Disease · MONDO_0016244

atypical hemolytic-uremic syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. atypical hemolytic-uremic syndrome. Diseases, record MONDO_0016244. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016244

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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: atypical hemolytic-uremic syndrome; Open Targets
Open Targets ID: MONDO_0016244; Open Targets
Other names: D-HUS, aHUS, atypical HUS, hemolytic-uremic syndrome without diarrhea, hemolytic-uremic syndrome without diarrhoea, non-diarrhea-associated hemolytic uremic syndrome; Open Targets
Disease Ontology: DOID:0080301; Disease Ontology
MeSH: D065766; MeSH
NCI Thesaurus: C123223; NCI Thesaurus
Orphanet: ORPHA:2134; Orphanet
Highest stage of its drugs: Approval, for 1 of its 8 drugs, from DailyMed; Open Targets

Drugs8

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
ECULIZUMAB	CHEMBL1201828	Antibody	Approval	DailyMed	12	11	11	Drugs and targets
IPTACOPAN	CHEMBL4594448	Small molecule	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
RAVULIZUMAB	CHEMBL3989986	Antibody	Phase 3	ClinicalTrials.gov	3	3	3	Drugs and targets
NARSOPLIMAB	CHEMBL4298195	Antibody	Phase 3	ClinicalTrials.gov and TTD	3	2	2	Drugs and targets
CROVALIMAB	CHEMBL4594316	Antibody	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
CEMDISIRAN	CHEMBL4297722	Oligonucleotide	Phase 2	ClinicalTrials.gov	3	2	2	Drugs and targets
AVACOPAN	CHEMBL3989871	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
RUXOPRUBART	CHEMBL6068459	Antibody	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

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
C5	Complement C5	4	Complement C5 inhibitor; Complement C5 mRNA RNAi inhibitor	Approval	https://conductscience.org/library/genes/727	Drugs and targets
C5AR1	C5a anaphylatoxin chemotactic receptor 1	1	C5a anaphylatoxin chemotactic receptor antagonist	Phase 2	https://conductscience.org/library/genes/728	Drugs and targets
CFB	Complement factor B	1	Complement factor B inhibitor	Phase 3	https://conductscience.org/library/genes/629	Drugs and targets
MASP2	Mannan-binding lectin serine protease 2	1	Mannan-binding lectin serine protease 2 inhibitor	Phase 3	https://conductscience.org/library/genes/10747	Drugs and targets

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
NCT02222545	Safety and Efficacy Study of OMS721 in Patients With Thrombotic Microangiopathies	Phase 2	58	2024-08-28	Thrombotic Microangiopathies	NARSOPLIMAB	Its record names the disease	ClinicalTrials.gov
NCT02949128	Study of ALXN1210 in Complement Inhibitor Treatment-Naïve Adult and Adolescent Participants With Atypical Hemolytic Uremic Syndrome (aHUS)	Phase 3	58	2020-02-10	Atypical Hemolytic Uremic Syndrome (aHUS)	RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT01194973	An Open-label, Multi-center Clinical Trial of Eculizumab in Adult Patients With Atypical Hemolytic-uremic Syndrome	Phase 2	44	2015-04-24	Atypical Hemolytic-Uremic Syndrome	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03131219	Study of Ravulizumab in Children and Adolescents With Atypical Hemolytic Uremic Syndrome (aHUS)	Phase 3	34	2020-09-16	Atypical Hemolytic Uremic Syndrome (aHUS)	RAVULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT05876351	Eculizumab in Pediatric and Adult Participants With Atypical Hemolytic Uremic Syndrome (aHUS) in China	Phase 3	25	2025-12-08	Atypical Hemolytic Uremic	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT01193348	An Open-Label, Multi-Center Clinical Trial of Eculizumab in Pediatric Patients With Atypical Hemolytic-Uremic Syndrome	Phase 2	22	2015-04-29	Atypical Hemolytic-Uremic Syndrome	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00844545	Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-Resistant aHUS	Phase 2	16	2014-12-01	Atypical Hemolytic Uremic Syndrome	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00838513	Open Label Controlled Trial of Eculizumab in Adult Patients With Plasma Therapy-sensitive Atypical Hemolytic Uremic Syndrome (aHUS)	Phase 2	15	2015-01-26	Atypical Hemolytic Uremic Syndrome	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT03205995	Safety and Efficacy Study of OMS721 in Patients With Atypical Hemolytic Uremic Syndrome	Phase 3	6	2025-12-10	Thrombotic Microangiopathies, Atypical Hemolytic Uremic Syndrome	NARSOPLIMAB	Its record names the disease	ClinicalTrials.gov
NCT00844428	Open Label Controlled Trial of Eculizumab in Adolescent Patients With Plasma Therapy-Sensitive aHUS	Phase 2	5	2015-01-26	Atypical Hemolytic Uremic Syndrome	ECULIZUMAB	Its record names the disease	ClinicalTrials.gov

1 to 10 of 25

See all 25 trials

Comparisons in its trials2

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
Complement Inhibitor Treatment Naïve vs Eculizumab Experienced: deaths from any cause	0 of 24	0 of 10	34	NCT03131219	Trial comparisons
Complement Inhibitor Treatment Naïve vs Eculizumab Experienced: serious adverse events	16 of 24	1 of 10	34	NCT03131219	Trial comparisons

Papers 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
34169200	RESULT	1	PubMed
33270832	RESULT	1	PubMed
32299680	RESULT	1	PubMed
25654478	RESULT	1	PubMed
39468592	DERIVED	1	PubMed
39105067	DERIVED	1	PubMed
36329366	DERIVED	1	PubMed
35439028	DERIVED	1	PubMed
34852172	DERIVED	1	PubMed
33783815	DERIVED	10	PubMed

1 to 10 of 15

See all 15 papers from its trials

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_0016244

GET https://conductscience.org/api/diseases/v1/MONDO_0016244/trials?page=2

API reference For AI assistants