Biomarker & Intervention Discovery

Hypothyroidism

Pharmacologically actionable gene targets, therapeutic agents ranked by evidence tier, and active clinical trials.

US DALYs1.70MWHO GHE 2021 · all ages
Global DALYs13.92MWHO GHE 2021 · worldwide
US mortality57.7K deaths/yrWHO GHE 2021
Global mortality221.3K deaths/yrWHO GHE 2021 · worldwide
Spontaneous remissionHashimoto's (most common cause) is irreversible in majority; spontaneous recovery in <5%; subclinical hypothyroidism…
Best-intervention remissionT4 monotherapy normalizes TSH in nearly all patients and resolves symptoms in ~60%; up to 40% of T4-treated patients…
Gap sizeModerate — within the treated population, a large minority are inadequately treated by the standard-of-care T4…
Primary barrierframing/practice
Full remission profile →

Remission & chronicity

Spontaneous remission
Hashimoto's (most common cause) is irreversible in majority; spontaneous recovery in <5%; subclinical hypothyroidism spontaneously normalizes in ~30-40% of cases within 2 years without treatment
Best-intervention remission
T4 monotherapy normalizes TSH in nearly all patients and resolves symptoms in ~60%; up to 40% of T4-treated patients remain symptomatic despite normalized TSH; T4+T3 combination or desiccated thyroid extract (DTE) preferred by ~50% of patients in blinded preference trials
Gap size
Moderate — within the treated population, a large minority are inadequately treated by the standard-of-care T4 monotherapy approach
Primary barrier
framing/practice
Up to 40% of patients remain symptomatic on T4 despite guideline-defined adequate treatment; T3 combination and DTE are rarely offered in primary care despite guideline acknowledgment that a trial may be appropriate; TSH-only monitoring misses tissue-level hypothyroidism
Biomarker Targets & Therapeutic Agents

Gene targets queried against DGIdb, Open Targets, ChEMBL, PubMed, and Europe PMC. Agents ranked: ● Clinical > ● Mechanistic > ● Correlative.

CTLA4 32DIO1 1DIO2 0TG 31THRA 146THRB 253TPO 55TSHR 294
Therapeutic Agent Effect Direction Evidence Tier Potency Source
TREMELIMUMAB inhibits Mechanistic
IPILIMUMAB inhibits Mechanistic
CYCLOSPORINE ? unclear Mechanistic
ATEZOLIZUMAB ? unclear Mechanistic
VISILIZUMAB ? unclear Mechanistic
BLEOMYCIN ? unclear Mechanistic
ANTIBIOTIC ? unclear Mechanistic
METHIMAZOLE ? unclear Mechanistic
RETRO-INVERSO PEPTIDE ? unclear Mechanistic
MELPHALAN ? unclear Mechanistic
RECOMBINANT FAS LIGAND ? unclear Mechanistic
ZALIFRELIMAB inhibits Mechanistic
RIBAVIRIN ? unclear Mechanistic
ANTI-THYMOCYTE GLOBULIN ? unclear Mechanistic
QUAVONLIMAB inhibits Mechanistic
COMPOUND 32 [PMID: 20471253] inhibits Mechanistic
SIROLIMUS ? unclear Mechanistic
BISPECIFIC ANTIBODY ? unclear Mechanistic
CADONILIMAB inhibits Mechanistic
APLITHIANINE A inhibits Mechanistic
PEGINTERFERON ALFA-2A ? unclear Mechanistic
INOSINE 5'-MONOPHOSPHATE DEHYDROGENASE INHIBITOR FF-10501-01 ? unclear Mechanistic
ABATACEPT ? unclear Mechanistic
WORTMANNIN ? unclear Mechanistic
ERFONRILIMAB inhibits Mechanistic
THALIDOMIDE ? unclear Mechanistic
FF-10501-01 ? unclear Mechanistic
PEGINTERFERON ALFA-2B ? unclear Mechanistic
TETRADECANOYLPHORBOL ACETATE ? unclear Mechanistic
DEHYDRATED ALCOHOL ? unclear Mechanistic
PREDNISONE ? unclear Mechanistic
CHEMBL5291170 inhibits Mechanistic 31000.0 nM (Kd)
Therapeutic Agent Effect Direction Evidence Tier Potency Source
PROPYLTHIOURACIL inhibits Mechanistic

Pipeline results not yet available for this target. Run the pipeline to populate.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
GENISTEIN ? unclear Mechanistic
VITAMIN A ? unclear Mechanistic
RECOMBINANT TRANSFORMING GROWTH FACTOR-BETA 1 ? unclear Mechanistic
DEXAMETHASONE ? unclear Mechanistic
ACEBUTOLOL ? unclear Mechanistic
AZACITIDINE ? unclear Mechanistic
VACCINE ? unclear Mechanistic
AMINOGLYCOSIDE ANTIBIOTIC ? unclear Mechanistic
FLUORODEOXYGLUCOSE F18 ? unclear Mechanistic
CALCITRIOL ? unclear Mechanistic
PREDNISONE ? unclear Mechanistic
HEXAMETHYLENEBISACETAMIDE ? unclear Mechanistic
PREDNISOLONE ? unclear Mechanistic
ALDESLEUKIN ? unclear Mechanistic
PEGINTERFERON ALFA-2A ? unclear Mechanistic
OCTREOTIDE ACETATE ? unclear Mechanistic
DEHYDRATED ALCOHOL ? unclear Mechanistic
ISOTRETINOIN ? unclear Mechanistic
RETINOIC ACID AGENT ? unclear Mechanistic
GAMMA-INTERFERON ? unclear Mechanistic
PHENYTOIN SODIUM ? unclear Mechanistic
AMIODARONE HYDROCHLORIDE ? unclear Mechanistic
HEXAMETHYLENE BISACETAMIDE ? unclear Mechanistic
POLYETHYLENE GLYCOL 4000 ? unclear Mechanistic
ROSIGLITAZONE ? unclear Mechanistic
TECHNETIUM TC 99M SESTAMIBI ? unclear Mechanistic
BISPECIFIC ANTIBODY ? unclear Mechanistic
AMINOGLUTETHIMIDE ? unclear Mechanistic
ANTIDEPRESSANT AGENT ? unclear Mechanistic
ADENOVIRUS VECTOR ? unclear Mechanistic
TRETINOIN ? unclear Mechanistic
Therapeutic Agent Effect Direction Evidence Tier Potency Source
ASPIRIN ? unclear Mechanistic
LIOTHYRONINE activates Mechanistic 0.1 nM (Kd)
LEVOTHYROXINE activates Mechanistic
LIOTHYRONINE SODIUM activates Mechanistic
TIRATRICOL ? unclear Mechanistic 0.14 nM (IC50)
DEXTROTHYROXINE activates Mechanistic
DEXTROTHYROXINE SODIUM activates Mechanistic
LEVOTHYROXINE SODIUM ? unclear Mechanistic
CHEMBL111953 inhibits Mechanistic 490.0 nM (Kd)
CHEMBL109430 inhibits Mechanistic 5.2 nM (Kd)
CHEMBL109279 inhibits Mechanistic 93.0 nM (Kd)
CHEMBL110158 inhibits Mechanistic 330.0 nM (Kd)
SOBETIROME inhibits Mechanistic 1.8 nM (Kd)
CHEMBL112087 inhibits Mechanistic 200.0 nM (Kd)
CHEMBL83953 inhibits Mechanistic 39000.0 nM (IC50)
CHEMBL86271 inhibits Mechanistic 1800.0 nM (IC50)
CHEMBL82131 inhibits Mechanistic 18100.0 nM (IC50)
CHEMBL85322 inhibits Mechanistic 69000.0 nM (IC50)
CHEMBL83829 inhibits Mechanistic 930.0 nM (IC50)
CHEMBL97187 inhibits Mechanistic 1400.0 nM (Kd)
CHEMBL318132 inhibits Mechanistic 112.0 nM (Kd)
CHEMBL97613 inhibits Mechanistic 430.0 nM (Kd)
CHEMBL98135 inhibits Mechanistic 1400.0 nM (Kd)
CHEMBL264527 inhibits Mechanistic 240.0 nM (Kd)
CHEMBL160883 inhibits Mechanistic 820.0 nM (IC50)
CHEMBL160703 inhibits Mechanistic 1400.0 nM (IC50)
AMIODARONE inhibits Mechanistic 650.0 nM (IC50)
CHEMBL157885 inhibits Mechanistic 4500.0 nM (IC50)
DETROTHYRONINE inhibits Mechanistic 0.24 nM (IC50)
CHEMBL39174 inhibits Mechanistic 1.4 nM (IC50)
THYROPROPIC ACID inhibits Mechanistic 0.041 nM (IC50)
CHEMBL40738 inhibits Mechanistic 130.0 nM (IC50)
CHEMBL289965 inhibits Mechanistic 9.7 nM (IC50)
CHEMBL41036 inhibits Mechanistic 25.0 nM (IC50)
CHEMBL289343 inhibits Mechanistic 0.1 nM (IC50)
CHEMBL418432 inhibits Mechanistic 0.76 nM (IC50)
CHEMBL41314 inhibits Mechanistic 0.14 nM (IC50)
CHEMBL125946 activates Mechanistic 1500.0 nM (EC50)
CHEMBL124205 activates Mechanistic 12.0 nM (EC50)
CHEMBL419016 inhibits Mechanistic 2.4 nM (Kd)

Showing top 40 of 146 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
METHYL PARATHIONE ? unclear Mechanistic
DIENOCHLOR ? unclear Mechanistic
AXITIROME ? unclear Mechanistic
3-METHYLCHOLANTHRENE ? unclear Mechanistic
CHEMBL:CHEMBL577415 ? unclear Mechanistic
DYCLONINE HYDROCHLORIDE ? unclear Mechanistic
LEVOTHYROXINE activates Mechanistic
ORTHO-PHTHALALDEHYDE ? unclear Mechanistic
QUINALIZARIN ? unclear Mechanistic
CHEMBL:CHEMBL297304 ? unclear Mechanistic
CHEMBL:CHEMBL1493528 ? unclear Mechanistic
RESMETIROM activates Mechanistic
CLOSANTEL ? unclear Mechanistic
IDARUBICIN ? unclear Mechanistic
FUMARPROTOCETRARIC ACID ? unclear Mechanistic
DIPYRIDAMOLE ? unclear Mechanistic
CHEMBL:CHEMBL1341830 ? unclear Mechanistic
CARUBICIN ? unclear Mechanistic
ALBUTEROL SULFATE ? unclear Mechanistic
PRIMAQUINE ? unclear Mechanistic
DEXTROTHYROXINE SODIUM activates Mechanistic
SOBETIROME activates Mechanistic 0.1 nM (Kd)
METHYLENE BLUE ANHYDROUS ? unclear Mechanistic
HEXACHLOROPHENE ? unclear Mechanistic
CHEMBL:CHEMBL1465049 ? unclear Mechanistic
COMPOUND 16G [PMID: 35507418] activates Mechanistic
CHLOROTHALONIL ? unclear Mechanistic
SLAZINIC ACID ? unclear Mechanistic
TRIAMTERENE ? unclear Mechanistic
CHEMBL:CHEMBL601952 ? unclear Mechanistic
CHEMBL:CHEMBL578741 ? unclear Mechanistic
CIRAZOLINE ? unclear Mechanistic
CHEMBL:CHEMBL581044 ? unclear Mechanistic
DAUNORUBICIN HYDROCHLORIDE ? unclear Mechanistic
RT3 activates Mechanistic
CHEMBL:CHEMBL592124 ? unclear Mechanistic
VK-2809 activates Mechanistic
CHEMBL:CHEMBL602969 ? unclear Mechanistic
EPROTIROME activates Mechanistic
TOLONIUM CHLORIDE ? unclear Mechanistic

Showing top 40 of 253 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
CARBIMAZOLE inhibits Mechanistic
PROPYLTHIOURACIL inhibits Mechanistic 3380.0 nM (IC50)
TRETINOIN ? unclear Mechanistic
METHIMAZOLE inhibits Mechanistic
CHEMBL4278946 inhibits Mechanistic 3900.0 nM (IC50)
CHEMBL4282403 inhibits Mechanistic 6000.0 nM (IC50)
CHEMBL4482878 inhibits Mechanistic 4000.0 nM (IC50)
CHEMBL333928 inhibits Mechanistic 42000.0 nM (IC50)
CHEMBL4637728 inhibits Mechanistic 30000.0 nM (IC50)
CHEMBL4645343 inhibits Mechanistic 5300.0 nM (IC50)
CHEMBL4633000 inhibits Mechanistic 5900.0 nM (IC50)
CHEMBL4753981 inhibits Mechanistic 2200.0 nM (IC50)
CHEMBL4792720 inhibits Mechanistic 6300.0 nM (IC50)
CHEMBL4751166 inhibits Mechanistic 59000.0 nM (IC50)
CHEMBL4755252 inhibits Mechanistic 12300.0 nM (IC50)
CHEMBL4869433 inhibits Mechanistic 700.0 nM (IC50)
CHEMBL4859908 inhibits Mechanistic 640.0 nM (IC50)
CHEMBL4855030 inhibits Mechanistic 2000.0 nM (IC50)
CHEMBL4855931 inhibits Mechanistic 710.0 nM (IC50)
CHEMBL4846080 inhibits Mechanistic 2300.0 nM (IC50)
CHEMBL4853722 inhibits Mechanistic 1100.0 nM (IC50)
CHEMBL4863015 inhibits Mechanistic 1100.0 nM (IC50)
CHEMBL4871587 inhibits Mechanistic 1800.0 nM (IC50)
CHEMBL4855956 inhibits Mechanistic 6700.0 nM (IC50)
CHEMBL4878518 inhibits Mechanistic 1900.0 nM (IC50)
CHEMBL4862053 inhibits Mechanistic 1100.0 nM (IC50)
CHEMBL4860429 inhibits Mechanistic 1700.0 nM (IC50)
CHEMBL4877407 inhibits Mechanistic 14000.0 nM (IC50)
CHEMBL4859470 inhibits Mechanistic 21000.0 nM (IC50)
PF-06282999 inhibits Mechanistic 990.0 nM (IC50)
CHEMBL5200638 inhibits Mechanistic 590.0 nM (IC50)
CHEMBL5181827 inhibits Mechanistic 4200.0 nM (IC50)
CHEMBL5182747 inhibits Mechanistic 8100.0 nM (IC50)
CHEMBL5185576 inhibits Mechanistic 1500.0 nM (IC50)
CHEMBL5172456 inhibits Mechanistic 1300.0 nM (IC50)
CHEMBL5197968 inhibits Mechanistic 1390.0 nM (IC50)
CHEMBL5197383 inhibits Mechanistic 3400.0 nM (IC50)
CHEMBL5175091 inhibits Mechanistic 1400.0 nM (IC50)
CHEMBL5207346 inhibits Mechanistic 4200.0 nM (IC50)
CHEMBL5186129 inhibits Mechanistic 1900.0 nM (IC50)

Showing top 40 of 55 agents ranked by evidence tier.

Therapeutic Agent Effect Direction Evidence Tier Potency Source
PENTACHLOROPHENOL ? unclear Mechanistic
THYROTROPIN ALFA activates Mechanistic
CILOSTAZOL ? unclear Mechanistic
ALPHA-NAPHTHOFLAVONE ? unclear Mechanistic
GRAMINE ? unclear Mechanistic
TESTOSTERONE PROPIONATE ? unclear Mechanistic
SB 222200 ? unclear Mechanistic
URAPIDIL ? unclear Mechanistic
CHEMBL:CHEMBL312032 ? unclear Mechanistic
CHEMBL:CHEMBL578276 ? unclear Mechanistic
CHEMBL:CHEMBL606166 ? unclear Mechanistic
AM-630 ? unclear Mechanistic
CHEMBL:CHEMBL607309 ? unclear Mechanistic
LEVALBUTEROL ? unclear Mechanistic
CHEMBL:CHEMBL140220 ? unclear Mechanistic
ICI-199441 ? unclear Mechanistic
LASALOCID A SODIUM ? unclear Mechanistic
CLOSANTEL ? unclear Mechanistic
DIPYRIDAMOLE ? unclear Mechanistic
ANTISERUM ? unclear Mechanistic
ISOPROTERENOL ? unclear Mechanistic
SEROTONIN ? unclear Mechanistic
NMDA ? unclear Mechanistic
BERGAMOTTIN ? unclear Mechanistic
3,6-DIMETHOXYFLAVONE ? unclear Mechanistic
L-733,060 ? unclear Mechanistic
TYRPHOSTIN AG 879 ? unclear Mechanistic
CARBONYL CYANIDE M-CHLOROPHENYLHYDRAZONE ? unclear Mechanistic
CP-339818 ? unclear Mechanistic
DICHLOROPHEN ? unclear Mechanistic
CLORGILINE ? unclear Mechanistic
DEXPROPRANOLOL ? unclear Mechanistic
TYRPHOSTIN A9 ? unclear Mechanistic
CHEMBL:CHEMBL429095 ? unclear Mechanistic
CLIOXANIDE ? unclear Mechanistic
PIPERONYL BUTOXIDE ? unclear Mechanistic
CHEMBL:CHEMBL1456848 ? unclear Mechanistic
KARANJIN ? unclear Mechanistic
THYROTROPIN activates Mechanistic
KINETIN ? unclear Mechanistic

Showing top 40 of 294 agents ranked by evidence tier.


Clinical Trials

Recruiting and recently completed trials from ClinicalTrials.gov. Data retrieved 2026-07-06.

No recent clinical trials found on ClinicalTrials.gov for this condition.


Agents Found By Disease-Level Search

Searched directly from “Hypothyroidism” via ClinicalTrials.gov interventions and Open Targets' disease→drug data — independent of the 8-gene target panel above. This surfaces agents whose mechanism doesn't route through a curated gene (combination therapies, standard-of-care drugs, targets outside the panel).

40 agents shown (approved or with trial evidence) — 35 not found by the gene-target search above.

Levothyroxine

Approved · 11 trials · source: ClinicalTrials.gov, Open Targets
NCT04124705 NCT05412979 NCT01950260

levothyroxine sodium

Approved · 3 trials · source: ClinicalTrials.gov, Open Targets
NCT05823012 NCT00921050 NCT03880578

L-carnitineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01769157

Armour® ThyroidNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04124705

ST-1891NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05412979

Quality of life assessment, biochemical measuresNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05015725

MNP groupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02280330

Quantitative POC TSH KitNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01921452

Qualitative POC TSH KitNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01921452

Third generation TSH KitNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01921452

Liothyronine, Sodium

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT01800617

LT3 (liothyronine)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05682482

Mini gastric bypass surgeryNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06015620

fatigue assessment and managementNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00336102

management of therapy complicationsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00336102

Eltroxin administrationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06154382

ThyroxineNOT in gene-target list

Unknown · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT05385029

Serum thyroid function testsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04860622

excipient without levothyroxine (placebo)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00921050

TRH (Thyrotropin Releasing Hormone)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00054756

Assesment of thyroïd functionNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT04831957

Liothyronine

Approved · 1 trial · source: ClinicalTrials.gov, Open Targets
NCT04782856

Thyroid function testNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT05033496

D-THIO (Deprescribing Thyroid Hormone In Older Adults)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06629272

Enhanced usual careNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06629272

L-thyroxineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00524238

Clinical Pharmacist Intervention GroupNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06408909

Thyroid Function MonitoringNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT07048548

Levothyroxine/Liothyronine once dailyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06731764

Levothyroxine/Liothyronine twice dailyNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT06731764

Levothyroxine sodium new formulationNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT01916304

Brand Name Levothyroxine (Synthroid)NOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00403390

Generic formulation of LevothyroxineNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT00403390

iodinated contrast agentsNOT in gene-target list

· 1 trial · source: ClinicalTrials.gov
NCT02766283

THYROTROPIN

Approved · 0 trials · source: Open Targets

OMEGA-3-ACID ETHYL ESTERSNOT in gene-target list

Approved · 0 trials · source: Open Targets

CHOLESTYRAMINENOT in gene-target list

Approved · 0 trials · source: Open Targets

FENOFIBRATENOT in gene-target list

Approved · 0 trials · source: Open Targets

MECASERMINNOT in gene-target list

Approved · 0 trials · source: Open Targets

THYROGLOBULINNOT in gene-target list

Approved · 0 trials · source: Open Targets

Part of the Open Source Medicine Foundation Network

Open Source Medicine Foundation The OSMF hub — advancing open-source biomedical research infrastructure, tools, and evidence synthesis.
SpikeProtein.site Database and research resource on SARS-CoV-2 spike protein biology, persistence, and clinical implications.
Vaccine Data Navigator Open-access navigation tool for vaccine adverse event reporting and surveillance data.
PACVS Research Summit Annual summit convening researchers, clinicians, and patients on PACVS science.