Disease · MONDO_0017816

primary systemic amyloidosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. primary systemic amyloidosis. Diseases, record MONDO_0017816. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0017816

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: primary systemic amyloidosis; Open Targets
Open Targets ID: MONDO_0017816; Open Targets
Other names: systemic AL amyloidosis, systemic Immunoglobulin Light chain amyloidosis, systemic amyloidosis; Open Targets
NCI Thesaurus: C8299; NCI Thesaurus
Orphanet: ORPHA:314701; Orphanet
Highest stage of its drugs: Phase 3, for 3 of its 21 drugs, from ClinicalTrials.gov; Open Targets

Drugs21

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
MELPHALAN	CHEMBL852	Small molecule	Phase 3	ClinicalTrials.gov	12	12	12	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Phase 3	ClinicalTrials.gov	11	11	11	Drugs and targets
FILGRASTIM	CHEMBL1201567	Protein	Phase 3	ClinicalTrials.gov	6	6	6	Drugs and targets
LENALIDOMIDE	CHEMBL848	Small molecule	Phase 2	ClinicalTrials.gov	6	6	6	Drugs and targets
CYCLOPHOSPHAMIDE	CHEMBL1200796	Small molecule	Phase 2	ClinicalTrials.gov	5	5	5	Drugs and targets
THALIDOMIDE	CHEMBL468	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
INTERFERON ALFA	CHEMBL2108506	Protein	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
BORTEZOMIB	CHEMBL325041	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
BUSULFAN	CHEMBL820	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DOXORUBICIN	CHEMBL53463	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 21

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Targets54

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
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
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
RBX1	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/9978	Drugs and targets
APCS	Serum amyloid P-component	2	Serum amyloid P-component cross-linking agent; Serum amyloid P-component inhibitor	Phase 1	https://conductscience.org/library/genes/325	Drugs and targets
ADRM1	26S proteasome	1	26S proteasome inhibitor	Phase 2	https://conductscience.org/library/genes/11047	Drugs and targets
CD3D	T cell surface glycoprotein CD3	1	T cell surface glycoprotein CD3 binding agent	Unknown	https://conductscience.org/library/genes/915	Drugs and targets
CD3E	T cell surface glycoprotein CD3	1	T cell surface glycoprotein CD3 binding agent	Unknown	https://conductscience.org/library/genes/916	Drugs and targets
CD3G	T cell surface glycoprotein CD3	1	T cell surface glycoprotein CD3 binding agent	Unknown	https://conductscience.org/library/genes/917	Drugs and targets
CSF2RA	Granulocyte-macrophage colony-stimulating factor receptor	1	Granulocyte-macrophage colony-stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1438	Drugs and targets

1 to 10 of 54

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Trials25

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
NCT00064337	S0115, High-Dose Melphalan and Autologous Peripheral Stem Cell Transplantation in Treating Patients With Multiple Myeloma or Primary Systemic Amyloidosis	Phase 2	104	2017-11-06	Multiple Myeloma, Plasma Cell Myeloma	CYCLOPHOSPHAMIDE, DEXAMETHASONE, FILGRASTIM, MELPHALAN, THALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT00477971	Low-Dose Melphalan and Dexamethasone Compared With High-Dose Melphalan Followed By Autologous Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis	Phase 3	89	2015-07-21	Multiple Myeloma and Plasma Cell Neoplasm	DEXAMETHASONE, FILGRASTIM, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT00091260	CC-5013 With or Without Dexamethasone in Treating Patients With Primary Systemic Amyloidosis	Phase 2	82	2017-02-20	Multiple Myeloma	DEXAMETHASONE, LENALIDOMIDE	Its record names the disease	ClinicalTrials.gov
NCT00564889	Lenalidomide, Cyclophosphamide, and Dexamethasone in Treating Patients With Primary Systemic Amyloidosis	Phase 2	35	2011-08-12	Multiple Myeloma and Plasma Cell Neoplasm	CYCLOPHOSPHAMIDE, DEXAMETHASONE, LENALIDOMIDE	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
NCT00679367	Melphalan, Lenalidomide, and Dexamethasone in Treating Patients With Primary Systemic Amyloidosis	Phase 2	16	2017-02-20	Multiple Myeloma	DEXAMETHASONE, LENALIDOMIDE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT00075608	2nd Autologous Stem Cell Transplant in Patients With Persistent/Recurrent (AL) Amyloidosis	Phase 2	12	2017-01-27	Multiple Myeloma, Plasma Cell Neoplasm	FILGRASTIM, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT00790647	Melphalan, Bortezomib, and Stem Cell Transplant in Treating Patients With Primary Systemic Amyloidosis	Phase 2	10	2017-02-06	Multiple Myeloma	BORTEZOMIB, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT02158052	Combined Bone Marrow and Renal Transplantation for Hematologic Disorders With End Stage Renal Disease	Not applicable	2	2022-12-13	Multiple Myeloma, Amyloidosis	ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE), TACROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT00002849	S9628 Dexamethasone Plus Interferon Alfa in Treating Patients With Primary Systemic Amyloidosis	Phase 2	93		Multiple Myeloma	DEXAMETHASONE, INTERFERON ALFA	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials3

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
Low-Dose Melphalan vs High-Dose Melphalan + Autologous HSC: serious adverse events	6 of 34	3 of 55	89	NCT00477971	Trial comparisons
Bone Marrow and Kidney RECIPIENT vs Bone Marrow and Kidney DONOR: deaths from any cause	0 of 1	0 of 1	2	NCT02158052	Trial comparisons
Bone Marrow and Kidney RECIPIENT vs Bone Marrow and Kidney DONOR: serious adverse events	1 of 1	0 of 1	2	NCT02158052	Trial comparisons

Papers from its trials13

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
22504925	RESULT	1	PubMed
15308571	RESULT	1	PubMed
15156165	RESULT	1	PubMed
12000194	RESULT	1	PubMed
35810311	DERIVED	1	PubMed
29298867	DERIVED	1	PubMed
27872060	DERIVED	1	PubMed
26176329	DERIVED	1	PubMed
25974382	DERIVED	1	PubMed
24668858	DERIVED	1	PubMed

1 to 10 of 13

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Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0017816

GET https://conductscience.org/api/diseases/v1/MONDO_0017816/drugs?page=2

API reference For AI assistants