Disease · MONDO_0019065
amyloidosis
6 sourcesdiseases-2026-09-18-0.1.0
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ConductScience Library. amyloidosis. Diseases, record MONDO_0019065. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019065
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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.
- Other names
- amyloid, amyloid disease, amyloidoses, amyloidosis (disease), systemic amyloidosis
- Open Targets
- Highest stage of its drugs
- Approval, for 7 of its 55 drugs, from DailyMed, EMA Human Drugs, FDA and TTD
- Open Targets
Drugs55
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 |
|---|---|---|---|---|---|---|---|---|
| PATISIRAN | CHEMBL3989987 | Oligonucleotide | Approval | TTD | 3 | 2 | 2 | Drugs and targets |
| TAFAMIDIS | CHEMBL2103837 | Small molecule | Approval | DailyMed, EMA Human Drugs and FDA | 4 | 0 | 0 | Drugs and targets |
| TAFAMIDIS MEGLUMINE | CHEMBL2105675 | Small molecule | Approval | FDA | 2 | 0 | 0 | Drugs and targets |
| INOTERSEN | CHEMBL4297770 | Oligonucleotide | Approval | EMA Human Drugs | 1 | 0 | 0 | Drugs and targets |
| INOTERSEN SODIUM | CHEMBL4297537 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| IXAZOMIB CITRATE | CHEMBL3545432 | Small molecule | Approval | TTD | 1 | 0 | 0 | Drugs and targets |
| PATISIRAN SODIUM | CHEMBL4297536 | Oligonucleotide | Approval | DailyMed | 1 | 0 | 0 | Drugs and targets |
| DEXAMETHASONE | CHEMBL384467 | Small molecule | Phase 3 | ClinicalTrials.gov | 11 | 11 | 11 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 6 | 6 | Drugs and targets |
| MELPHALAN | CHEMBL852 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 6 | 6 | Drugs and targets |
1 to 10 of 55
See all 55 drugsTargets91
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 | 12 | Transthyretin mRNA RNAi inhibitor; Transthyretin mRNA antisense inhibitor; Transthyretin mRNA rnai inhibitor; Transthyretin stabiliser | Approval | https://conductscience.org/library/genes/7276 | Drugs and targets |
| ADRM1 | 26S proteasome | 3 | 26S proteasome inhibitor | Approval | https://conductscience.org/library/genes/11047 | Drugs and targets |
| CRBN | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 2 | https://conductscience.org/library/genes/51185 | Drugs and targets |
| CSF3R | Granulocyte colony-stimulating factor receptor | 3 | Granulocyte colony stimulating factor receptor agonist | Phase 2 | https://conductscience.org/library/genes/1441 | Drugs and targets |
| CUL4A | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 2 | https://conductscience.org/library/genes/8451 | Drugs and targets |
| DDB1 | CRL4(CRBN) E3 ubiquitin ligase | 3 | CRL4(CRBN) E3 ubiquitin ligase inhibitor; CRL4(CRBN) E3 ubiquitin ligase modulator | Phase 2 | https://conductscience.org/library/genes/1642 | Drugs and targets |
| PSMA1 | 26S proteasome | 3 | 26S proteasome inhibitor | Approval | https://conductscience.org/library/genes/5682 | Drugs and targets |
| PSMA2 | 26S proteasome | 3 | 26S proteasome inhibitor | Approval | https://conductscience.org/library/genes/5683 | Drugs and targets |
| PSMA3 | 26S proteasome | 3 | 26S proteasome inhibitor | Approval | https://conductscience.org/library/genes/5684 | Drugs and targets |
| PSMA4 | 26S proteasome | 3 | 26S proteasome inhibitor | Approval | https://conductscience.org/library/genes/5685 | Drugs and targets |
1 to 10 of 91
See all 91 targetsTrials35
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. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00558896 | CC-4047 and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma or Amyloidosis | Phase 2 | 378 | 2014-06-05 | Multiple Myeloma and Plasma Cell Neoplasm | DEXAMETHASONE, POMALIDOMIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02510261 | The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran) | Phase 3 | 211 | 2023-12-06 | Amyloidosis | PATISIRAN | Its record names the disease | ClinicalTrials.gov |
| NCT00536601 | High-Dose Chemotherapy With or Without Total-Body Irradiation Followed by Autologous Stem Cell Transplant in Treating Patients With Hematologic Cancer or Solid Tumors | Not applicable | 174 | 2021-04-22 | Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Del(5q), Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22), Adult Acute Myeloid Leukemia With t(8;21)(q22;q22), Adult Nasal Type Extranodal NK/T-cell Lymphoma, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission, Childhood Burkitt Lymphoma, Childhood Diffuse Large Cell Lymphoma, Childhood Immunoblastic Large Cell Lymphoma, Childhood Nasal Type Extranodal NK/T-cell Lymphoma, Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor (PNET), Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue, Hepatosplenic T-cell Lymphoma, Intraocular Lymphoma, Nodal Marginal Zone B-cell Lymphoma, Peripheral T-cell Lymphoma, Plasma Cell Neoplasm, Primary Systemic Amyloidosis, Recurrent Adult Acute Lymphoblastic Leukemia, Recurrent Adult Acute Myeloid Leukemia, Recurrent Adult Burkitt Lymphoma, Recurrent Adult Diffuse Large Cell Lymphoma, Recurrent Adult Diffuse Mixed Cell Lymphoma, Recurrent Adult Diffuse Small Cleaved Cell Lymphoma, Recurrent Adult Grade III Lymphomatoid Granulomatosis, Recurrent Adult Hodgkin Lymphoma, Recurrent Adult Immunoblastic Large Cell Lymphoma, Recurrent Adult Lymphoblastic Lymphoma, Recurrent Childhood Acute Lymphoblastic Leukemia, Recurrent Childhood Acute Myeloid Leukemia, Recurrent Childhood Anaplastic Large Cell Lymphoma, Recurrent Childhood Grade III Lymphomatoid Granulomatosis, Recurrent Childhood Large Cell Lymphoma, Recurrent Childhood Lymphoblastic Lymphoma, Recurrent Childhood Small Noncleaved Cell Lymphoma, Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma, Recurrent Ewing Sarcoma/Peripheral Primitive Neuroectodermal Tumor, Recurrent Grade 1 Follicular Lymphoma, Recurrent Grade 2 Follicular Lymphoma, Recurrent Grade 3 Follicular Lymphoma, Recurrent Malignant Testicular Germ Cell Tumor, Recurrent Mantle Cell Lymphoma, Recurrent Marginal Zone Lymphoma, Recurrent Neuroblastoma, Recurrent Small Lymphocytic Lymphoma, Recurrent/Refractory Childhood Hodgkin Lymphoma, Refractory Chronic Lymphocytic Leukemia, Refractory Multiple Myeloma, Regional Neuroblastoma, Splenic Marginal Zone Lymphoma, Testicular Lymphoma, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific, Waldenström Macroglobulinemia | BUSULFAN, CARBOPLATIN, CARMUSTINE, CYCLOPHOSPHAMIDE, ETOPOSIDE, MELPHALAN, THIOTEPA | Its record names the disease | ClinicalTrials.gov |
| NCT00651937 | Trial of Two Stem Cell Doses To Reduce Transplant Induced Symptom Burden | Phase 2 | 80 | 2020-01-14 | Multiple Myeloma, Primary Amyloidosis | MELPHALAN | Its record names the disease | ClinicalTrials.gov |
| NCT01855360 | Doxycycline and TUDCA in Patients With Transthyretin Amyloid Cardiomyopathy | Phase 1/2 | 40 | 2020-12-04 | Amyloidosis; Heart (Manifestation), Senile Cardiac Amyloidosis | DOXYCYCLINE, TAURURSODIOL | Its record names the disease | ClinicalTrials.gov |
| NCT01570387 | A Phase I/II Trial of Pomalidomide and Dexamethasone in Subjects With Previously-Treated AL Amyloidosis | Phase 1/2 | 27 | 2019-12-16 | AL Amyloidosis | DEXAMETHASONE, POMALIDOMIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02994784 | Propylene Glycol-Free Melphalan Hydrochloride (Evomela) in AL Amyloidosis Patients | Phase 2 | 27 | 2023-05-24 | Amyloidosis; Systemic | MELPHALAN HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01677286 | Safety and Effect of Doxycycline in Patients With Amyloidosis | Phase 2 | 25 | 2017-06-06 | Amyloidosis | DOXYCYCLINE | Its record names the disease | ClinicalTrials.gov |
| NCT04304144 | A Study to Evaluate the Safety and Tolerability of CAEL-101 in Patients With AL Amyloidosis | Phase 2 | 25 | 2025-03-05 | AL Amyloidosis | ANSELAMIMAB, BORTEZOMIB, CYCLOPHOSPHAMIDE, DARATUMUMAB, DEXAMETHASONE | Its record names the disease | ClinicalTrials.gov |
| NCT02603172 | A Safety Study of GSK3039294 in Healthy Volunteers and Patients With Systemic Amyloidosis | Phase 1 | 23 | 2019-09-23 | Amyloidosis | GSK-294 | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 35
See all 35 trialsComparisons in its trials15
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 |
|---|---|---|---|---|---|
| Standard Dose Stem Cell Group vs High Dose Stem Cell Group: deaths from any cause | 0 of 35 | 0 of 40 | 75 | NCT00651937 | Trial comparisons |
| Standard Dose Stem Cell Group vs High Dose Stem Cell Group: serious adverse events | 1 of 35 | 0 of 40 | 75 | NCT00651937 | Trial comparisons |
| Amyloidosis vs Monoclonal Ig DepositionDisease (MIDD): Participants Evaluated for Toxicity | 19 of 19 | 1 of 1 | 20 | NCT01383759 | Trial comparisons |
| Amyloidosis vs Monoclonal Ig DepositionDisease (MIDD): deaths from any cause | 7 of 19 | 0 of 1 | 20 | NCT01383759 | Trial comparisons |
| Amyloidosis vs Monoclonal Ig DepositionDisease (MIDD): serious adverse events | 17 of 19 | 1 of 1 | 20 | NCT01383759 | Trial comparisons |
| Dose Level 0- Safety Population vs Dose Level 1- Safety Population: Time to Amyloidotic Organ Response Based on Reported Data. | 0 of 3 | 0 of 7 | 10 | NCT02545907 | Trial comparisons |
| Dose Level 0- Safety Population vs Dose Level 1- Safety Population: Number of Patients Progression-free at 6 Months Based on Reported Data. | 3 of 3 | 7 of 7 | 10 | NCT02545907 | Trial comparisons |
| Dose Level 0- Safety Population vs Dose Level 1- Safety Population: Number of Patients Experiencing Dose Delays Based on Reported Data. | 2 of 3 | 1 of 7 | 10 | NCT02545907 | Trial comparisons |
| Dose Level 0- Safety Population vs Dose Level 1- Safety Population: Compliance Profile of KTD Based on Reported Chemotherapy Compliance Data. | 1 of 3 | 5 of 7 | 10 | NCT02545907 | Trial comparisons |
| Dose Level 0- Safety Population vs Dose Level 1- Safety Population: Number of Patients Withdrawing From Treatment Based on Reported Data. | 0 of 3 | 0 of 7 | 10 | NCT02545907 | Trial comparisons |
1 to 10 of 15
See all 15 comparisons in its trialsPapers from its trials15
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 | Source |
|---|---|---|---|
| 42212672 | DERIVED | 2 | PubMed |
| 40226119 | DERIVED | 1 | PubMed |
| 39804640 | DERIVED | 1 | PubMed |
| 38548524 | DERIVED | 1 | PubMed |
| 37607644 | DERIVED | 1 | PubMed |
| 35810311 | DERIVED | 1 | PubMed |
| 33212063 | DERIVED | 1 | PubMed |
| 31119947 | DERIVED | 1 | PubMed |
| 29298867 | DERIVED | 1 | PubMed |
| 27381904 | DERIVED | 1 | PubMed |
1 to 10 of 15
See all 15 papers from its trialsData 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/MONDO_0019065
GET https://conductscience.org/api/diseases/v1/MONDO_0019065/drugs?page=2