Lipoic Acid, Alpha

Summary

Lipoic Acid, Alpha (CHEMBL33864) is an approved small-molecule geroprotector (ATC A16AX01); indicated across 31 conditions including stroke disorder and diabetic neuropathy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A16AX01
• Indications: 31 conditions
• Clinical trials: 89
• Chemistry: 206.3 Da · C8H14O2S2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL33864
Name	Lipoic Acid, Alpha
Type	Small molecule
Max phase	3
FDA approved	yes
PubChem CID	864
ChEBI	CHEBI:16494
ATC	A16AX01
Molecular formula	C8H14O2S2
Molecular weight	206.3
InChIKey	AGBQKNBQESQNJD-UHFFFAOYSA-N

SMILES: C1CSSC1CCCCC(=O)O

IUPAC name: 5-(dithiolan-3-yl)pentanoic acid

ChEBI definition: A heterocyclic thia fatty acid comprising pentanoic acid with a 1,2-dithiolan-3-yl group at the 5-position.

Pharmacological roles (ChEBI): geroprotector.

Other ChEBI roles (chemical / environmental): fundamental metabolite.

Also known as: .alpha.-lipoic acid, A-lipoicum acidum, Acidum thiocticum, Alpha lipoic acid, Alpha-lipoic acid, Biletan, Lipoic acid, alpha, dl-, NSC-628502, NSC-90788, Thiocacid

Patent coverage: 8,127 distinct patent families (19,339 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: 20 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Lethal(3)malignant brain tumor-like protein 1, Nuclear receptor ROR-gamma, Prelamin-A/C, ATP-dependent DNA helicase Q1, RecQ-like DNA helicase BLM, Endonuclease 4, Bifunctional protein GlmU, Peripheral myelin protein 22, Thyroid hormone receptor beta.

Bioactivity

ChEMBL activities: 16 potent at pChembl ≥ 5 of 31 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
ACHE	9	IC50	1	nM	CHEMBL_ACT_2077379
TDP1	6.46	Potency	344.4	nM	CHEMBL_ACT_3935562
LMNA	6.45	Potency	354.8	nM	CHEMBL_ACT_3665644
TDP1	6.4	Potency	398.1	nM	CHEMBL_ACT_3928909
APEX1	6.3	Potency	501.2	nM	CHEMBL_ACT_3798955
SAE1	6.21	IC50	620	nM	CHEMBL_ACT_5882689
THRB	6.05	Potency	891.3	nM	CHEMBL_ACT_4016200
P0A6C1	6.05	Potency	891.3	nM	CHEMBL_ACT_4069650
POLB	5.95	Potency	1122	nM	CHEMBL_ACT_5035174
NR1I2	5.55	EC50	2800	nM	CHEMBL_ACT_15463845
RECQL	5.55	Potency	2818	nM	CHEMBL_ACT_3693068
L3MBTL1	5.5	Potency	3162	nM	CHEMBL_ACT_4624274
P9WMN3	5.41	IC50	3910	nM	CHEMBL_ACT_3606264
SAE1	5.32	IC50	4740	nM	CHEMBL_ACT_5540943
BLM	5.1	Potency	7943	nM	CHEMBL_ACT_4753873
BLM	5.1	Potency	7943	nM	CHEMBL_ACT_4945725

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

31 indications (0 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
stroke disorder	3	MONDO:0005098	EFO:0000712
diabetic neuropathy	3	MONDO:0006626	MONDO:0001583
type 1 diabetes mellitus	3	MONDO:0005147	MONDO:0005147
kidney disorder	3	MONDO:0005240	EFO:0003086
carpal tunnel syndrome	2	MONDO:0007275	EFO:0004143
chronic progressive multiple sclerosis	2	MONDO:0005284	EFO:0003840
AIDS	2	MONDO:0012268	EFO:0000765
chronic kidney disease	2	MONDO:0005300	EFO:0003884
primary progressive multiple sclerosis	2	MONDO:0000451	EFO:0008520
secondary progressive multiple sclerosis	2	MONDO:0000450	EFO:0008522
Guillain-Barre syndrome, familial	2	MONDO:0007691	EFO:0009538
obstructive sleep apnea syndrome	2	MONDO:0007147	EFO:0003918
mucositis	2	MONDO:0020579	EFO:1001898
type 2 diabetes mellitus	2	MONDO:0005148	MONDO:0005148
multiple sclerosis	2	MONDO:0005301	MONDO:0005301
cystinuria	2	MONDO:0009067	MONDO:0009067
adrenoleukodystrophy	2	MONDO:0018544	MONDO:0015339
severe cutaneous adverse reaction	2	MONDO:0005594	EFO:0006346
bipolar disorder	2	MONDO:0004985	MONDO:0004985
HIV infectious disease	2	MONDO:0005109	EFO:0000764
peripheral neuropathy	1	MONDO:0005244	EFO:0003100
optic neuritis	1	MONDO:0005885	EFO:0007405
age-related macular degeneration	1	MONDO:0005150	EFO:0001365
hypertensive disorder	1	MONDO:0005044	EFO:0000537
Alzheimer disease	1	MONDO:0004975	MONDO:0004975
progressive supranuclear palsy	1	MONDO:0019037	MONDO:0019037
head and neck squamous cell carcinoma	1	MONDO:0010150	EFO:0000181

4 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 89.

Phase distribution

Phase	Trials
Not specified	24
PHASE2	18
PHASE4	13
PHASE3	9
PHASE1/PHASE2	9
PHASE2/PHASE3	7
PHASE1	7
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT01056497	PHASE4	COMPLETED	The Effect of A-lipoic Acid (ALA) on Fatty Acid-induced Impairment of Glucose-stimulated Insulin Secretion
NCT01406704	PHASE4	TERMINATED	Effects of Rosiglitazone and Alpha-lipoic Acid on the Patients With Pathologically Proved NASH
NCT01524861	PHASE4	COMPLETED	Sympathetic Heart Innervation in Patients With Tako-Tsubo Cardiomyopathy
NCT01650181	PHASE4	COMPLETED	Effects of Siliphos-Selenium-Methionine-Alpha Lipoic Acid in Patients With Fatty Liver and Non-alcoholic Steatohepatitis
NCT01877590	PHASE4	COMPLETED	Alpha-lipoic Acid Reduces Left Ventricular Mass in Normotensive Type 2 Diabetic Patients With Coronary Artery Disease
NCT01978405	PHASE4	UNKNOWN	Short-Term Alpha Lipoic Acid Therapy for Prevention of Contrast-induced Acute Kidney Injury and Endothelial Dysfunction in Type 2 Diabetes
NCT02439879	PHASE4	COMPLETED	Alpha Lipoic Acid for Treatment of Diabetic Neuropathy
NCT02601898	PHASE4	COMPLETED	Vaginal Administration of ALA vs Progesterone for the Subchorionic Hematoma Treatment
NCT03023514	PHASE4	COMPLETED	Lipoic Acid Supplementation in IVF
NCT03914404	PHASE4	COMPLETED	Efficacy of γ-linolenic Acid and Thioctic Acid in Patients With Diabetic Neuropathy
NCT04064814	PHASE4	COMPLETED	Evaluation of Efficacy and Safety of add-on Alpha-lipoic Acid on Migraine Prophylaxis in Adolescent Population
NCT04846673	PHASE4	COMPLETED	Pregabalin and Alpha-lipoic Acid Combination Versus Each Monotherapy in Patients With Diabetic Peripheral Neuropathy
NCT05266794	PHASE4	COMPLETED	A Novel Role of Alpha Lipoic Acid in Comparison With Selenium in Mitochondrial Resuscitation and miRNA-126 Expression in Hemodialysis Patients
NCT06201611	PHASE2/PHASE3	RECRUITING	Evaluating a Nitric Oxide Generator, Nebivolol as a Disease Modifier in Patients With Diabetic Neuropathy.
NCT06286098	PHASE3	ENROLLING_BY_INVITATION	Alpha Lipoic Acid in Pediatrics on Hemodialysis
NCT07456176	PHASE3	NOT_YET_RECRUITING	Effect of Low-Dose Versus High-Dose Alpha-Lipoic Acid on Oxidative Stress, Inflammation, and Clinical Outcomes in Acute Exacerbations of Chronic Obstructive Pulmonary Disease
NCT00112996	PHASE3	COMPLETED	Alpha-Lipoic Acid in Preventing Peripheral Neuropathy in Patients Receiving Chemotherapy for Cancer
NCT00328601	PHASE3	COMPLETED	Assessment of Efficacy and Safety of Thioctic Acid in the Oral Treatment of Symptomatic Diabetic Neuropathy (SYDNEY 2)
NCT00477607	PHASE2/PHASE3	COMPLETED	Alpha-Lipoic Acid in Preventing Hearing Loss in Cancer Patients Undergoing Treatment With Cisplatin
NCT00977483	PHASE3	COMPLETED	Assessment of Efficacy and Safety of Thioctic Acid in the Oral Treatment of Diabetic Polyneuropathy (Stage 1 or 2)
NCT00994513	PHASE2/PHASE3	COMPLETED	Effect of Alpha Lipoic Acid on Obesity Related Comorbidities
NCT01188811	PHASE2/PHASE3	COMPLETED	Lipoic Acid for Secondary Progressive Multiple Sclerosis (MS)
NCT01224353	PHASE2/PHASE3	UNKNOWN	Efficacy and Safety Study of Thioctacid Oral Tablets 600mg to Treat Chinese Diabetic Patients With Distal Symmetric Polyneuropathy
NCT01888861	PHASE3	UNKNOWN	Effect of Alpha-lipoic Acid on Biochemical Markers and Important Outcomes in Patients Admitted to Intensive Care Units
NCT03788759	PHASE2/PHASE3	COMPLETED	Alpha-lipoic Acid Adjunctive Therapy in Schizophrenia
NCT04041167	PHASE3	UNKNOWN	Impact of Lipoic Acid Use on Stroke Outcome After Reperfusion Therapy in Patients With Diabetes (IMPORTANT)
NCT05369793	PHASE3	COMPLETED	Clinical Study Evaluating the Safety and Efficacy of Roflumilast in Type 2 Diabetic Patients With Diabetic Neuropathy
NCT05808946	PHASE2/PHASE3	UNKNOWN	Alpha-Lipoic Acid in Patients With Sepsis
NCT06418347	PHASE3	UNKNOWN	Effect of ALA Combined With Letrozole on Polycystic Ovary Syndrome Clinical Outcome in Infertile Females
NCT05713799	PHASE2	NOT_YET_RECRUITING	Trial of the Combination of Alpha-Lipoic Acid and Mirabegron in Women and in Men With Obesity
NCT06568185	PHASE2	RECRUITING	The Effect of Alpha Lipoid Acid and Vitamin B Preparation on Diabetic Polyneuropathy in Type 2 Diabetes Mellitus Patient
NCT07574684	PHASE2	NOT_YET_RECRUITING	Clinical Effects of Alpha-Lipoic Acid in Advanced NSCLC
NCT00002154	PHASE2	COMPLETED	A Study of Thioctic Acid and Deprenyl in HIV-Infected Patients With Dementia
NCT00010842	PHASE1/PHASE2	COMPLETED	Natural Antioxidants in the Treatment of Multiple Sclerosis
NCT00033176	PHASE2	COMPLETED	Immune Restoration by Lipoic Acid in AIDS
NCT00065624	PHASE2	COMPLETED	Antioxidant Functions of Lipoic Acid
NCT00079807	PHASE1/PHASE2	COMPLETED	Painful HIV Neuropathy and Alpha-Lipoic Acid
NCT00090402	PHASE1/PHASE2	COMPLETED	Fish Oil and Alpha Lipoic Acid in Treating Alzheimer’s Disease
NCT00237718	PHASE2	COMPLETED	Provision of Antioxidant Therapy in Hemodialysis (PATH) Study
NCT00308971	PHASE2	COMPLETED	Tocopherols and Alpha Lipoic Acid Treatment Chronic Kidney Disease (TALAT)

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).