Disease · MONDO_0010122
congenital thrombotic thrombocytopenic purpura
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. congenital thrombotic thrombocytopenic purpura. Diseases, record MONDO_0010122. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010122
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.
- Other names
Congenital thrombotic thrombocytopenic purpura due to ADAMTS-13 deficiency, Upshaw-Schulman syndrome, congenital ADAMTS-13 deficiency, congenital ADAMTS13 deficiency, congenital TTP, familial TTP, hereditary thrombotic thrombocytopenic purpura, thrombotic thrombocytopenic purpura, hereditary
- Open Targets
Drugs1
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 |
|---|---|---|---|---|---|---|---|
| APADAMTASE ALFA | CHEMBL4594607 | Enzyme | Phase 3 | ClinicalTrials.gov and TTD | 4 | 3 | 2 |
Source Drugs and targets
Targets1
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 |
|---|---|---|---|---|---|
| ADAMTS13 | A disintegrin and metalloproteinase with thrombospondin motifs 13 | 1 | A disintegrin and metalloproteinase with thrombospondin motifs 13 exogenous protein | Phase 3 | https://conductscience.org/library/genes/11093 |
Source Drugs and targets
Trials2
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 |
|---|---|---|---|---|---|---|---|---|
| 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 |
| 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 |
Comparisons in its trials4
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 |
|---|---|---|---|---|---|
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Neutralizing Antibodies to ADAMTS13 During Prophylactic Treatment | 0 of 21 | 1 of 27 | 48 | NCT03393975 | Trial comparisons |
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Anti-Chinese Hamster Ovary (Anti-CHO) Protein Antibodies During Prophylactic Treatment | 0 of 21 | 2 of 27 | 48 | NCT03393975 | Trial comparisons |
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Total Binding Antibodies to ADAMTS13 During Prophylactic Treatment | 0 of 21 | 2 of 27 | 48 | NCT03393975 | Trial comparisons |
| Prophylaxis Cohort: TAK-755 vs Prophylaxis Cohort: SoC: Number of Participants With Inhibitory Antibodies to ADAMTS13 | 1 of 6 | 0 of 4 | 10 | NCT03393975 | Trial comparisons |
Papers from its trials1
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 |
|---|---|---|
| 38692292 | DERIVED | 1 |
Source PubMed
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_0010122
GET https://conductscience.org/api/diseases/v1/MONDO_0010122/drugs