Disease · MONDO_0011382

sickle cell disease

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. sickle cell disease. Diseases, record MONDO_0011382. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0011382

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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: sickle cell disease; Open Targets
Open Targets ID: MONDO_0011382; Open Targets
Other names: Anemia, Sickle Cell, Drepanocythaemia, Drepanocythemia, HB-S DIS W/O CRISIS NEC, HB-SS DIS NEC W CRISIS, Haemoglobin S disease, Haemoglobin S disease without crisis, Haemoglobin S-S disease, Hb S disease, Hb SC disease, Hb SS disease, Hb-S/Hb-C disease, Hb-SS disease without crisis, Hemoglobin S disease, Hemoglobin S disease without crisis (disorder), Hemoglobin S-S disease, Hemoglobin SS disease without crisis (disorder), Hereditary hemoglobinopathy disorder homozygous for hemoglobin S, Hereditary hemoglobinopathy disorder homozygous for hemoglobin S (disorder), Other sickle-cell disease with crisis, Other sickle-cell disease without crisis, SICKLE CELL DISEASE NOS, Sickle cell anaemia, Sickle cell anaemia NOS, Sickle cell anaemia of unspecified type, Sickle cell anemia NOS, Sickle cell anemia NOS (disorder), Sickle cell anemia of unspecified type, Sickle cell anemia of unspecified type (disorder), Sickle cell syndrome, Sickle-cell disease, unspecified, hemoglobin SC disease, sickle cell anemia, sickle-cell/Hb-C disease without crisis, sickling disorder due to Haemoglobin S, sickling disorder due to Hemoglobin S; Open Targets
Disease Ontology: DOID:0081445; Disease Ontology
Disease Ontology: DOID:10923; Disease Ontology
MeSH: D000755; MeSH
NCI Thesaurus: C34383; NCI Thesaurus
Orphanet: ORPHA:232; Orphanet
Highest stage of its drugs: Approval, for 8 of its 163 drugs, from DailyMed, EMA Human Drugs, FDA and TTD; Open Targets

Drugs163

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
HYDROXYUREA	CHEMBL467	Small molecule	Approval	DailyMed and EMA Human Drugs	59	55	55	Drugs and targets
VOXELOTOR	CHEMBL4101807	Small molecule	Approval	DailyMed and FDA	23	20	20	Drugs and targets
CRIZANLIZUMAB	CHEMBL4297734	Antibody	Approval	DailyMed	10	8	8	Drugs and targets
GLUTAMINE	CHEMBL930	Small molecule	Approval	DailyMed	7	6	6	Drugs and targets
EXAGAMGLOGENE AUTOTEMCEL	CHEMBL5095474	Gene	Approval	EMA Human Drugs and FDA	8	5	4	Drugs and targets
DEFERIPRONE	CHEMBL70927	Small molecule	Approval	DailyMed	2	1	1	Drugs and targets
LOVOTIBEGLOGENE AUTOTEMCEL	CHEMBL4650269	Gene	Approval	FDA	2	0	0	Drugs and targets
CETIEDIL	CHEMBL419380	Small molecule	Approval	TTD	1	0	0	Drugs and targets
ALEMTUZUMAB	CHEMBL1201587	Antibody	Phase 3	ClinicalTrials.gov	29	29	29	Drugs and targets
FLUDARABINE	CHEMBL1568	Small molecule	Phase 3	ClinicalTrials.gov	27	27	27	Drugs and targets

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Targets270

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
GUCY1A1	Soluble guanylate cyclase	5	Soluble guanylate cyclase activator; Soluble guanylate cyclase positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2982	Drugs and targets
GUCY1A2	Soluble guanylate cyclase	5	Soluble guanylate cyclase activator; Soluble guanylate cyclase positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2977	Drugs and targets
GUCY1B1	Soluble guanylate cyclase	5	Soluble guanylate cyclase activator; Soluble guanylate cyclase positive allosteric modulator	Phase 2	https://conductscience.org/library/genes/2983	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	4	Granulocyte colony stimulating factor receptor agonist; Granulocyte colony stimulating factor receptor antagonist	Phase 3	https://conductscience.org/library/genes/1441	Drugs and targets
OPRM1	Mu-type opioid receptor	4	Mu opioid receptor agonist	Phase 3	https://conductscience.org/library/genes/4988	Drugs and targets
EDNRA	Endothelin receptor, ET-A/ET-B	3	Endothelin receptor, ET-A/ET-B antagonist	Phase 3	https://conductscience.org/library/genes/1909	Drugs and targets
EDNRB	Endothelin receptor, ET-A/ET-B	3	Endothelin receptor, ET-A/ET-B antagonist	Phase 3	https://conductscience.org/library/genes/1910	Drugs and targets
EPOR	Erythropoietin receptor	3	Erythropoietin receptor agonist	Phase 1/2	https://conductscience.org/library/genes/2057	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	3	FK506-binding protein 1A inhibitor	Phase 2	https://conductscience.org/library/genes/2280	Drugs and targets
GRIN1	Glutamate [NMDA] receptor	3	Glutamate [NMDA] receptor negative allosteric modulator	Phase 3	https://conductscience.org/library/genes/2902	Drugs and targets

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Trials302

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. 8 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT02516579	European Sickle Cell Disease Cohort - Hydroxyurea		1,906	2020-03-19	Sickle Cell Disease	HYDROXYUREA	Its record names the disease	ClinicalTrials.gov
NCT01966731	Realizing Effectiveness Across Continents With Hydroxyurea (REACH)	Phase 1/2	635	2020-11-09	Sickle Cell Disease	HYDROXYUREA	Its record names the disease	ClinicalTrials.gov
NCT03036813	Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)	Phase 3	449	2021-01-07	Sickle Cell Disease	VOXELOTOR	Its record names the disease	ClinicalTrials.gov
NCT01825512	Efficacy/Safety Study of Deferiprone Compared to Deferasirox in Paediatric Patients	Phase 3	435	2021-04-08	Chronic Iron Overload	DEFERASIROX, DEFERIPRONE	Its record names the disease	ClinicalTrials.gov
NCT01794000	A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)	Phase 3	341	2016-07-27	Sickle Cell Disease	PRASUGREL	Its record names the disease	ClinicalTrials.gov
NCT03431285	Ketamine for Acute Painful Crisis in Sickle Cell Disease Patients	Not applicable	278	2025-05-28	Sickle Cell Crisis	KETAMINE, MORPHINE	Its record names the disease	ClinicalTrials.gov
NCT04839354	Sickle Cell Disease Treatment With Arginine Therapy (STArT) Trial	Phase 3	271	2025-07-30	Sickle Cell Disease	ARGININE HYDROCHLORIDE	Its record names the disease	ClinicalTrials.gov
NCT04930445	Oxbryta® Product Registry An Observational Study Designed to Evaluate the Effect of Oxbryta in Individuals With SCD		265	2025-12-31	Sickle Cell Disease	VOXELOTOR	Its record names the disease	ClinicalTrials.gov
NCT03814746	Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients	Phase 3	253	2024-01-08	Sickle Cell Disease (SCD)	CRIZANLIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT04935879	A Study to Assess the Safety and Efficacy of Inclacumab in Participants With Sickle Cell Disease Experiencing Vaso-occlusive Crises	Phase 3	241	2025-12-02	Sickle Cell Disease, Vaso-occlusive Pain Episode in Sickle Cell Disease, Vaso-occlusive Crisis	INCLACUMAB	Its record names the disease	ClinicalTrials.gov

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Comparisons in its trials88

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
Deferiprone vs Deferasirox: deaths from any cause	0 of 193	0 of 197	390	NCT01825512	Trial comparisons
Deferiprone vs Deferasirox: serious adverse events	13 of 193	14 of 197	390	NCT01825512	Trial comparisons
Morphine Group vs Ketamine Group: The Rate of Hospital Admission	34 of 140	26 of 138	278	NCT03431285	Trial comparisons
Morphine Group vs Ketamine Group: Drug-related Adverse Effects	3 of 140	8 of 138	278	NCT03431285	Trial comparisons
Morphine Group vs Ketamine Group: deaths from any cause	0 of 140	0 of 138	278	NCT03431285	Trial comparisons
Morphine Group vs Ketamine Group: serious adverse events	0 of 140	0 of 138	278	NCT03431285	Trial comparisons
Deferiprone vs Deferasirox: Percentage of Successfully Chelated Patients	69 of 125	80 of 146	271	NCT01825512	Trial comparisons
L-Arginine Hydrochloride vs Placebo: deaths from any cause	0 of 129	0 of 142	271	NCT04839354	Trial comparisons
L-Arginine Hydrochloride vs Placebo: serious adverse events	22 of 129	25 of 142	271	NCT04839354	Trial comparisons
Inclacumab vs Placebo: Number of Participants With Treatment Emergent Adverse Events (TEAEs)	87 of 118	97 of 121	239	NCT04935879	Trial comparisons

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Papers from its trials181

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
41190764	RESULT	1	PubMed
39998301	RESULT	1	PubMed
39998298	RESULT	1	PubMed
39471440	RESULT	1	PubMed
38527291	RESULT	1	PubMed
37722599	RESULT	1	PubMed
37587492	RESULT	1	PubMed
34724240	RESULT	1	PubMed
32750127	RESULT	1	PubMed
32678452	RESULT	1	PubMed

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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_0011382

GET https://conductscience.org/api/diseases/v1/MONDO_0011382/drugs?page=2

API reference For AI assistants