Disease · MONDO_0100244
Trials93
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. 5 more are not shown, since the checks do not confirm them.
1 to 20 of 93
Download this page as JSON| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02946463 | ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | 272 | 2019-02-12 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | ECULIZUMAB, RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04434092 | A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors | Phase 3 | 210 | 2025-11-10 | Paroxysmal Nocturnal Hemoglobinuria | CROVALIMAB, ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT00003838 | Specialized Blood Cell Transplants for Cancers of the Blood and Bone Marrow | Phase 2 | 202 | 2022-10-07 | Myeloproliferative Disorders, Acute Myelogenous Leukemia, Chronic Myelogenous Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia | CYCLOSPORINE, METHOTREXATE | Its record names the disease | ClinicalTrials.gov |
| NCT03056040 | ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Phase 3 | 202 | 2019-03-21 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | ECULIZUMAB, RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT00122317 | Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | 187 | 2017-06-05 | Paroxysmal Hemoglobinuria, Nocturnal | ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT03748823 | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Phase 3 | 139 | 2022-10-20 | Paroxysmal Nocturnal Hemoglobinuria | RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT00587054 | Trial of Allogeneic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Adult Patients With Lymphohematopoietic Disorders | Phase 2 | 129 | 2017-03-10 | Allogeneic Stem Cell Transplant, Leukemia, Non-Hodgkins, Lymphoblastic Lymphoma, Myelodysplastic Syndrome, Paroxysmal Nocturnal Hemoglobinuria (PNH) | FLUDARABINE, THIOTEPA | Its record names the disease | ClinicalTrials.gov |
| NCT04558918 | Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment | Phase 3 | 97 | 2023-11-28 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | IPTACOPAN | Its record names the disease | ClinicalTrials.gov |
| NCT04469465 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) | Phase 3 | 86 | 2023-07-24 | Paroxysmal Nocturnal Hemoglobinuria | DANICOPAN, ECULIZUMAB, RAVULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT03500549 | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Phase 3 | 80 | 2022-03-25 | Paroxysmal Nocturnal Hemoglobinuria | PEGCETACOPLAN | Its record names the disease | ClinicalTrials.gov |
| NCT02352493 | A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH | Phase 1/2 | 62 | 2020-03-30 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | CEMDISIRAN, ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04085601 | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH | Phase 3 | 53 | 2022-10-21 | Paroxysmal Nocturnal Hemoglobinuria | PEGCETACOPLAN | Its record names the disease | ClinicalTrials.gov |
| NCT05630001 | Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. | Phase 3 | 52 | 2025-12-15 | Paroxysmal Nocturnal Hemoglobinuria | IPTACOPAN | Its record names the disease | ClinicalTrials.gov |
| NCT04654468 | A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition | Phase 3 | 51 | 2023-06-05 | Paroxysmal Nocturnal Hemoglobinuria | CROVALIMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04058158 | A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria | Phase 3 | 50 | 2024-03-26 | Paroxysmal Nocturnal Hemoglobinuria | ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT03818607 | A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH | Phase 3 | 42 | 2023-05-23 | Paroxysmal Nocturnal Hemoglobinuria | ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04820530 | Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | Phase 3 | 40 | 2023-11-15 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | IPTACOPAN | Its record names the disease | ClinicalTrials.gov |
| NCT01192399 | Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Phase 2 | 29 | 2018-03-13 | Paroxysmal Nocturnal Hemoglobinuria | ECULIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04170023 | Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy | Phase 2 | 29 | 2024-08-09 | Paroxysmal Nocturnal Hemoglobinuria (PNH) | DANICOPAN, VEMIRCOPAN | Its record names the disease | ClinicalTrials.gov |
| NCT00058825 | Stem Cell Transplant for Hematologic Diseases | Phase 1/2 | 27 | 2015-10-22 | Hematologic Malignancies | ALEMTUZUMAB, CYCLOSPORINE, FLUDARABINE, TACROLIMUS, TACROLIMUS ANHYDROUS | Its record names the disease | ClinicalTrials.gov |
diseases-2026-09-18-0.1.0
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 list from software.
Twenty rows a page, as JSON. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0100244/trials