Disease · MONDO_0016487
beta-thalassemia intermedia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. beta-thalassemia intermedia. Diseases, record MONDO_0016487. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016487
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.
Drugs11
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 | Phase 2/3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| DARBEPOETIN ALFA | CHEMBL1201566 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| EPOETIN ALFA | CHEMBL1201565 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| EPOETIN BETA | CHEMBL2109092 | Protein | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| SODIUM 2,2-DIMETHYLBUTYRATE | CHEMBL1650317 | Small molecule | Phase 2 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| CARNITINE | CHEMBL172513 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| LEVOCARNITINE | CHEMBL1149 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| MAGNESIUM CHLORIDE | CHEMBL3185229 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| SAPABLURSEN | CHEMBL4650478 | Oligonucleotide | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| THALIDOMIDE | CHEMBL468 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets10
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 |
|---|---|---|---|---|---|---|
| EPOR | Erythropoietin receptor | 3 | Erythropoietin receptor agonist | Phase 2/3 | https://conductscience.org/library/genes/2057 | Drugs and targets |
| CRBN | CRL4(CRBN) E3 ubiquitin ligase | 1 | CRL4(CRBN) E3 ubiquitin ligase inhibitor | Phase 2 | https://conductscience.org/library/genes/51185 | Drugs and targets |
| CUL4A | CRL4(CRBN) E3 ubiquitin ligase | 1 | CRL4(CRBN) E3 ubiquitin ligase inhibitor | Phase 2 | https://conductscience.org/library/genes/8451 | Drugs and targets |
| DDB1 | CRL4(CRBN) E3 ubiquitin ligase | 1 | CRL4(CRBN) E3 ubiquitin ligase inhibitor | Phase 2 | https://conductscience.org/library/genes/1642 | Drugs and targets |
| DDC | Aromatic-L-amino-acid decarboxylase | 1 | DOPA decarboxylase inhibitor | Phase 1/2 | https://conductscience.org/library/genes/1644 | Drugs and targets |
| RBX1 | CRL4(CRBN) E3 ubiquitin ligase | 1 | CRL4(CRBN) E3 ubiquitin ligase inhibitor | Phase 2 | https://conductscience.org/library/genes/9978 | Drugs and targets |
| RRM1 | Ribonucleoside-diphosphate reductase RR1 | 1 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2/3 | https://conductscience.org/library/genes/6240 | Drugs and targets |
| RRM2 | Ribonucleoside-diphosphate reductase RR1 | 1 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2/3 | https://conductscience.org/library/genes/6241 | Drugs and targets |
| RRM2B | Ribonucleoside-diphosphate reductase RR1 | 1 | Ribonucleoside-diphosphate reductase RR1 inhibitor | Phase 2/3 | https://conductscience.org/library/genes/50484 | Drugs and targets |
| TMPRSS6 | Transmembrane protease serine 6 mRNA | 1 | Transmembrane protease serine 6 mRNA antisense inhibitor | Phase 2 | https://conductscience.org/library/genes/164656 | Drugs and targets |
Trials8
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 |
|---|---|---|---|---|---|---|---|---|
| NCT04059406 | Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) | Phase 2 | 29 | 2025-02-18 | Beta Thalassemia Intermedia | SAPABLURSEN | Its record names the disease | ClinicalTrials.gov |
| NCT00809042 | Combination Therapy of Hydroxyurea With L-Carnitine and Magnesium Chloride in Thalassemia Intermedia | Phase 2 | 120 | β-Thalassemia Intermedia | HYDROXYUREA, MAGNESIUM CHLORIDE | Its record names the disease | ClinicalTrials.gov | |
| NCT07292259 | Combination of Thalidomide and Hydroxyuria in Transfusion Dependent Thalasemmia | Phase 2 | 100 | Transfusion Dependent Beta Thalassemia | HYDROXYUREA, THALIDOMIDE | Its record names the disease | ClinicalTrials.gov | |
| NCT01624038 | Therapeutic Effect and Safety of Combined Hydroxyurea With Recombinant Human Erythropoietin. | Phase 2/3 | 40 | Thalassemia Intermedia | HYDROXYUREA | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT04432623 | The BENeFiTS Trial in Beta Thalassemia Intermedia | Phase 1/2 | 36 | Beta Thalassemia Intermedia, Sickle Cell Disease | BENSERAZIDE | Its record names the disease | ClinicalTrials.gov | |
| NCT00790127 | Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia | Phase 1/2 | 21 | Beta Thalassemia | SODIUM 2,2-DIMETHYLBUTYRATE | Its record names the disease | ClinicalTrials.gov | |
| NCT01609595 | Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand | Phase 2 | 10 | Beta Thalassemia Intermedia | SODIUM 2,2-DIMETHYLBUTYRATE | Its record names the disease | ClinicalTrials.gov | |
| NCT01642758 | Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon | Phase 2 | 10 | Beta Thalassemia Intermedia | SODIUM 2,2-DIMETHYLBUTYRATE | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials0
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.
None in this release.
Papers from its trials8
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
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_0016487
GET https://conductscience.org/api/diseases/v1/MONDO_0016487/drugs