Betaine
drugOn this page
Also known as AbromineAcidol pepsinAmversioBetaine anhydrousanhydrousCystadaneFEMA NO. 4223NSC-166511SID11110845SID90341438GlycinebetaineGlycine betaineSID144211476SID144207060SID174007375
Summary
Betaine (CHEMBL95889) is an approved small molecule (ATC A16AA06); indicated across 10 conditions including homocystinuria and depressive disorder.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- ATC class: A16AA06
- Indications: 10 conditions
- Clinical trials: 29
- Chemistry: 118.15 Da · C5H12NO2+
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL95889 |
| Name | Betaine |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | no |
| PubChem CID | 248 |
| ChEBI | CHEBI:41139 |
| ATC | A16AA06 |
| Molecular formula | C5H12NO2+ |
| Molecular weight | 118.15 |
| InChIKey | KWIUHFFTVRNATP-UHFFFAOYSA-O |
SMILES: C[N+](C)(C)CC(=O)O
IUPAC name: carboxymethyl(trimethyl)azanium
ChEBI definition: A quaternary ammonium ion in which the substituents on nitrogen are methyl (three) and carboxymethyl.
Other ChEBI roles (chemical / environmental): fundamental metabolite.
Also known as: Abromine, Acidol pepsin, Amversio, Betaine, Betaine anhydrous, anhydrous, Cystadane, FEMA NO. 4223, NSC-166511, SID11110845, SID90341438, betaine
Parent form; salt/anhydrous children: CHEMBL1201130
Patent coverage: 2,369 distinct patent families (5,997 SureChEMBL compound mentions), from 1 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Thyrotropin receptor.
Bioactivity
ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| TSHR | 8.4 | Potency | 4 | nM | CHEMBL_ACT_3922759 |
| TSHR | 8.4 | Potency | 4 | nM | CHEMBL_ACT_4618646 |
Target pathways
No target-pathway data for this drug (no mapped target genes).
Indications & clinical
Indications
10 indications (2 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).
| Indication | Trial phase | MONDO | EFO |
|---|---|---|---|
| homocystinuria | 4 | MONDO:0004737 | MONDO:0004737 |
| depressive disorder | 4 | MONDO:0002050 | MONDO:0002009 |
| obesity disorder | 2 | MONDO:0011122 | EFO:0001073 |
| hepatitis C virus infection | 2 | MONDO:0005231 | EFO:0003047 |
| metabolic dysfunction-associated steatohepatitis | 2 | MONDO:0007027 | EFO:1001249 |
| primary hyperoxaluria | 2 | MONDO:0002474 | MONDO:0002474 |
| syringomyelia | 2 | MONDO:0017987 | MONDO:0017987 |
| male infertility | 1 | MONDO:0005372 | EFO:0004248 |
| chronic hepatitis C virus infection | 0 | MONDO:0005354 | EFO:0004220 |
1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.
Clinical trials
Total trials: 29.
Phase distribution
| Phase | Trials |
|---|---|
| Not specified | 16 |
| PHASE2 | 6 |
| PHASE2/PHASE3 | 2 |
| PHASE4 | 1 |
| PHASE3 | 1 |
| PHASE1/PHASE2 | 1 |
| EARLY_PHASE1 | 1 |
| PHASE1 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT01749982 | PHASE4 | COMPLETED | A Preliminary Study of Choline and Betaine Supplementation Among Adults Exposed to Arsenic in Bangladesh |
| NCT00310336 | PHASE2/PHASE3 | COMPLETED | Chronic Hepatitis C Non-Responder Study With AdoMet and Betaine |
| NCT01838941 | PHASE3 | COMPLETED | Betaine and Peroxisome Biogenesis Disorders |
| NCT03702205 | PHASE2/PHASE3 | UNKNOWN | Betaine Effect in Speed-strength Athletes |
| NCT04633044 | PHASE1/PHASE2 | ACTIVE_NOT_RECRUITING | Maternal Betaine Supplementation During Breastfeeding |
| NCT06308367 | PHASE2 | RECRUITING | The Therapeutic Effect of Betaine in Syringomyelia |
| NCT07276204 | PHASE2 | NOT_YET_RECRUITING | Betaine vs. Placebo for Serologically Diagnosed Metabolic Dysfunction-associated Steatohepatitis (MASH) |
| NCT00283387 | PHASE2 | COMPLETED | Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria |
| NCT00586911 | PHASE2 | COMPLETED | Betaine in Patients With Nonalcoholic Steatohepatitis |
| NCT01950039 | PHASE2 | COMPLETED | Metabolic Effects of Betaine Supplementation |
| NCT02404337 | PHASE2 | COMPLETED | Betaine METABOLISM OF PATIENTS With Homocystinuria |
| NCT02122211 | PHASE1 | COMPLETED | Choline Dehydrogenase and Sperm Function: Effects of Betaine |
| NCT00882193 | EARLY_PHASE1 | TERMINATED | Pilot Study of Betaine + Combination Antiviral Therapy for Chronic Hepatitis C Genotype 1 Non-responder/Relapsers |
| NCT03073343 | Not specified | RECRUITING | A Prospective, Randomized, Open Label Trial of Two Doses of Oral Betaine |
| NCT06881511 | Not specified | RECRUITING | Exploratory Study on the Efficacy of Betaine Hydrochloride in Treating Autoimmune Gastritis |
| NCT07144527 | Not specified | RECRUITING | Nicotinamide Mononucleotide Supplementation for Exercise Tolerance Improvement in Healthy Older Adults |
| NCT07243678 | Not specified | ACTIVE_NOT_RECRUITING | Pharmacokinetic Assessment of Betaine Supplementation in Lactating Mothers |
| NCT07607106 | Not specified | RECRUITING | Efficacy of a New Composition (BU002) for Debridement of Chronic Cutaneous Ulcers |
| NCT00102843 | Not specified | COMPLETED | Effect of Betaine and Folic Acid on Vascular Function in Healthy Humans |
| NCT00348933 | Not specified | COMPLETED | Dietary Supplements for the Treatment of Angelman Syndrome |
| NCT00571714 | Not specified | WITHDRAWN | Pilot Comparison of Standard Antiviral Therapy With and Without 12 Weeks of Betaine in Genotype 1 Naive Patients |
| NCT01213719 | Not specified | COMPLETED | Effects of Betaine Supplementation on Strength and Muscle Creatine Content |
| NCT02118142 | Not specified | COMPLETED | Modulation of Insulin Sensitivity by Betaine Upregulation of FGF21 |
| NCT02758015 | Not specified | COMPLETED | Effect of Betaine and Food on Gastric pH |
| NCT04585295 | Not specified | COMPLETED | The Effects of Pre-Loaded Betaine Supplementation on Thermoregulation and Exercise Metabolism in the Heat |
| NCT05790070 | Not specified | COMPLETED | Potential Synergistic Effect of Combined Blood Flow Restriction Training and Betaine Supplementation on Skeletal Muscle |
| NCT06042270 | Not specified | COMPLETED | Firefighter Thermoregulatory Responses in Active Fire with Betaine Supplementation |
| NCT06344377 | Not specified | COMPLETED | Betaine Supplementation and Lipid Metabolism in Overweight and Obese Pre-menopausal Women |
| NCT06392360 | Not specified | COMPLETED | Betaine, Exercise Performance, and Gut Permeability |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).
Related Atlas pages
- Diseases: homocystinuria, depressive disorder