Disease · MONDO_0019737
thrombotic microangiopathy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. thrombotic microangiopathy. Diseases, record MONDO_0019737. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019737
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.
- Highest stage of its drugs
- Phase 3, for 3 of its 14 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs14
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 |
|---|---|---|---|---|---|---|---|---|
| RAVULIZUMAB | CHEMBL3989986 | Antibody | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| NOMACOPAN | CHEMBL4297928 | Phase 3 | ClinicalTrials.gov and TTD | 2 | 1 | 1 | Drugs and targets | |
| ECULIZUMAB | CHEMBL1201828 | Antibody | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ACETYLCYSTEINE | CHEMBL600 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NARSOPLIMAB | CHEMBL4298195 | Antibody | Phase 2 | ClinicalTrials.gov and TTD | 4 | 2 | 2 | Drugs and targets |
| BORTEZOMIB | CHEMBL325041 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DARATUMUMAB | CHEMBL1743007 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DEXAMETHASONE | CHEMBL384467 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| EGAPTIVON PEGOL | CHEMBL2108339 | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 14
See all 14 drugsTargets47
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 | 3 | Complement C5 inhibitor | Phase 3 | https://conductscience.org/library/genes/727 | Drugs and targets |
| ADRM1 | 26S proteasome | 1 | 26S proteasome inhibitor | Phase 2 | https://conductscience.org/library/genes/11047 | Drugs and targets |
| C3 | Complement C3 | 1 | Complement C3 inhibitor | Phase 2 | https://conductscience.org/library/genes/718 | Drugs and targets |
| CD38 | ADP-ribosyl cyclase/cyclic ADP-ribose hydrolase 1 | 1 | Lymphocyte differentiation antigen CD38 inhibitor | Phase 2 | https://conductscience.org/library/genes/952 | Drugs and targets |
| CFB | Complement factor B | 1 | Complement factor B inhibitor | Unknown | https://conductscience.org/library/genes/629 | Drugs and targets |
| LTB4R | Leukotriene B4 receptor | 1 | Leukotriene B4 receptor inhibitor | Phase 3 | https://conductscience.org/library/genes/1241 | Drugs and targets |
| LTB4R2 | Leukotriene B4 receptor | 1 | Leukotriene B4 receptor inhibitor | Phase 3 | https://conductscience.org/library/genes/56413 | Drugs and targets |
| MASP2 | Mannan-binding lectin serine protease 2 | 1 | Mannan-binding lectin serine protease 2 inhibitor | Phase 2 | https://conductscience.org/library/genes/10747 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Phase 2 | https://conductscience.org/library/genes/2908 | Drugs and targets |
| PSMA1 | 26S proteasome | 1 | 26S proteasome inhibitor | Phase 2 | https://conductscience.org/library/genes/5682 | Drugs and targets |
1 to 10 of 47
See all 47 targetsTrials14
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 |
|---|---|---|---|---|---|---|---|---|
| NCT04557735 | Study of Ravulizumab in Pediatric Participants With HSCT-TMA | Phase 3 | 41 | 2026-01-12 | Thrombotic Microangiopathy | RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04743804 | Ravulizumab in Thrombotic Microangiopathy Associated With a Trigger | Phase 3 | 16 | 2023-12-12 | Thrombotic Microangiopathy | RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT05148299 | A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation | Phase 2 | 12 | 2025-11-28 | Transplant-Associated Thrombotic Microangiopathy | PEGCETACOPLAN | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT04784455 | Nomacopan (rVA576) in Transplant Associated Thrombotic Microangiopathy | Phase 3 | 10 | 2025-05-15 | Thrombotic Microangiopathies | NOMACOPAN | Its record names the disease | ClinicalTrials.gov |
| NCT04543591 | Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant | Phase 3 | 148 | Thrombotic Microangiopathy | RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT00726544 | Clinical Outcome Study of ARC1779 Injection in Patients With Thrombotic Microangiopathy | Phase 2 | 100 | Thrombotic Microangiopathy, Thrombotic Thrombocytopenic Purpura | EGAPTIVON PEGOL | Its record names the disease | ClinicalTrials.gov | |
| NCT07279610 | N-Acetylcysteine as Therapy for Transplantation- Associated Thrombotic Microangiopathy | Phase 2/3 | 44 | Thrombotic Microangiopathy | ACETYLCYSTEINE | Its record names the disease | ClinicalTrials.gov | |
| NCT04570397 | Ravulizumab and COVID-19 | Phase 3 | 32 | Covid19, Thrombotic Microangiopathies, Acute Kidney Injury | RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT07347990 | Safety and Efficacy of Iptacopan in Patients With High-Risk Transplantation-Associated Thrombotic Microangiopathy | Not applicable | 30 | Thrombotic Microangiopathy, Hematopoietic Stem Cell Transplantation (HSCT) | IPTACOPAN | Its record names the disease | ClinicalTrials.gov | |
| NCT05855083 | Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA | Phase 2 | 18 | Thrombotic Microangiopathies, Hematopoietic Stem Cell Transplantation | NARSOPLIMAB | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 14
See all 14 trialsComparisons in its trials8
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 |
|---|---|---|---|---|---|
| Treatment Period: Ravulizumab vs Follow-up Period: Ravulizumab: deaths from any cause | 6 of 41 | 4 of 29 | 70 | NCT04557735 | Trial comparisons |
| Treatment Period: Ravulizumab vs Follow-up Period: Ravulizumab: serious adverse events | 30 of 41 | 15 of 29 | 70 | NCT04557735 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants With Renal Response at Week 26 | 4 of 9 | 4 of 7 | 16 | NCT04743804 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants With Hematologic Response at Week 26 | 5 of 9 | 6 of 7 | 16 | NCT04743804 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants With Complete Thrombotic Microangiopathy (TMA) Response at Week 26 | 2 of 9 | 3 of 7 | 16 | NCT04743804 | Trial comparisons |
| Ravulizumab vs Placebo: deaths from any cause | 3 of 9 | 0 of 7 | 16 | NCT04743804 | Trial comparisons |
| Ravulizumab vs Placebo: serious adverse events | 6 of 9 | 3 of 7 | 16 | NCT04743804 | Trial comparisons |
| Ravulizumab vs Placebo: Number of Participants On Dialysis at Week 26 | 1 of 5 | 0 of 4 | 9 | NCT04743804 | Trial comparisons |
Papers from its trials7
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_0019737
GET https://conductscience.org/api/diseases/v1/MONDO_0019737/targets?page=2