paroxysmal nocturnal hemoglobinuria
Information
- Disease name
- paroxysmal nocturnal hemoglobinuria
- Disease ID
- DOID:0060284
- Description
- "An acquired hemolytic anemia that is characterized by abdominal pain, hematuria, esophageal dysmotility and thrombosis, has_material_basis_in defect in the cell membrane glycosyl phosphatidylinositols that protect red blood cells from the innate complement immune system." [url:https\://en.wikipedia.org/wiki/Paroxysmal_nocturnal_hemoglobinuria]
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04434092 | Active, not recruiting | Phase 3 | A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors. | October 8, 2020 | June 30, 2028 |
| NCT05630001 | Active, not recruiting | Phase 3 | Single Arm, Open Label Trial With Iptacopan Treatment for 24 Weeks, in Patients on Stable Regimen of Anti-C5 Who Switch to Iptacopan. | April 24, 2023 | October 18, 2024 |
| NCT05389449 | Active, not recruiting | Phase 3 | A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH | October 28, 2022 | January 31, 2027 |
| NCT03329365 | Active, not recruiting | Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS | November 1, 2018 | March 29, 2024 | |
| NCT03333486 | Active, not recruiting | Phase 2 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | December 7, 2017 | August 28, 2024 |
| NCT05886244 | Active, not recruiting | Phase 3 | Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China | July 5, 2023 | June 13, 2025 |
| NCT06051357 | Active, not recruiting | Phase 2 | Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria | November 15, 2023 | October 2024 |
| NCT04654468 | Active, not recruiting | Phase 3 | A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibition | March 17, 2021 | February 18, 2028 |
| NCT05741346 | Active, not recruiting | Phase 2 | Long-term Safety of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria | January 18, 2023 | February 2026 |
| NCT05982938 | Available | Danicopan Early Access Program | |||
| NCT06028594 | Available | Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria | |||
| NCT01529827 | Completed | Phase 2 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | February 28, 2012 | August 29, 2019 |
| NCT02588833 | Completed | Phase 1 | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. Pegcetacoplan (APL-2) in PNH Subjects. | December 1, 2015 | August 26, 2019 |
| NCT02605993 | Completed | Phase 2 | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | January 4, 2016 | January 12, 2022 |
| NCT03181633 | Completed | Phase 2 | A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | June 22, 2017 | January 4, 2022 |
| NCT03406507 | Completed | Phase 3 | A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria | February 22, 2018 | August 25, 2022 |
| NCT03500549 | Completed | Phase 3 | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | June 14, 2018 | August 13, 2020 |
| NCT03748823 | Completed | Phase 3 | Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | February 19, 2019 | August 31, 2023 |
| NCT03818607 | Completed | Phase 3 | A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH | January 22, 2019 | July 12, 2022 |
| NCT03896152 | Completed | Phase 2 | Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | April 5, 2019 | February 9, 2022 |
| NCT04058158 | Completed | Phase 3 | A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria | August 7, 2019 | October 21, 2021 |
| NCT04060264 | Completed | Phase 3 | Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria | April 4, 2019 | December 30, 2020 |
| NCT04079257 | Completed | The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria | February 23, 2021 | July 1, 2022 | |
| NCT04085601 | Completed | Phase 3 | A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With PNH | August 27, 2019 | June 23, 2021 |
| NCT04128943 | Completed | Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria | November 1, 2019 | July 6, 2020 | |
| NCT04320602 | Completed | Phase 4 | Ravulizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With High-Dose Eculizumab | March 26, 2021 | December 20, 2022 |
| NCT04330534 | Completed | Phase 1/Phase 2 | First-in-Human Study of BCX9930 in Healthy Volunteers and Patients With PNH | March 3, 2020 | January 25, 2021 |
| NCT00012376 | Completed | Phase 1 | Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer | March 2001 | |
| NCT00143559 | Completed | Phase 2 | Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies | August 2005 | January 2009 |
| NCT00397813 | Completed | Phase 2 | Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders | January 2006 | March 2018 |
| NCT00731328 | Completed | Phase 2 | Donor Stem Cell Transplant After Busulfan, Fludarabine, Methylprednisolone, and Antithymocyte Globulin in Treating Patients With Bone Marrow Failure Syndrome | April 2008 | December 2013 |
| NCT00856388 | Completed | N/A | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders | January 14, 2009 | March 13, 2019 |
| NCT01192399 | Completed | Phase 2 | Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | November 2007 | June 2008 |
| NCT01194804 | Completed | Phase 2 | E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | April 2008 | March 2011 |
| NCT01272817 | Completed | N/A | Nonmyeloablative Allogeneic Transplant | October 2001 | October 2015 |
| NCT01294891 | Completed | Microvascular and Cardiac Dysfunction in Paroxysmal Nocturnal Hemoglobinuria and Sickle Cell Disease | February 2011 | October 2014 | |
| NCT01364363 | Completed | N/A | Unrelated Donor Stem Cell Transplantation | March 2005 | July 2015 |
| NCT01412047 | Completed | Paroxysmal Nocturnal Hemoglobinuria Human Anti-Human Antibodies Study | March 2012 | June 30, 2013 | |
| NCT04463056 | Completed | Phase 3 | Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH | November 29, 2017 | October 16, 2018 |
| NCT04469465 | Completed | Phase 3 | Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) | December 16, 2020 | January 16, 2024 |
| NCT04671810 | Completed | Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria® | June 13, 2019 | May 5, 2023 | |
| NCT04679103 | Completed | Phase 3 | A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation) | May 3, 2018 | April 20, 2020 |
| NCT04811716 | Completed | Phase 2 | Pozelimab and Cemdisiran Combination Treatment in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy | July 29, 2021 | October 18, 2023 |
| NCT04888507 | Completed | Phase 2 | Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy | July 8, 2021 | May 4, 2023 |
| NCT05490017 | Completed | Phase 1 | To Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Escalating Single and Multiple Doses of KP104 | December 30, 2020 | September 2, 2022 |
| NCT05642585 | Completed | Phase 1 | A Study of Single-dose MY008211A in Healthy Adults | May 27, 2022 | June 4, 2023 |
| NCT05828472 | Completed | Phase 1 | A Study of Multiple Ascending Doses MY008211A in Healthy Adults | January 10, 2023 | June 30, 2023 |
| NCT05828485 | Completed | Phase 1 | Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects | September 26, 2022 | October 23, 2022 |
| NCT05842486 | Completed | Hematological Response in Patients With Paroxysmal Nocturnal Hemoglobinuria Treated With Anti-C5 Antibody: an External Control Arm Study for Iptacopan Use in Anti-C5 naïve Patients | January 18, 2023 | March 1, 2023 | |
| NCT06326814 | Completed | Phase 1 | A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults | October 11, 2021 | May 5, 2023 |
| NCT06449001 | Not yet recruiting | Phase 3 | Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis | August 23, 2024 | January 28, 2028 |
| NCT06411626 | Not yet recruiting | Home Reported Outcomes in PNH | June 15, 2024 | May 15, 2025 | |
| NCT05646524 | Not yet recruiting | Phase 2 | Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy | January 2025 | September 2026 |
| NCT05646563 | Not yet recruiting | Phase 2 | Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris | March 2025 | January 2027 |
| NCT06298955 | Recruiting | Phase 2 | Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria | February 19, 2024 | April 2027 |
| NCT03047746 | Recruiting | N/A | Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF | February 1, 2017 | March 2026 |
| NCT05744921 | Recruiting | Phase 3 | A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works. | March 7, 2023 | February 5, 2029 |
| NCT05755867 | Recruiting | Global PNH Patient Registry | May 6, 2021 | May 6, 2031 | |
| NCT05776472 | Recruiting | A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | June 26, 2023 | July 3, 2027 | |
| NCT05133531 | Recruiting | Phase 3 | A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment | August 1, 2022 | March 29, 2027 |
| NCT05274633 | Recruiting | Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria | June 17, 2022 | February 28, 2025 | |
| NCT05476887 | Recruiting | Phase 2 | To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104 | November 25, 2022 | February 2025 |
| NCT06238544 | Recruiting | Phase 2 | Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria | February 6, 2024 | December 2027 |
| NCT04432584 | Recruiting | Phase 3 | A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors | September 30, 2020 | September 1, 2029 |
| NCT06050226 | Recruiting | Phase 2 | A Study of MY008211A in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | July 6, 2023 | July 30, 2024 |
| NCT01374360 | Recruiting | Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry | October 29, 2004 | December 31, 2024 | |
| NCT04747613 | Recruiting | Phase 3 | Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria | July 27, 2021 | January 10, 2028 |
| NCT06100900 | Recruiting | Phase 1 | Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria | October 24, 2023 | August 31, 2024 |
| NCT06154512 | Recruiting | A Real-world, Multi-center, Prospective, Observational Study for PNH in China | November 10, 2023 | November 30, 2025 | |
| NCT04965597 | Recruiting | Phase 2 | Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904) | April 19, 2022 | December 2026 |
| NCT06412497 | Recruiting | Phase 2 | MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure. | June 5, 2024 | May 1, 2036 |
| NCT02179359 | Recruiting | N/A | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | September 2, 2014 | August 2025 |
| NCT05889299 | Recruiting | Phase 1 | Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria | December 9, 2022 | June 30, 2024 |
| NCT05972967 | Recruiting | Phase 2 | Safety and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria Patients With a Sub-optimal Response to Ravulizumab | March 27, 2023 | July 31, 2025 |
| NCT04162470 | Terminated | Phase 3 | REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate Its Long Term Safety, Efficacy and Tolerability. | December 3, 2019 | April 7, 2022 |
| NCT04702568 | Terminated | Phase 2 | A Long Term Safety Study of BCX9930 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | December 18, 2020 | October 4, 2023 |
| NCT05131204 | Terminated | Phase 3 | Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | October 6, 2022 | July 12, 2023 |
| NCT03829449 | Terminated | Phase 3 | rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | March 13, 2017 | August 29, 2020 |
| NCT01642979 | Unknown status | Phase 2 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | July 2012 | July 2017 |
| NCT03866681 | Unknown status | Phase 4 | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH | April 1, 2019 | April 1, 2022 |
| NCT01760096 | Unknown status | Phase 2 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Subclinical Paroxysmal Nocturnal Hemoglobinuria and PNH in the Setting of Another Bone Marrow Failure Syndromes(PNH-2013) | January 2013 | July 2017 |
- Disase is a (Disease Ontology)
- DOID:582
- Cross Reference ID (Disease Ontology)
- GARD:7337
- Cross Reference ID (Disease Ontology)
- ICD10CM:D59.5
- Cross Reference ID (Disease Ontology)
- ICD10CM:D59.6
- Cross Reference ID (Disease Ontology)
- MESH:D006457
- Cross Reference ID (Disease Ontology)
- MIM:300818
- Cross Reference ID (Disease Ontology)
- MIM:615399
- Cross Reference ID (Disease Ontology)
- NCI:C61233
- Cross Reference ID (Disease Ontology)
- ORDO:447
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:1468004
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:154805009
- Cross Reference ID (Disease Ontology)
- SNOMEDCT_US_2023_03_01:191226006
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0019050
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0024790
- Cross Reference ID (Disease Ontology)
- UMLS_CUI:C0086774
- HPO Human Phenotype ID (Human Phenotype Ontology)
- HP:0004818
- OMIM Phenotype Series Number (OMIM)
- PS300818
- OrphaNumber from OrphaNet (Orphanet)
- 447
- MedGen concept unique identifier (MedGen Concept name)
- C0024790
- MedGen unique identifier (MedGen Concept name)
- 7471