Disease · MONDO_0019402

beta thalassemia

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. beta thalassemia. Diseases, record MONDO_0019402. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019402

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: beta thalassemia; Open Targets
Open Targets ID: MONDO_0019402; Open Targets
Disease Ontology: DOID:12241; Disease Ontology
MeSH: D017086; MeSH
NCI Thesaurus: C34375; NCI Thesaurus
Orphanet: ORPHA:848; Orphanet
Highest stage of its drugs: Approval, for 5 of its 42 drugs, from EMA Human Drugs; Open Targets

Drugs42

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
LUSPATERCEPT	CHEMBL3039545	Protein	Approval	EMA Human Drugs	13	11	11	Drugs and targets
DEFERASIROX	CHEMBL550348	Small molecule	Approval	EMA Human Drugs	13	10	10	Drugs and targets
DEFERIPRONE	CHEMBL70927	Small molecule	Approval	EMA Human Drugs	5	3	3	Drugs and targets
EXAGAMGLOGENE AUTOTEMCEL	CHEMBL5095474	Gene	Approval	EMA Human Drugs	4	1	1	Drugs and targets
BETIBEGLOGENE AUTOTEMCEL	CHEMBL4594311	Gene	Approval	EMA Human Drugs, at withdrawal	2	0	0	Drugs and targets
THALIDOMIDE	CHEMBL468	Small molecule	Phase 3	ClinicalTrials.gov	7	7	7	Drugs and targets
DEFEROXAMINE	CHEMBL556	Small molecule	Phase 3	ClinicalTrials.gov	4	4	4	Drugs and targets
MITAPIVAT	CHEMBL4299940	Small molecule	Phase 3	ClinicalTrials.gov	2	2	2	Drugs and targets
ACETYLCYSTEINE	CHEMBL600	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets
CARNITINE	CHEMBL172513	Small molecule	Phase 3	ClinicalTrials.gov	1	1	0	Drugs and targets

1 to 10 of 42

See all 42 drugs

Targets26

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
CSF3R	Granulocyte colony-stimulating factor receptor	3	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
ADA	Adenosine deaminase	1	Adenosine deaminase inhibitor	Phase 2	https://conductscience.org/library/genes/100	Drugs and targets
BCL11A	BCL11 transcription factor A	1	B-cell lymphoma/leukemia 11A gene editing negative modulator	Approval	https://conductscience.org/library/genes/53335	Drugs and targets
CD52	CAMPATH-1 antigen	1	CAMPATH-1 antigen inhibitor	Phase 2	https://conductscience.org/library/genes/1043	Drugs and targets
CD80	T-lymphocyte activation antigen CD80	1	T-lymphocyte activation antigen CD80 inhibitor	Phase 1/2	https://conductscience.org/library/genes/941	Drugs and targets
CD86	T-lymphocyte activation antigen CD86	1	T-lymphocyte activation antigen CD86 inhibitor	Phase 1/2	https://conductscience.org/library/genes/942	Drugs and targets
CRBN	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 3	https://conductscience.org/library/genes/51185	Drugs and targets
CUL4A	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 3	https://conductscience.org/library/genes/8451	Drugs and targets
CXCR4	C-X-C chemokine receptor type 4	1	C-X-C chemokine receptor type 4 partial agonist	Phase 1/2	https://conductscience.org/library/genes/7852	Drugs and targets
DDB1	CRL4(CRBN) E3 ubiquitin ligase	1	CRL4(CRBN) E3 ubiquitin ligase inhibitor	Phase 3	https://conductscience.org/library/genes/1642	Drugs and targets

1 to 10 of 26

See all 26 targets

Trials58

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

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00171210	An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload	Phase 3	506	2011-05-02	Transfusional Iron Overload in β-thalassemia	DEFERASIROX	Its record names the disease	ClinicalTrials.gov
NCT02604433	An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia	Phase 3	336	2020-01-27	Erythrocyte Transfusion, Beta-Thalassemia	LUSPATERCEPT	Its record names the disease	ClinicalTrials.gov
NCT04770779	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)	Phase 3	258	2025-05-25	Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia	MITAPIVAT	Its record names the disease	ClinicalTrials.gov
NCT00171301	Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)	Phase 4	233	2011-07-29	Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia	DEFERASIROX	Its record names the disease	ClinicalTrials.gov
NCT04770753	A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)	Phase 3	194	2025-01-24	Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia	MITAPIVAT	Its record names the disease	ClinicalTrials.gov
NCT00303329	Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients	Phase 2	184	2011-05-09	Anemia, Hemosiderosis	DEFERASIROX	Its record names the disease	ClinicalTrials.gov
NCT03342404	A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia	Phase 2	145	2021-11-18	Thalassemia	LUSPATERCEPT	Its record names the disease	ClinicalTrials.gov
NCT00061568	Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias	Phase 1/2	130	2024-02-29	Congenital Hemolytic Anemia, Sickle Cell Disease	ALEMTUZUMAB, SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT04411082	A Study of IMR-687 in Subjects With Beta Thalassemia	Phase 2	122	2022-06-30	β Thalassemia	TOVINONTRINE	Its record names the disease	ClinicalTrials.gov
NCT01749540	Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)	Phase 2	64	2024-07-18	B-Thalassemia	LUSPATERCEPT	Its record names the disease	ClinicalTrials.gov

1 to 10 of 58

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Comparisons in its trials19

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
ICL670 vs Crossover: serious adverse events	102 of 296	61 of 259	555	NCT00171210	Trial comparisons
Luspatercept + BSC vs Placebo + BSC: deaths from any cause	4 of 223	1 of 109	332	NCT02604433	Trial comparisons
Luspatercept + BSC vs Placebo + BSC: serious adverse events	53 of 223	8 of 109	332	NCT02604433	Trial comparisons
Mitapivat vs Placebo: deaths from any cause	0 of 171	0 of 87	258	NCT04770779	Trial comparisons
Mitapivat vs Placebo: serious adverse events	19 of 172	13 of 85	257	NCT04770779	Trial comparisons
Deferasirox (Between 2 <16 Years) vs Deferasirox (16 Years or Older): serious adverse events	5 of 162	12 of 69	231	NCT00171301	Trial comparisons
Mitapivat vs Placebo: deaths from any cause	0 of 130	0 of 64	194	NCT04770753	Trial comparisons
Mitapivat vs Placebo: serious adverse events	8 of 129	0 of 63	192	NCT04770753	Trial comparisons
Beta-thalassemia vs Rare Anemias: serious adverse events	36 of 85	50 of 99	184	NCT00303329	Trial comparisons
Luspatercept vs Placebo: Number of Participants With Anti-drug Antibody (ADA) Positive Test for Luspatercept	5 of 96	3 of 49	145	NCT03342404	Trial comparisons

1 to 10 of 19

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Papers from its trials42

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
35755297	RESULT	1	PubMed
32395210	RESULT	1	PubMed
30617198	RESULT	3	PubMed
30504333	RESULT	1	PubMed
29472357	RESULT	1	PubMed
24976388	RESULT	1	PubMed
20424435	RESULT	1	PubMed
42721980	DERIVED	1	PubMed
42274009	DERIVED	1	PubMed
42133908	DERIVED	3	PubMed

1 to 10 of 42

See all 42 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_0019402

GET https://conductscience.org/api/diseases/v1/MONDO_0019402/drugs?page=2

API reference For AI assistants