Disease · MONDO_0018896
thrombotic thrombocytopenic purpura
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. thrombotic thrombocytopenic purpura. Diseases, record MONDO_0018896. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018896
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
- Approval, for 3 of its 11 drugs, from ClinicalTrials.gov, EMA Human Drugs, FDA, PMDA and TTD
- Open Targets
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 |
|---|---|---|---|---|---|---|---|---|
| APADAMTASE ALFA | CHEMBL4594607 | Enzyme | Approval | ClinicalTrials.gov, EMA Human Drugs, FDA and PMDA | 10 | 4 | 4 | Drugs and targets |
| CAPLACIZUMAB | CHEMBL2109624 | Antibody | Approval | EMA Human Drugs, FDA, PMDA and TTD | 7 | 2 | 2 | Drugs and targets |
| CINAXADAMTASE ALFA | CHEMBL5095472 | Enzyme | Approval | PMDA | 2 | 0 | 0 | Drugs and targets |
| RITUXIMAB | CHEMBL1201576 | Antibody | Phase 3 | ClinicalTrials.gov | 6 | 6 | 6 | Drugs and targets |
| ACETYLCYSTEINE | CHEMBL600 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 1 | Drugs and targets |
| CYCLOSPORINE | CHEMBL160 | Protein | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| MAGNESIUM SULFATE | CHEMBL3989857 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| PREDNISONE | CHEMBL635 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ANFIBATIDE | CHEMBL4297694 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DANAZOL | CHEMBL1479 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets8
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 |
|---|---|---|---|---|---|---|
| VWF | von Willebrand factor | 2 | von Willebrand factor inhibitor | Approval | https://conductscience.org/library/genes/7450 | Drugs and targets |
| ADAMTS13 | A disintegrin and metalloproteinase with thrombospondin motifs 13 | 1 | A disintegrin and metalloproteinase with thrombospondin motifs 13 exogenous protein | Approval | https://conductscience.org/library/genes/11093 | Drugs and targets |
| AR | Androgen receptor | 1 | Androgen Receptor agonist | Phase 2 | https://conductscience.org/library/genes/367 | Drugs and targets |
| GP1BA | Platelet glycoprotein Ib alpha chain | 1 | Platelet glycoprotein Ib alpha chain antagonist | Phase 2 | https://conductscience.org/library/genes/2811 | Drugs and targets |
| MS4A1 | B-lymphocyte antigen CD20 | 1 | B-lymphocyte antigen CD20 binding agent | Phase 3 | https://conductscience.org/library/genes/931 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Phase 3 | https://conductscience.org/library/genes/2908 | Drugs and targets |
| PGR | Progesterone receptor | 1 | Progesterone receptor agonist | Phase 2 | https://conductscience.org/library/genes/5241 | Drugs and targets |
| PPIA | Peptidyl-prolyl cis-trans isomerase A | 1 | Cyclophilin A modulator | Phase 3 | https://conductscience.org/library/genes/5478 | Drugs and targets |
Trials18
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 |
|---|---|---|---|---|---|---|---|---|
| NCT03252925 | A Safety and Efficacy Study of NAC in Patients With TA-TMA | Phase 3 | 170 | 2022-02-14 | Thrombotic Microangiopathies, Hematologic Diseases | ACETYLCYSTEINE | Its record names the disease | ClinicalTrials.gov |
| NCT02878603 | Follow-up Study for Patients Who Completed Study ALX0681-C301 (Post-HERCULES) | Phase 3 | 104 | 2021-11-22 | Acquired Thrombotic Thrombocytopenic Purpura | CAPLACIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01151423 | Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) | Phase 2 | 75 | 2019-05-29 | Acquired Thrombotic Thrombocytopenic Purpura | CAPLACIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT03393975 | A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP) | Phase 3 | 52 | 2025-03-19 | Thrombotic Thrombocytopenic Purpura (TTP) | APADAMTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT03922308 | Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) | Phase 2 | 28 | 2022-12-01 | Acquired Thrombotic Thrombocytopenic Purpura (aTTP) | APADAMTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT00713193 | Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP) | Phase 3 | 26 | 2023-04-28 | Thrombotic Thrombocytopenic Purpura | CYCLOSPORINE, PREDNISONE | Its record names the disease | ClinicalTrials.gov |
| NCT01554514 | Low Dose Rituximab in Thrombotic Thrombocytopenic Purpura | Phase 2 | 19 | 2021-08-13 | Thrombotic Thrombocytopenic Purpura | RITUXIMAB | Its record names the disease | ClinicalTrials.gov |
| NCT00953771 | Safety Study of Danazol With Plasma Exchange and Steroids for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP) | Phase 2 | 9 | 2023-08-04 | Thrombotic Thrombocytopenic Purpura | DANAZOL | Its record names the disease | ClinicalTrials.gov |
| NCT00799773 | Evaluating the Effectiveness of Adding Rituximab to Standard Treatment for Thrombotic Thrombocytopenic Purpura (TTP) | Phase 3 | 3 | 2013-07-22 | Thrombotic Thrombocytopenic Purpura | RITUXIMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04683003 | A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura | Phase 3 | 77 | Thrombotic Thrombocytopenic Purpura (TTP) | APADAMTASE ALFA | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 18
See all 18 trialsComparisons in its trials22
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 |
|---|---|---|---|---|---|
| NAC Group vs Placebo Group: The Incidence of TA-TMA. | 5 of 66 | 15 of 77 | 143 | NCT03252925 | Trial comparisons |
| NAC Group vs Placebo: deaths from any cause | 16 of 66 | 27 of 74 | 140 | NCT03252925 | Trial comparisons |
| NAC Group vs Placebo: serious adverse events | 0 of 66 | 0 of 74 | 140 | NCT03252925 | Trial comparisons |
| Caplacizumab (Treated in Study C301) vs Standard of Care (SoC) (Treated in Study C301): deaths from any cause | 0 of 75 | 1 of 29 | 104 | NCT02878603 | Trial comparisons |
| Caplacizumab (Treated in Study C301) vs Standard of Care (SoC) (Treated in Study C301): serious adverse events | 28 of 75 | 16 of 29 | 104 | NCT02878603 | Trial comparisons |
| Caplacizumab (Treated in Study C301) vs Standard of Care (SoC) (Treated in Study C301): Number of Participants With aTTP Related Deaths Reported During the Study | 0 of 49 | 1 of 29 | 78 | NCT02878603 | Trial comparisons |
| Caplacizumab vs Placebo: Mortality | 0 of 36 | 2 of 39 | 75 | NCT01151423 | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Complete Remission Following Initial Daily Plasma Exchange (PE) | 29 of 36 | 18 of 39 | 75 | NCT01151423 | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With PE Related AEs | 20 of 36 | 20 of 39 | 75 | NCT01151423 | Trial comparisons |
| Caplacizumab vs Placebo: Number and Percentage of Subjects With Exacerbations of TTP | 3 of 36 | 11 of 39 | 75 | NCT01151423 | Trial comparisons |
1 to 10 of 22
See all 22 comparisons in its trialsPapers from its trials13
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_0018896
GET https://conductscience.org/api/diseases/v1/MONDO_0018896/comparisons?page=2