Disease · EFO_1001984
cardiac amyloidosis
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. cardiac amyloidosis. Diseases, record EFO_1001984. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/EFO_1001984
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Name
- cardiac amyloidosis
- Open Targets ID
- EFO_1001984
- Other names
- Amyloid heart disease, Amyloid heart muscle disease, Amyloidosis of heart, Senile cardiac amyloidosis, amyloid cardiomyopathy
- Highest stage of its drugs
- Approval, for 1 of its 9 drugs, from PMDA
Source Open Targets
Drugs9
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| ACORAMIDIS HYDROCHLORIDE | CHEMBL4650226 | Small molecule | Approval | PMDA | 1 | 0 | 0 | Drugs and targets |
| DOXYCYCLINE | CHEMBL1200699 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| TAURURSODIOL | CHEMBL272427 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| PATISIRAN | CHEMBL3989987 | Oligonucleotide | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| TAFAMIDIS MEGLUMINE | CHEMBL2105675 | Small molecule | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| INOTERSEN | CHEMBL4297770 | Oligonucleotide | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| MIRIDESAP | CHEMBL25263 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| REVUSIRAN | CHEMBL4594492 | Oligonucleotide | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DAPAGLIFLOZIN | CHEMBL429910 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets7
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| TTR | Transthyretin | 5 | Transthyretin mRNA RNAi inhibitor; Transthyretin mRNA antisense inhibitor; Transthyretin stabiliser | Approval | https://conductscience.org/library/genes/7276 | Drugs and targets |
| APCS | Serum amyloid P-component | 1 | Serum amyloid P-component cross-linking agent | Phase 2 | https://conductscience.org/library/genes/325 | Drugs and targets |
| MMP1 | Interstitial collagenase | 1 | Matrix metalloproteinase-1 inhibitor | Phase 3 | https://conductscience.org/library/genes/4312 | Drugs and targets |
| MMP13 | Collagenase 3 | 1 | Matrix metalloproteinase 13 inhibitor | Phase 3 | https://conductscience.org/library/genes/4322 | Drugs and targets |
| MMP7 | Matrilysin | 1 | Matrix metalloproteinase 7 inhibitor | Phase 3 | https://conductscience.org/library/genes/4316 | Drugs and targets |
| MMP8 | Neutrophil collagenase | 1 | Matrix metalloproteinase 8 inhibitor | Phase 3 | https://conductscience.org/library/genes/4317 | Drugs and targets |
| SLC5A2 | Sodium/glucose cotransporter 2 | 1 | Sodium/glucose cotransporter 2 inhibitor | Unknown | https://conductscience.org/library/genes/6524 | Drugs and targets |
Trials7
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02207556 | Doxycycline to Upgrade Organ Response in Light Chain (AL) Amyloidosis Trial | Phase 2 | 32 | 2020-01-07 | Primary Systemic Amyloidosis | DOXYCYCLINE | Its record names the disease | ClinicalTrials.gov |
| NCT03044353 | Multiple Treatment Session Study to Assess GSK2398852 Administered Following and Along With GSK2315698 | Phase 2 | 7 | 2019-10-16 | Amyloidosis | MIRIDESAP | Its record names the disease | ClinicalTrials.gov |
| NCT03481972 | A Study of Doxycycline and Tauroursodeoxycholic Acid (Doxy/TUDCA) Plus Standard Supportive Therapy Versus Standard Supportive Therapy Alone in Cardiac Amyloidosis Caused by Transthyretin | Phase 3 | 102 | TTR Cardiac Amyloidosis | DOXYCYCLINE, TAURURSODIOL | Its record names the disease | ClinicalTrials.gov | |
| NCT05795400 | Administration of the SGLT-2 Inhibitor Dapagliflozin in the Patients With Amyloid Cardiomyopathy | Not applicable | 50 | Heart Failure With Reduced Ejection Fraction, Heart Failure With Preserved Ejection Fraction, Heart Failure With Mid Range Ejection Fraction, Amyloid Cardiomyopathy | DAPAGLIFLOZIN | Its record names the disease | ClinicalTrials.gov | |
| NCT03702829 | 24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients | Phase 2 | 31 | Amyloidosis | INOTERSEN | Its record names the disease | ClinicalTrials.gov | |
| NCT01981837 | Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis | Phase 2 | 26 | TTR-mediated Amyloidosis | REVUSIRAN | Its record names the disease | ClinicalTrials.gov | |
| NCT03474458 | A Trial of Doxycycline vs. Standard Supportive Therapy in Newly-diagnosed Cardiac AL Amyloidosis Patients Undergoing Bortezomib-based Therapy | Phase 2/3 | 19 | Cardiac AL Amyloidosis | DOXYCYCLINE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials9
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Doxycycline (Localized Disease) vs Doxycycline (Systemic Disease): deaths from any cause | 0 of 5 | 5 of 25 | 30 | NCT02207556 | Trial comparisons |
| Doxycycline (Localized Disease) vs Doxycycline (Systemic Disease): serious adverse events | 1 of 5 | 13 of 25 | 30 | NCT02207556 | Trial comparisons |
| Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: Number of Participants With Any Serious Adverse Events (SAEs) | 2 of 6 | 1 of 1 | 7 | NCT03044353 | Trial comparisons |
Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: Number of Participants With Abnormalities in Immunohistochemical Examination of Skin Biopsies | 2 of 6 | 0 of 1 | 7 | NCT03044353 | Trial comparisons |
| Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: Number of Participants With Skin Rashes | 4 of 6 | 0 of 1 | 7 | NCT03044353 | Trial comparisons |
Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: Number of Participants With Abnormalities in Histopathological Examination of Skin Biopsies | 2 of 6 | 0 of 1 | 7 | NCT03044353 | Trial comparisons |
| Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: Number of Participants With Any On-treatment Adverse Events (AEs) | 6 of 6 | 1 of 1 | 7 | NCT03044353 | Trial comparisons |
| Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: deaths from any cause | 0 of 6 | 0 of 1 | 7 | NCT03044353 | Trial comparisons |
| Group 1: Cardiac TTR Amyloidosis (ATTR-CM) Participants vs Group 2: Post-chemotherapy AL Amyloidosis Participants: serious adverse events | 2 of 6 | 1 of 1 | 7 | NCT03044353 | Trial comparisons |
Papers from its trials1
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it |
|---|---|---|
| 32529175 | DERIVED | 1 |
Source PubMed
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/EFO_1001984
GET https://conductscience.org/api/diseases/v1/EFO_1001984/drugs