Disease · MONDO_0019438

AL amyloidosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. AL amyloidosis. Diseases, record MONDO_0019438. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019438

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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.

Name: AL amyloidosis; Open Targets
Open Targets ID: MONDO_0019438; Open Targets
Other names: Immunoglobulinic amyloidosis, Light-chain amyloidosis, Systemic monoclonal immunoglobulin light chain amyloidosis, primary amyloidosis, systemic AL amyloidosis; Open Targets
Disease Ontology: DOID:0080933; Disease Ontology
MeSH: C531616; MeSH
NCI Thesaurus: C158963; NCI Thesaurus
Orphanet: ORPHA:85443; Orphanet
Highest stage of its drugs: Approval, for 2 of its 29 drugs, from FDA; Open Targets

Drugs29

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
TAFAMIDIS	CHEMBL2103837	Small molecule	Approval	FDA	2	0	0	Drugs and targets
TAFAMIDIS MEGLUMINE	CHEMBL2105675	Small molecule	Approval	FDA	2	0	0	Drugs and targets
DEXAMETHASONE	CHEMBL384467	Small molecule	Phase 3	ClinicalTrials.gov	28	28	28	Drugs and targets
BORTEZOMIB	CHEMBL325041	Small molecule	Phase 3	ClinicalTrials.gov	14	14	14	Drugs and targets
DARATUMUMAB	CHEMBL1743007	Antibody	Phase 3	ClinicalTrials.gov	13	13	13	Drugs and targets
MELPHALAN	CHEMBL852	Small molecule	Phase 3	ClinicalTrials.gov	8	8	8	Drugs and targets
BIRTAMIMAB	CHEMBL3833345	Antibody	Phase 3	ClinicalTrials.gov and TTD	6	5	5	Drugs and targets
COAGULATION FACTOR X HUMAN	CHEMBL4297951		Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
ANSELAMIMAB	CHEMBL5315016	Antibody	Phase 3	TTD	2	0	0	Drugs and targets
DOXYCYCLINE	CHEMBL1200699	Small molecule	Phase 2/3	ClinicalTrials.gov	2	2	2	Drugs and targets

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Targets59

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
ADRM1	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/11047	Drugs and targets
PSMA1	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5682	Drugs and targets
PSMA2	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5683	Drugs and targets
PSMA3	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5684	Drugs and targets
PSMA4	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5685	Drugs and targets
PSMA5	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5686	Drugs and targets
PSMA6	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5687	Drugs and targets
PSMA7	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5688	Drugs and targets
PSMA8	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/143471	Drugs and targets
PSMB1	26S proteasome	4	26S proteasome inhibitor	Phase 3	https://conductscience.org/library/genes/5689	Drugs and targets

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Trials53

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
NCT02312206	The VITAL Amyloidosis Study, a Global Phase 3, Efficacy and Safety Study of NEOD001 in Patients With AL Amyloidosis	Phase 3	260	2019-05-29	Primary Systemic (AL) Amyloidosis	BIRTAMIMAB	Its record names the disease	ClinicalTrials.gov
NCT04973137	A Study to Evaluate the Efficacy and Safety of Birtamimab in Mayo Stage IV Patients With AL Amyloidosis	Phase 3	208	2025-12-16	Light Chain (AL) Amyloidosis	BIRTAMIMAB	Its record names the disease	ClinicalTrials.gov
NCT02632786	The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis	Phase 2	129	2019-04-05	AL Amyloidosis	BIRTAMIMAB	Its record names the disease	ClinicalTrials.gov
NCT01864018	Ixazomib With Cyclophosphamide and Dexamethasone in Patients With Previously Untreated Symptomatic Multiple Myeloma or Light Chain Amyloidosis	Phase 1/2	87	2022-09-21	Amyloidosis, Plasma Cell Myeloma	CYCLOPHOSPHAMIDE, DEXAMETHASONE, IXAZOMIB CITRATE	Its record names the disease	ClinicalTrials.gov
NCT00298766	Open-Label Phase 1/2 Study of VELCADE for Injection in Patients With Light-chain (AL)-Amyloidosis	Phase 1/2	70	2010-08-13	Amyloidosis	BORTEZOMIB	Its record names the disease	ClinicalTrials.gov
NCT03499808	S1702 Isatuximab in Treating Patients With Relapsed or Refractory Primary Amyloidosis	Phase 2	43	2025-02-27	Amorphous, Eosinophilic, and Acellular Deposit, Constipation, Diarrhea, Early Satiety, Gastrointestinal Hemorrhage, Hepatomegaly, Lymphadenopathy, Macroglossia, Nausea, Primary Systemic Amyloidosis, Purpura, Recurrent Primary Amyloidosis, Refractory Primary Amyloidosis	ISATUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT00520767	Bortezomib, Melphalan, and Dexamethasone in Treating Patients With Primary Amyloidosis or Light Chain Deposition Disease	Phase 2	35	2015-03-30	Primary Systemic Amyloidosis, Light Chain Deposition Disease	BORTEZOMIB, DEXAMETHASONE, MELPHALAN	Its record names the disease	ClinicalTrials.gov
NCT01083316	Bortezomib and Dexamethasone Followed by High-Dose Melphalan and Stem Cell Transplantation for Primary (AL) Amyloidosis	Phase 2	35	2017-02-06	Amyloidosis	BORTEZOMIB, DEXAMETHASONE, MELPHALAN	Open Targets' reading of its record	ClinicalTrials.gov
NCT02613182	Open-label Extension Study of NEOD001 in Subjects With Light Chain (AL) Amyloidosis	Phase 2	34	2019-05-16	AL Amyloidosis	BIRTAMIMAB	Its record names the disease	ClinicalTrials.gov
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

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Comparisons in its trials13

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
NEOD001 (24 mg/kg) + Standard of Care vs Placebo + Standard of Care: deaths from any cause	41 of 130	42 of 130	260	NCT02312206	Trial comparisons
NEOD001 (24 mg/kg) + Standard of Care vs Placebo + Standard of Care: serious adverse events	88 of 130	91 of 130	260	NCT02312206	Trial comparisons
NEOD001 24 mg/kg vs Placebo: deaths from any cause	3 of 66	6 of 63	129	NCT02632786	Trial comparisons
NEOD001 24 mg/kg vs Placebo: serious adverse events	14 of 66	15 of 63	129	NCT02632786	Trial comparisons
Phase I/II, Cohort A vs Phase II, Cohort B: deaths from any cause	2 of 48	7 of 35	83	NCT01864018	Trial comparisons
Phase I/II, Cohort A vs Phase II, Cohort B: Number of Patients Experiencing a Grade 3 or Greater Adverse Event at Least Possibly Related to Treatment as Assessed by the National Cancer Institute Common Terminology Criteria for Adverse Events Version 4.0	36 of 48	16 of 35	83	NCT01864018	Trial comparisons
Phase I/II, Cohort A vs Phase II, Cohort B: serious adverse events	16 of 48	15 of 35	83	NCT01864018	Trial comparisons
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
Melphalan vs Melphalan + Bortezomib: deaths from any cause	1 of 2	1 of 1	3	NCT02489500	Trial comparisons

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Papers from its trials20

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
20085941	RESULT	1	PubMed
42312318	DERIVED	1	PubMed
42296025	DERIVED	1	PubMed
40925099	DERIVED	1	PubMed
40226119	DERIVED	2	PubMed
38600883	DERIVED	1	PubMed
37366170	DERIVED	2	PubMed
35737873	DERIVED	1	PubMed
32730181	DERIVED	1	PubMed
32529175	DERIVED	1	PubMed

1 to 10 of 20

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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_0019438

GET https://conductscience.org/api/diseases/v1/MONDO_0019438/drugs?page=2

API reference For AI assistants