic dysfunction-associated steatohepatitis | Mayo Clinic | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT00571714 Comparator | Apr 2008 → Mar 2010 | chronic hepatitis C virus infection | University of Nebraska | Withdrawn | No outcome recorded |
Evidence & citations 19 cited values
Every value below carries the sentence it was read from. 20 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Betaine | “different dietary agents were used in the past for the treatment of elevated total plasma homocysteine (T-HCy), e. g. betaine, choline (betaine precursor) or folic acid.” NCT01371357 ↗ |
| Known as | Abromine | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | Acidol pepsin | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | Amversio | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | anhydrous betaine HCl | “This pilot study evaluated the extent and time course of gastric reacidification after a solid oral dosage form of anhydrous betaine HCl in healthy volunteers with...” PMID 23980906 ↗ Sep 2013 |
| Known as | Betaine anhydrous | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | betaine HCl | “After gastric pH > 4 was confirmed for 15 min, 1500 mg of betaine HCl was given orally with 90 mL of water and gastric pH was continuously monitored for 2 h.” PMID 23980906 ↗ Sep 2013 |
| Known as | Betaine Hydrochloride | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01398046 ↗ |
| Known as | Betainum muriaticum | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | Cystadane | ClinicalTrials.gov intervention name — accepted as the source's own label NCT00586911 ↗ |
| Known as | FEMA NO. 4223 | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | NSC-166511 | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | NSC-32191 | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | NSC-3976 | ChEMBL registry synonym — accepted as the source's own label CHEMBL95889 ↗ |
| Known as | trimethylglycine | “Betaine, or trimethylglycine, is a Health Canada and FDA approved orphan drug for the treatment of homocystinuria” NCT01838941 ↗1“It is a moisturizing and regenerating gel whose active ingredients are olive oil, trimethylglycine and xylitol.” NCT05585476 ↗ |
| Action | Restore | “we reported the recovery of peroxisome functions by treatment with Betaine” NCT01838941 ↗ |
| Modality | Small molecule | — CHEMBL95889 ↗ |
| Route | Oral | “Subjects were randomly assigned oral betaine 12 grams/day in subjects younger than 10 years of age, and 20 grams/day in subjects 10 years of age and older, in two divided doses.” NCT00283387 ↗ |
| Target | PEX1 | “acting as a nonspecific chemical chaperone for the misfolded PEX1 protein” NCT01838941 ↗ |