62. Paroxysmal nocturnal hemoglobinuria
[
80 clinical trials,
68 drugs（DrugBank:
26 drugs),
14 target genes / 83 target pathways ]
Searched query = "Paroxysmal nocturnal hemoglobinuria"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04079257 | October 2019 | 16 September 2019 | The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria | The Pharmacokinetics and Pharmacodynamics of Eculizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;Eculizumab;PK-PD | Other: Blood collection for measurement of eculizumab peak concentrations | Radboud University | Not recruiting | 18 Years | N/A | All | 60 | N/A | ||
| 2 | NCT04085601 | August 14, 2019 | 30 September 2019 | A Phase III Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH | A Phase 3, Randomized, Multicenter, Open-Label, Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | Recruiting | 18 Years | N/A | All | 54 | Phase 3 | Malaysia;Poland | |
| 3 | NCT04058158 | August 7, 2019 | 14 October 2019 | A Study to Compare SB12 (Proposed Eculizumab Biosimilar) to Soliris in Subjects With Paroxysmal Nocturnal Haemoglobinuria | A Phase III Randomised, Double-blind, Multicentre Study to Compare the Efficacy, Safety, Pharmacokinetics, and Immunogenicity Between SB12 (Proposed Eculizumab Biosimilar) and Soliris® in Subjects With Paroxysmal Nocturnal Haemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Drug: SB12 (proposed eculizumab biosimilar);Drug: Soliris (eculizumab) | Samsung Bioepis Co., Ltd. | Recruiting | 18 Years | N/A | All | 50 | Phase 3 | India;Korea, Republic of;Taiwan;Ukraine | |
| 4 | NCT04060264 | June 17, 2019 | 2 September 2019 | Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria | Randomized, Open-Label, International, Multi-center, Comparative Study of Efficacy and Safety of BCD-148 (JSC BIOCAD, Russia) and Soliris® in Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria | Biological: BCD-148;Biological: Soliris | Biocad | Recruiting | 18 Years | 65 Years | All | 28 | Phase 3 | Russian Federation | |
| 5 | NCT03946748 | May 16, 2019 | 24 June 2019 | Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label, Single Arm Study to Evaluate the Efficacy and Safety of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor-Naive or Have Not Recently Received Complement Inhibitor Therapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: REGN3918 | Regeneron Pharmaceuticals | Recruiting | 18 Years | N/A | All | 42 | Phase 2 | Korea, Republic of | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03818607 | April 24, 2019 | 4 November 2019 | A Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Participants With PNH | A Randomized, Double-Blind, Active-Controlled Phase 3 Study Evaluating the Efficacy and Safety of ABP 959 Compared With Eculizumab in Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: ABP 959;Drug: Eculizumab | Amgen | Recruiting | 18 Years | N/A | All | 40 | Phase 3 | United States;France;Germany;Italy;Netherlands;Spain;United Kingdom | |
| 7 | NCT03896152 | April 5, 2019 | 4 November 2019 | Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria | A Multi-center, Randomized, Open-label, Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria and Active Hemolysis | Paroxysmal Nocturnal Hemoglobinuria | Drug: LNP023 | Novartis Pharmaceuticals | Recruiting | 18 Years | N/A | All | 10 | Phase 2 | Korea, Republic of;Malaysia;Russian Federation;Singapore;Taiwan | |
| 8 | NCT03866681 | April 1, 2019 | 18 March 2019 | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory PNH | Sirolimus Combined With Low-dose Warfarin for the Treatment of Refractory Classic Paroxysmal Nocturnal Hemoglobinuria ,a Prospective Study | Paroxysmal Nocturnal Hemoglobinuria | Drug: sirolimus | Peking Union Medical College Hospital | Not recruiting | 18 Years | 70 Years | All | 40 | Phase 4 | China | |
| 9 | EUCTR2017-002370-39-ES | 08/02/2019 | 28 February 2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Ravulizumab Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 70- Product Name: Ravulizumab Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Ravulizumab Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Alexion Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 105 | Phase 3 | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | |||
| 10 | EUCTR2017-001418-27-NL | 22/01/2019 | 28 February 2019 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 Pharmaceutical Form: Solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Current Sponsor code: ABP 959 Other descriptive name: ABP 959 - biosimilar to eculizumab Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 900- Trade Name: Soliris Product Name: Soliris Pharmaceutical Form: Solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 900- | Amgen Inc. | Authorised | Female: yes Male: yes | 40 | Phase 3 | Spain;Norway;Germany;Netherlands;Italy;United Kingdom;Korea, Republic of | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | EUCTR2017-002370-39-FR | 28/12/2018 | 30 April 2019 | This study compares the pharmacokinetics (PK) of ravulizumab subcutaneous (SC) administered via an on-body delivery system (OBDS) to ravulizumab intravenous (IV) in patients with paroxysmal nocturnal hemoglobinuria (PNH) currently treated With Eculizumab | A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: Ravulizumab Product Code: ALXN1210 Pharmaceutical Form: Solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Ravulizumab Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 70- Product Name: Ravulizumab Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Ravulizumab Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Alexion Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 105 | Phase 3 | United States;Finland;Spain;Turkey;Austria;Russian Federation;Italy;United Kingdom;France;Czech Republic;Canada;Belgium;Brazil;Australia;Netherlands;Germany;Korea, Republic of;Sweden | |||
| 12 | EUCTR2017-004268-36-ES | 19/09/2018 | 1 October 2018 | A Research Study to Gather Scientific Information About the Efficacy, Safety, and Tolerability of the Investigational Drug APL-2 In Treating Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH), a Disease Associated With Anemia, In A Randomly Assigned Comparison With the Current Standard of Care Treatment Approved for PNH At Multiple Research Centres. | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) - PEGASUS | PNH is an acquired, rare, clonal, non-malignant hematologic disease characterized by complement-mediated RBC hemolysis with or without hemoglobinuria, an increased susceptibility to thrombotic episodes, and/or some degree of bone marrow dysfunction. PNH is caused by complement-mediated lysis of erythrocyte clones lacking functional CD55 and CD59 on their surface. These RBCs are particularly susceptible to the MAC and have been shown to lyse readily in the presence of complement activation. MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: APL-2 Product Code: APL-2 Pharmaceutical Form: Solution for infusion INN or Proposed INN: n/a CAS Number: n/a Current Sponsor code: APL-2 Other descriptive name: n/a Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 54- Trade Name: Soliris Product Name: Soliris Pharmaceutical Form: Infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Apellis Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 70 | Phase 3 | United States;Canada;Spain;Australia;Russian Federation;Japan;Korea, Republic of | |||
| 13 | NCT03531255 | August 27, 2018 | 17 September 2018 | Multi-center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase III, Open Label, Non-Randomized, Multi-Center Extension Study to Evaluate the Long Term Safety and Efficacy of APL-2 in the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) | PNH | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | Recruiting | 18 Years | N/A | All | 109 | Phase 3 | United States | |
| 14 | NCT03593200 | August 16, 2018 | 4 February 2019 | A Phase IIa Study to Assess the Safety, Efficacy, and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With PNH | Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH). | PNH | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 4 | Phase 2 | Bulgaria;Serbia;Greece;Poland;Romania | |
| 15 | EUCTR2017-005140-16-BG | 18/07/2018 | 30 April 2019 | Phase IIa study of APL-2 in patients with PNH | A Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: APL-2 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: Not yet assigned CAS Number: 2019171-69-6 Current Sponsor code: APL-2 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | Apellis Pharmaceuticals | Authorised | Female: yes Male: yes | 20 | Phase 2 | Serbia;Greece;Poland;Romania;Bulgaria | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03588026 | July 9, 2018 | 4 November 2019 | Treating Paroxysmal Nocturnal Haemoglobinuria Patients With rVA576 | Investigational Product ; Coversin. Phase III Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in Patients With Paroxysmal Nocturnal Haemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: rVA576;Other: Standard of care (SOC) | AKARI Therapeutics | Recruiting | 18 Years | N/A | All | 30 | Phase 3 | Kazakhstan;Lithuania;Sri Lanka | |
| 17 | NCT03500549 | June 14, 2018 | 27 May 2019 | Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH | A Phase III, Randomized, Multi-Center, Open-Label, Active-Comparator Controlled Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2;Drug: Soliris | Apellis Pharmaceuticals, Inc. | Recruiting | 18 Years | N/A | All | 70 | Phase 3 | United States;Australia;Canada;Germany;Japan | |
| 18 | NCT03427060 | May 14, 2018 | 26 August 2019 | Coversin in PNH in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms | CONSENT II: Coversin in Paroxysmal Nocturnal Hemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Coversin | AKARI Therapeutics | Recruiting | 18 Years | N/A | All | 6 | Phase 2 | United States;Argentina | |
| 19 | NCT03472885 | May 8, 2018 | 2 September 2019 | A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab | A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ACH-0144471;Drug: Eculizumab | Achillion Pharmaceuticals | Not recruiting | 18 Years | 65 Years | All | 12 | Phase 2 | United States | |
| 20 | EUCTR2017-002820-26-NL | 21/03/2018 | 28 February 2019 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ALXN1210 Other descriptive name: ALXN1210 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Alexion Pharmaceuticals Incorporated | Authorised | Female: yes Male: yes | 12 | Phase 3 | France;United States;Netherlands;United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | EUCTR2017-003847-39-LT | 12/03/2018 | 26 March 2018 | A study to assess the safety and efficacy of rVA576 in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) | CAPSTONE: Phase III Confirmatory Assessment Protocol: rVA576 Safety and Efficacy in Three-Part, Two-Arm, Randomised Open Label Evaluation in patients with Paroxysmal Nocturnal Haemoglobinuria (PNH) - CAPSTONE | Paroxysmal Nocturnal Haemoglobinuria (PNH) MedDRA version: 20.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: rVA576 Pharmaceutical Form: Powder for solution for injection INN or Proposed INN: NA Current Sponsor code: rVA576 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 18- | Akari Therapeutics Plc. | Authorised | Female: yes Male: yes | 30 | Phase 3 | Ecuador;Romania;Turkey;Lithuania;Peru;Kazakhstan;Sri Lanka | |||
| 22 | NCT03406507 | February 22, 2018 | 28 October 2019 | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Biological: ALXN1210 | Alexion Pharmaceuticals | Recruiting | N/A | 18 Years | All | 12 | Phase 3 | United States;France;Netherlands;Norway;Russian Federation;United Kingdom | |
| 23 | EUCTR2017-000888-33-DE | 05/02/2018 | 10 October 2018 | A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) | An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: HLGT Classification code 10018911 Term: Haemolyses and related conditions System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LNP023 Pharmaceutical Form: Capsule, hard INN or Proposed INN: not yet established Current Sponsor code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- | Novartis Pharma AG | Authorised | Female: yes Male: yes | 10 | Phase 2 | France;Germany;Italy | |||
| 24 | EUCTR2017-000888-33-FR | 15/01/2018 | 28 February 2019 | A Study evaluating the safety and efficacy of LNP023 in patients with a blood disorder called paroxysmal nocturnal hemoglobinuria (PNH) | An open label, single arm, multiple dose study to assess efficacy, safety, pharmacokinetics and pharmacodynamics of LNP023 when administered in addition to Standard of Care (SoC) in patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: HLGT Classification code 10018911 Term: Haemolyses and related conditions System Organ Class: 10005329 - Blood and lymphatic system disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LNP023 Pharmaceutical Form: Capsule, hard INN or Proposed INN: not yet established Current Sponsor code: LNP023 Other descriptive name: LNP023 HYDROCHLORIDE SALT Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- | Novartis Pharma AG | Authorised | Female: yes Male: yes | 10 | Phase 2 | France;Germany;Italy | |||
| 25 | NCT03333486 | December 7, 2017 | 25 June 2018 | Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer | A Phase II Trial of Haploidentical Allogeneic Stem Cell Transplantation Utilizing Mobilized Peripheral Blood Stem Cells | Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Acute Leukemia in Remission;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Acute Myeloid Leukemia With FLT3/ITD Mutation;Acute Myeloid Leukemia With Gene Mutations;Aplastic Anemia;B-Cell Non-Hodgkin Lymphoma;CD40 Ligand Deficiency;Chronic Granulomatous Disease;Chronic Leukemia in Remission;Chronic Lymphocytic Leukemia;Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive;Congenital Amegakaryocytic Thrombocytopenia;Congenital Neutropenia;Congenital Pure Red Cell Aplasia;Glanzmann Thrombasthenia;Immunodeficiency Syndrome;Myelodysplastic Syndrome;Myelofibrosis;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Plasma Cell Myeloma;Polycythemia Vera;Recurrent Non-Hodgkin Lymphoma;Refractory Non-Hodgkin Lymphoma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndrome;Severe Aplastic Anemia;Shwachman-Diamond Syndrome;Sickle Cell Disease;T-Cell Non-Hodgkin Lymphoma;Thalassemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome | Drug: Cyclophosphamide;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Recruiting | 1 Year | 75 Years | All | 58 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | EUCTR2017-001418-27-ES | 28/08/2017 | 16 October 2017 | This trial is designed to determine what effects the investigational medicine, ABP 959, has on the human body, and what effects the body has on the investigational medicine after you have been given it, and if this is comparable to what is seen for the licensed medicine, eculizumab, in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). This study will assess if the investigational medicine is safe and effective in treating PNH compared to the licensed medicine. | A RANDOMIZED, DOUBLE-BLIND, ACTIVE-CONTROLLED PHASE 3 STUDY EVALUATING THE EFFICACY AND SAFETY OF ABP 959 COMPARED WITH ECULIZUMAB IN ADULT SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - Not applicable | Paroxysmal Nocturnal Hemoglobinuria (PNH);Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: ABP 959 Product Code: ABP 959 Pharmaceutical Form: Solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Current Sponsor code: ABP 959 Other descriptive name: ABP 959 - biosimilar to eculizumab Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 900- Trade Name: Soliris Product Name: Soliris Pharmaceutical Form: Solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 900- | Amgen Inc. | Authorised | Female: yes Male: yes | 40 | Phase 3 | France;Spain;Russian Federation;Netherlands;Germany;Japan;Italy;United Kingdom;Korea, Republic of | |||
| 27 | NCT03225287 | July 17, 2017 | 15 July 2019 | Extension Study of RA101495 for Patients With PNH Who Have Completed a Zilucoplan (RA101495) Clinical Study | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Zilucoplan (RA101495) | Ra Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 28 | Phase 2 | United States;Australia;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom | |
| 28 | NCT03181633 | June 22, 2017 | 2 September 2019 | A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100 | Paroxysmal Nocturnal Hemoglobinuria | Drug: ACH-0144471 | Achillion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 8 | Phase 2 | New Zealand | |
| 29 | EUCTR2016-003522-16-FI | 20/06/2017 | 7 May 2018 | Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A PHASE 2 MULTICENTER,OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RA101495 IN SUBJECTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000012950 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 Pharmaceutical Form: Solution for injection INN or Proposed INN: RA101495 sodium CAS Number: 1841136-73-9 Current Sponsor code: RA101495 Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | Ra Pharmaceuticals, Inc. | Not Recruiting | Female: yes Male: yes | 20 | Phase 2 | Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | |||
| 30 | NCT03056040 | June 5, 2017 | 1 April 2019 | ALXN1210 Versus Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Biological: Ravulizumab;Biological: Eculizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 195 | Phase 3 | United States;Australia;Canada;France;Germany;Italy;Japan;Korea, Republic of;Netherlands;Spain;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | EUCTR2016-002026-36-GB | 30/05/2017 | 23 July 2018 | ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) currently Treated With Eculizumab | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Trade Name: Soliris Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Alexion Pharmaceuticals Incorporated | Authorised | Female: yes Male: yes | 192 | Phase 3 | United States;Spain;Netherlands;Germany;United Kingdom | |||
| 32 | NCT03030183 | April 17, 2017 | 15 July 2019 | Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab | A Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria Who Have an Inadequate Response to Eculizumab | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: Zilucoplan (RA101495) | Ra Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 3 | Phase 2 | United States | |
| 33 | EUCTR2016-003523-34-FI | 06/04/2017 | 9 October 2017 | Extension Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000012950 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: RA101495 Product Code: RA101495 Pharmaceutical Form: Solution for injection INN or Proposed INN: RA101495 sodium CAS Number: 1841136-73-9 Current Sponsor code: RA101495 Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 40- | Ra Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 28 | Phase 2 | United States;Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom | |||
| 34 | NCT03053102 | March 31, 2017 | 2 September 2019 | A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ACH-0144471 | Achillion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 10 | Phase 2 | Italy;Korea, Republic of;New Zealand;United Kingdom | |
| 35 | EUCTR2016-004129-18-GB | 17/03/2017 | 5 June 2017 | An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study | CONSERVE: Coversin Long Term Safety and Efficacy Surveillance Study | Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 20.0 Level: PT Classification code 10018932 Term: Haemolytic uraemic syndrome System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 20.0 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 Pharmaceutical Form: Powder for solution for injection INN or Proposed INN: COVERSIN Current Sponsor code: rVA576 Other descriptive name: COVERSIN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 18- | Akari Therapeutics Plc | Authorised | Female: yes Male: yes | 50 | Phase 3 | Argentina;Poland;Netherlands;United Kingdom | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | NCT03829449 | March 13, 2017 | 11 March 2019 | rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study | Paroxysmal Nocturnal Hemoglobinuria;Atypical Hemolytic Uremic Syndrome | Drug: rVA576 (Coversin) | AKARI Therapeutics | Recruiting | 18 Years | N/A | All | 50 | Phase 3 | Poland | |
| 37 | NCT03078582 | March 8, 2017 | 17 September 2018 | Phase 2 Safety and Efficacy Study of RA101495 to Treat PNH Patients | Phase 2 Multicenter, Open-Label, Uncontrolled Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of RA101495 in Subjects With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: RA101495 | Ra Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 26 | Phase 2 | Australia;Canada;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom | |
| 38 | EUCTR2016-002025-11-DE | 20/12/2016 | 3 April 2017 | Study of ALXN1210 compared to eculizumab in PNH patients who have never been treated with a complement inhibitor. | A Phase 3, Randomized, Open-Label, Active-Controlled Study of ALXN1210 Versus Eculizumab in Complement Inhibitor-Naïve Adult Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 19.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Current Sponsor code: ALXN1210 Other descriptive name: Fc- and CDR-modified humanised monoclonal antibody against C5 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Trade Name: Soliris Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | Alexion Pharmaceuticals Incorporated | Authorised | Female: yes Male: yes | 214 | Phase 3 | Portugal;United States;Estonia;Poland;Spain;Austria;Denmark;Germany;United Kingdom;Korea, Republic of | |||
| 39 | EUCTR2016-002943-40-ES | 21/11/2016 | 9 January 2017 | A Clinical Study with Investigational Drug, ALN-CC5, in patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | A Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALN-CC5 Pharmaceutical Form: Solution for injection INN or Proposed INN: ALN-62643 Current Sponsor code: ALN-62643 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 200- | Alnylam Pharmaceuticals Inc | Not Recruiting | Female: yes Male: yes | 15 | Phase 2 | Taiwan;Finland;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Denmark;Bulgaria;Norway;Netherlands;Germany;Korea, Republic of;Sweden | |||
| 40 | NCT03157635 | November 14, 2016 | 22 October 2019 | Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Participants With Paroxysmal Nocturnal Hemoglobinuria | An Adaptive Phase I/II Study to Assess Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Hemoglobinuria, Nocturnal | Drug: RO7112689;Drug: Placebo | Hoffmann-La Roche | Recruiting | 18 Years | 75 Years | All | 49 | Phase 1/Phase 2 | France;Germany;Hungary;Italy;Japan;Korea, Republic of;Netherlands | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | EUCTR2016-002128-10-NL | 14/09/2016 | 2 October 2017 | A Study to Assess Safety, Effectiveness, Pharmacokinetics, and Pharmacodynamics of RO7112689 in Healthy Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | An adaptive Phase I/II study to assess safety, efficacy, pharmacokinetics and pharmacodynamics of RO7112689 in healthy volunteers and patients with paroxysmal nocturnal hemoglobinuria (PNH) | Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 19.0 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: RO7112689/F01 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: Not available CAS Number: 1917321-26-6 Current Sponsor code: RO7112689 Other descriptive name: C5 inh MAb, SKY59, RO/CH7092230 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 170- Pharmaceutical form of the placebo: Solution for injection/infusion Route of administration of the placebo: Intravenous use Pharmaceutical form of the placebo: Solution for injection/infusion Route of administration of the placebo: Subcutaneous use | F. Hoffmann-La Roche Ltd | Not Recruiting | Female: no Male: yes | 74 | Phase 1;Phase 2 | France;Hungary;Czech Republic;Brazil;Germany;Netherlands;Japan;Italy;United Kingdom | |||
| 42 | EUCTR2016-002067-33-GB | 18/07/2016 | 28 February 2019 | An Open Clincal Trial of Coversin in Patients with PNH | COBALT: Coversin Global Study: An Open-Label, Safety and Efficacy Trial in PNH Patients | Paroxysmal nocturnal haemoglobinuria (PNH) MedDRA version: 20.0 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 Pharmaceutical Form: Powder for solution for injection INN or Proposed INN: COVERSIN Current Sponsor code: rVA576 Other descriptive name: COVERSIN Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 18- | Akari Therapeutics Plc | Not Recruiting | Female: yes Male: yes | 10 | Phase 2 | United Kingdom | |||
| 43 | NCT02591862 | February 2016 | 18 June 2018 | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in Patients With Resistance to Eculizumab Due to Complement C5 Polymorphisms | Paroxysmal Nocturnal Haemoglobinuria (PNH) | Drug: Coversin | AKARI Therapeutics | Radboud University | Not recruiting | 18 Years | 80 Years | All | 6 | Phase 2 | Netherlands |
| 44 | EUCTR2015-003778-34-NL | 13/01/2016 | 5 June 2018 | Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms | Coversin in Paroxysmal Nocturnal Haemoglobulinuria (PNH) in patients with resistance to Eculizumab due to complement C5 Polymorphisms - Coversin in PNH patients with resistance to Eculizumab | Coversin in Paroxysmal Nocturnal Haemoglobinuria (PNH) in patients with resistance to Eculizumab due to complement C5 polymorphisms. MedDRA version: 18.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Name: Coversin Product Code: rVA576 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: Not applicable Current Sponsor code: rVA576 Other descriptive name: coversin Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 7.2- | Volution Immuno Pharmaceuticals (UK) Ltd | Not Recruiting | Female: yes Male: yes | 6 | Phase 2 | Netherlands | |||
| 45 | NCT02605993 | January 4, 2016 | 4 March 2019 | Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 2, Open-label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;PNH | Biological: Ravulizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 26 | Phase 2 | Canada;France;Germany;Korea, Republic of;Spain;Taiwan;United Kingdom;Sweden | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | EUCTR2015-002674-20-DE | 04/12/2015 | 20 August 2018 | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | A Phase 2, Open-Label, Multiple Ascending Dose Study to Evaluate the Efficacy, Safety, Tolerability, Immunogenicity, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients with Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ALXN1210 Current Sponsor code: ALXN1210 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Product Code: ALXN1210 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ALXN1210 Current Sponsor code: ALXN1210 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- | Alexion Pharmaceuticals, Inc. | Authorised | Female: yes Male: yes | 26 | Phase 2 | France;United States;Taiwan;Canada;Finland;Spain;Russian Federation;Germany;United Kingdom;Korea, Republic of;Sweden | |||
| 47 | NCT02588833 | November 2015 | 4 February 2019 | Pilot Study to Assess Safety, Preliminary Efficacy and Pharmacokinetics of S.C. APL-2 in PNH Subjects | A Phase Ib, Open Label, Multiple Ascending Dose, Pilot Study to Assess the Safety, Preliminary Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 20 | Phase 1 | Hong Kong;Malaysia;New Zealand;Thailand | |
| 48 | NCT02598583 | November 2015 | 16 December 2017 | An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open-Label, Intrapatient, Dose-Escalation Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of ALXN1210 Administered Intravenously to Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Biological: Study Drug - ALXN1210 | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 13 | Phase 1/Phase 2 | Australia;Korea, Republic of | |
| 49 | NCT02534909 | September 9, 2015 | 11 November 2019 | Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316 in Patients With Paroxysmal Nocturnal Hemoglobinuria | An Open-label Proof of Concept Study to Assess the Efficacy, Safety and Pharmacokinetics of LFG316, an Anti-C5 Monoclonal Antibody in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria PNH | Biological: LFG316 | Novartis Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 10 | Phase 2 | Czechia;Japan;Lithuania;Czech Republic;Moldova, Republic of | |
| 50 | EUCTR2014-005338-74-CZ | 17/06/2015 | 14 January 2019 | To find out whether LFG316 is able to reduce the destruction of red blood cells in patients with PNH | An open-label proof of concept study to assess the efficacy, safety and pharmacokinetics of LFG316, an anti-C5 monoclonal antibody in patients with paroxysmal nocturnal hemoglobinuria (PNH) - CLFG316X2201 | Paroxysmal nocturnal hemoglobinuria MedDRA version: 20.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857 ;Therapeutic area: Diseases [C] - Immune System Diseases [C20] | Product Code: LFG316 Pharmaceutical Form: Lyophilisate for solution for infusion INN or Proposed INN: No Current Sponsor code: LFG316 Other descriptive name: human monoclonal antibody directed against complement 5 protein Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 100- Product Code: LFG316 Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: No Current Sponsor code: LFG316 Other descriptive name: human monoclonal antibody directed against complement 5 protein Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Novartis Pharma Services AG | Authorised | Female: yes Male: yes | 15 | Phase 2 | Czech Republic;Lithuania;Japan | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT02352493 | January 2015 | 16 December 2017 | A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH | A Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl) | Alnylam Pharmaceuticals | Not recruiting | 18 Years | 45 Years | All | 62 | Phase 1/Phase 2 | Spain;United Kingdom | |
| 52 | NCT02264639 | November 2014 | 9 July 2018 | A Phase I Study to Assess the Safety APL-2 as an Add-On to Standard of Care in Subjects With PNH | An Open Label, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of APL-2 as an Add-On to Standard of Care in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH). | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: APL-2 | Apellis Pharmaceuticals, Inc. | Not recruiting | 18 Years | N/A | All | 15 | Phase 1 | United States | |
| 53 | NCT02179359 | September 2, 2014 | 7 October 2019 | Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies | MT2014-10C: Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders | Sickle Cell Disease;Transfusion Dependent Alpha- or Beta- Thalassemia;Diamond Blackfan Anemia;Paroxysmal Nocturnal Hemoglobinuria;Glanzmann Thrombasthenia;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Non-Malignant Hematologic Disorders | Drug: Reduced Toxicity Ablative Regimen;Drug: Reduced Intensity Preparative Regimen;Drug: Myeloablative Preparative Regimen | Masonic Cancer Center, University of Minnesota | Recruiting | N/A | 55 Years | All | 25 | N/A | United States | |
| 54 | ChiCTR-TRC-14004219 | 2014-01-24 | 18 April 2017 | A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | A prospective, randomized, open label, single-center trial of comparing intravenous iron sucrose versus iron dextran tablet in the treatment of iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | iron deficiency anemia and classic paroxysmal nocturnal hemoglobinuria | intravenous iron sucrose:intravenous iron sucrose;iron dextran tablet:Oral iron dextran tablet; | Institute of Hematology and Blood Diseases Hospital,Chinese Academy of Medical Sciences | Recruiting | 18 | 65 | Both | intravenous iron sucrose:24;iron dextran tablet:24; | Other | China | |
| 55 | NCT01760096 | January 2013 | 19 February 2015 | 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) | Phase II Study of the 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) | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | Not recruiting | 18 Years | N/A | Both | 50 | Phase 2 | China | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | NCT01642979 | July 2012 | 19 February 2015 | Safety and Efficacy of Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Phase II Study of the Levamisole Combined With Cyclosporine A in Patients With Classic Paroxysmal Nocturnal Hemoglobinuria | Paroxysmal Nocturnal Hemoglobinuria;Hemoglobinuria;Hemoglobinuria, Paroxysmal;Proteinuria;Urination Disorders;Thrombosis;Bone Marrow Failure;Aplastic Anemia,;Anemia, Hemolytic | Drug: Levamisole+cyclosporin A+Glucocorticoids;Drug: cyclosporin A+Glucocorticoids;Drug: Glucocorticoids | Institute of Hematology & Blood Diseases Hospital | Not recruiting | 18 Years | N/A | Both | 120 | Phase 2 | China | |
| 57 | NCT01529827 | February 28, 2012 | 30 September 2019 | Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies | A Phase II Trial of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan and Low Dose Total Body Irradiation | Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Hairy Cell Leukemia;Refractory Multiple Myeloma;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Secondary Myelofibrosis;Severe Combined Immunodeficiency;Severe Congenital Neutropenia;Shwachman-Diamond Syndrome;Splenic Marginal Zone Lymphoma;T-cell Large Granular Lymphocyte Leukemia;Waldenstrom Macroglobulinemia;Wiskott-Aldrich Syndrome;Chronic Phase Chronic Myelogenous Leukemia;Congenital Amegakaryocytic Thrombocytopenia;Diamond-Blackfan Anemia;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Hepatosplenic T-cell Lymphoma;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Peripheral T-cell Lymphoma;Polycythemia Vera;Post-transplant Lymphoproliferative Disorder;Previously Treated Myelodysplastic Syndromes;Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Grade III Lymphomatoid Granulomatosis;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Burkitt Lymphoma;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Grade III Lymphomatoid Granulomatosis;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Myelomonocytic Leukemia | Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation;Drug: tacrolimus;Drug: mycophenolate mofetil;Drug: methotrexate;Other: laboratory biomarker analysis;Procedure: allogeneic hematopoietic stem cell transplantation;Procedure: peripheral blood stem cell transplantation | Roswell Park Cancer Institute | Not recruiting | 3 Years | 75 Years | All | 94 | Phase 2 | United States | |
| 58 | NCT01335165 | April 2011 | 19 February 2015 | Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | A Phase 1, Single Ascending Dose Study of the Safety and Pharmacokinetics of TT30 in Subjects With Paroxysmal Nocturnal Hemoglobinuria (PNH) | Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: ALXN1102;Drug: ALXN1103 | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | Both | 10 | Phase 1 | United States;Czech Republic;Italy;Poland;United Kingdom | |
| 59 | NCT00867932 | May 2009 | 16 December 2017 | Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent PNH Subjects | An Open Label Multi Center Study of Eculizumab in Children and Adolescents With Diagnosis of Paroxysmal Nocturnal Hemoglobinuria | Hemoglobinuria, Paroxysmal | Drug: Eculizumab | Alexion Pharmaceuticals | Not recruiting | 2 Years | 17 Years | All | 7 | Phase 4 | United States | |
| 60 | NCT00856388 | January 14, 2009 | 18 December 2018 | Fludarabine Phosphate, Melphalan, Total-Body Irradiation, Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Bone Marrow Failure Disorders | A Pilot Trial Of Reduced Intensity Allogeneic Stem Cell Transplantation With Fludarabine, Melphalan, And Low Dose Total Body Irradiation | Accelerated Phase Chronic Myelogenous Leukemia;Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome;Adult Acute Lymphoblastic Leukemia in Remission;Adult Acute Myeloid Leukemia in Remission;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Adult Nasal Type Extranodal NK/T-cell Lymphoma;Anaplastic Large Cell Lymphoma;Angioimmunoblastic T-cell Lymphoma;Aplastic Anemia;Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Childhood Acute Lymphoblastic Leukemia in Remission;Childhood Acute Myeloid Leukemia in Remission;Childhood Chronic Myelogenous Leukemia;Childhood Diffuse Large Cell Lymphoma;Childhood Immunoblastic Large Cell Lymphoma;Childhood Myelodysplastic Syndromes;Childhood Nasal Type Extranodal NK/T-cell Lymphoma;Chronic Eosinophilic Leukemia;Chronic Myelomonocytic Leukemia;Chronic Neutrophilic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;de Novo Myelodysplastic Syndromes;Extranodal Marginal Zone B-cell Lymphoma of Mucosa-associated Lymphoid Tissue;Fanconi Anemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic/Myeloproliferative Neoplasm, Unclassifiable;Nodal Marginal Zone B-cell Lymphoma;Noncontiguous Stage II Adult Burkitt Lymphoma;Noncontiguous Stage II Adult Diffuse Large Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Mixed Cell Lymphoma;Noncontiguous Stage II Adult Diffuse Small Cleaved Cell Lymphoma;Noncontiguous Stage II Adult Immunoblastic Large Cell Lymphoma;Noncontiguous Stage II Adult Lymphoblastic Lymphoma;Noncontiguous Stage II Grade 1 Follicular Lymphoma;Noncontiguous Stage II Grade 2 Follicular Lymphoma;Noncontiguous Stage II Grade 3 Follicular Lymphoma;Noncontiguous Stage II Mantle Cell Lymphoma;Noncontiguous Stage II Marginal Zone Lymphoma;Noncontiguous Stage II Small Lymphocytic Lymphoma;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Primary Myelofibrosis;Recurrent Adult Acute Lymphoblastic Leukemia;Recurrent Adult Acute Myeloid Leukemia;Recurrent Adult Burkitt Lymphoma;Recurrent Adult Diffuse Large Cell Lymphoma;Recurrent Adult Diffuse Mixed Cell Lymphoma;Recurrent Adult Diffuse Small Cleaved Cell Lymphoma;Recurrent Adult Grade III Lymphomatoid Granulomatosis;Recurrent Adult Hodgkin Lymphoma;Recurrent Adult Immunoblastic Large Cell Lymphoma;Recurrent Adult Lymphoblastic Lymphoma;Recurrent Adult T-cell Leukemia/Lymphoma;Recurrent Childhood Acute Lymphoblastic Leukemia;Recurrent Childhood Acute Myeloid Leukemia;Recurrent Childhood Anaplastic Large Cell Lymphoma;Recurrent Childhood Grade III Lymphomatoid Granulomatosis;Recurrent Childhood Large Cell Lymphoma;Recurrent Childhood Lymphoblastic Lymphoma;Recurrent Childhood Small Noncleaved Cell Lymphoma;Recurrent Cutaneous T-cell Non-Hodgkin Lymphoma;Recurrent Grade 1 Follicular Lymphoma;Recurrent Grade 2 Follicular Lymphoma;Recurrent Grade 3 Follicular Lymphoma;Recurrent Mantle Cell Lymphoma;Recurrent Marginal Zone Lymphoma;Recurrent Mycosis Fungoides/Sezary Syndrome;Recurrent Small Lymphocytic Lymphoma;Recurrent/Refractory Childhood Hodgkin Lymphoma;Refractory Chronic Lymphocytic Leukemia;Refractory Multiple Myeloma;Relapsing Chronic Myelogenous Leukemia;Secondary Acute Myeloid Leukemia;Secondary Myelodysplastic Syndromes;Splenic Marginal Zone Lymphoma;Stage III Adult Diffuse Small Cleaved Cell Lymphoma;Stage III Adult Immunoblastic Large Cell Lymphoma;Stage III Adult Lymphoblastic Lymphoma;Stage III Grade 1 Follicular Lymphoma;Stage III Grade 2 Follicular Lymphoma;Stage III Grade 3 Follicular Lymphoma;Stage III Mantle Cell Lymphoma;Stage III Marginal Zone Lymphoma;Stage III Small Lymphocytic Lymphoma;Stage IV Adult Burkitt Lymphoma;Stage IV Adult Diffuse Small Cleaved Cell Lymphoma;Stage IV Adult Immunoblastic Large Cell Lymphoma;Stage IV Adult Lymphoblastic Lymphoma;Stage IV Grade 1 Follicular Lymphoma;Stage IV Grade 2 Follicular Lymphoma;Stage IV Grade 3 Follicular Lymphoma;Stage IV Mantle Cell Lymphoma;Stage IV Marginal Zone Lymphoma;Stage IV Small Lymphocytic Lymphoma;Waldenström Macroglobulinemia | Procedure: allogeneic hematopoietic stem cell transplantation;Biological: anti-thymocyte globulin;Drug: fludarabine phosphate;Drug: melphalan;Radiation: total-body irradiation | Roswell Park Cancer Institute | National Cancer Institute (NCI) | Not recruiting | 3 Years | 75 Years | All | 62 | N/A | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | EUCTR2009-010402-11-Outside-EU/EEA | 8 May 2012 | Not Applicable | AN OPEN-LABEL MULTI-CENTER STUDY OF ECULIZUMAB IN CHILDREN AND ADOLESCENTS WITH A DIAGNOSIS OF PAROXYSMAL NOCTURNAL HEMOGLOBINURIA | PAROXYSMAL NOCTURNAL HEMOGLOBINURIA MedDRA version: 14.1 Level: PT Classification code 10034042 Term: Paroxysmal nocturnal haemoglobinuria System Organ Class: 10038359 - Renal and urinary disorders ;Therapeutic area: Body processes [G] - Immune system processes [G12] | Trade Name: SOLIRIS Pharmaceutical Form: Concentrate for solution for infusion INN or Proposed INN: ECULIZUMAB CAS Number: 219685-50-4 Current Sponsor code: h5G1.1-mAb Other descriptive name: Anti-C5 antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | ALEXION PHARMACEUTICALS, INC. | Not Available | Female: yes Male: yes | 6 | United States | |||||
| 62 | NCT01194804 | April 2008 | 30 September 2019 | E07-001: Safety and Efficacy Extension Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients: Extension Study to C07-001 Protocol | Paroxysmal Nocturnal Hemoglobinuria | Drug: Eculizumab | Alexion Pharmaceuticals | CMIC Co, Ltd. Japan | Not recruiting | 12 Years | N/A | All | 27 | Phase 2 | |
| 63 | NCT01192399 | November 2007 | 16 December 2017 | C07-001: Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Phase II Clinical Study of Eculizumab (h5G1.1-mAb) in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients | Paroxysmal Nocturnal Hemoglobinuria | Biological: Eculizumab | Alexion Pharmaceuticals | Not recruiting | 12 Years | N/A | All | 29 | Phase 2 | ||
| 64 | NCT00438789 | February 2007 | 19 February 2015 | The Paroxysmal Nocturnal Hemoglobinuria Early Access Treatment Protocol | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | Both | Phase 3 | United States | |||
| 65 | NCT00397813 | January 2006 | 3 June 2019 | Fludarabine Phosphate and Total Body Irradiation Followed by a Donor Peripheral Stem Cell Transplant in Treating Patients With Myelodysplastic Syndromes or Myeloproliferative Disorders | Low-Dose TBI Dose Escalation to Decrease Risks of Progression and Graft Rejection After Hematopoietic Cell Transplantation With Nonmyeloablative Conditioning as Treatment for Untreated Myelodysplastic Syndrome or Myeloproliferative Disorders - A Multi-Center Trial | Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative;Chronic Myelomonocytic Leukemia;de Novo Myelodysplastic Syndrome;Essential Thrombocythemia;Myeloproliferative Neoplasm;Paroxysmal Nocturnal Hemoglobinuria;Polycythemia Vera;Polycythemia Vera, Post-Polycythemic Myelofibrosis Phase;Primary Myelofibrosis;Refractory Anemia;Refractory Anemia With Excess Blasts;Refractory Anemia With Ring Sideroblasts;Refractory Cytopenia With Multilineage Dysplasia;Refractory Cytopenia With Multilineage Dysplasia and Ring Sideroblasts | Drug: Cyclosporine;Drug: Fludarabine Phosphate;Other: Laboratory Biomarker Analysis;Drug: Mycophenolate Mofetil;Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation;Procedure: Peripheral Blood Stem Cell Transplantation;Radiation: Total-Body Irradiation | Fred Hutchinson Cancer Research Center | National Cancer Institute (NCI);National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | 50 Years | 75 Years | All | 77 | Phase 2 | United States;Denmark;Germany;Italy |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | NCT00130000 | August 9, 2005 | 16 December 2017 | Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria | SHEPHERD: Safety in Hemolytic PNH Patients Treated With Eculizumab: A Multi-Center Open-Label Research Design | Paroxysmal Hemoglobinuria, Nocturnal | Drug: Eculizumab | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | 18 Years | N/A | All | 87 | Phase 3 | United States | |
| 67 | NCT00143559 | August 2005 | 19 February 2015 | Stem Cell Transplantation as Immunotherapy for Hematologic Malignancies | Haploidentical Hematopoietic Stem Cell Transplantation Utilizing Partial T-Cell Depletion as Immunotherapy for Hematologic Malignancies | Leukemia;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria;Hodgkin's Lymphoma;Non-Hodgkin Lymphoma | Drug: Systematic chemotherapy and antibodies;Procedure: Allogeneic stem cell transplantation;Device: Miltenyi CliniMACS | St. Jude Children's Research Hospital | Not recruiting | 2 Years | 21 Years | Both | 17 | Phase 2 | United States | |
| 68 | NCT00122317 | May 2005 | 16 December 2017 | Extension Study of Eculizumab in Patients With Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) | Open Label Extension Study of Eculizumab in Patients With Transfusion Dependent PNH | Paroxysmal Hemoglobinuria, Nocturnal | Drug: eculizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | All | 187 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;United Kingdom | |
| 69 | EUCTR2005-000043-28-GB | 19/04/2005 | 18 April 2012 | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | A Phase III, Open-Label, Extension Study of Eculizumab in Patients with Transfusion Dependent, Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Participated in the TRIUMPH (C04-001), SHEPHERD (C04-002) or X03-001 Studies. - E05 Extension | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0 Level: PT Classification code 10034042 | Product Name: eculizumab Pharmaceutical Form: Solution for infusion INN or Proposed INN: eculizumab Current Sponsor code: h5G1.1-mAb Other descriptive name: Anti-C5 antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Solution for infusion Route of administration of the placebo: Intravenous use | ALEXION PHARMACEUTICALS INCORPORATED | Authorised | Female: yes Male: yes | 170 | Phase 3 | Germany;United Kingdom;Spain;Ireland;Italy;Sweden | |||
| 70 | EUCTR2004-002795-42-SE | 16/02/2005 | 8 May 2012 | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | Safety in Hemolytic PNH Patients Treated with Eculizumab: A Multi-center Open-label Research Design Study - SHEPHERD | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0 Level: PT Classification code 10034042 | Product Name: eculizumab Pharmaceutical Form: Solution for infusion INN or Proposed INN: eculizumab Current Sponsor code: h5G1.1-mAb Other descriptive name: Anti-C5 antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- | ALEXION PHARMACEUTICALS INCORPORATED | Not Recruiting | Female: yes Male: yes | 85 | United Kingdom;Germany;Spain;Ireland;Italy;Sweden | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | NCT00098280 | December 2004 | 19 February 2015 | Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Hemoglobinuria, Paroxysmal | Drug: Eculizumab | National Heart, Lung, and Blood Institute (NHLBI) | Not recruiting | N/A | N/A | Both | 75 | Phase 3 | United States | |
| 72 | NCT00122304 | December 2004 | 19 February 2015 | Study of Safety in Hemolytic Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Treated With Eculizumab | Safety in Hemolytic PNH Patients Treated With Eculizumab: a Multi-Center Open-Label Research Design Study | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | Both | 85 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Spain;Sweden;Switzerland;United Kingdom | |
| 73 | NCT00112983 | November 2004 | 19 February 2015 | Eculizumab in Treating Patients With Paroxysmal Nocturnal Hemoglobinuria | TRIUMPH: A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients | Leukemia | Biological: eculizumab | Jonsson Comprehensive Cancer Center | Not recruiting | 18 Years | N/A | Both | Phase 3 | United States | ||
| 74 | NCT00122330 | October 2004 | 19 February 2015 | Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients | Randomized, Double-Blind, and Placebo-Controlled Study Using Eculizumab in Transfusion Dependent PNH Patients | Hemoglobinuria, Paroxysmal | Drug: eculizumab | Alexion Pharmaceuticals | Not recruiting | 18 Years | N/A | Both | 75 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Ireland;Italy;Netherlands;Sweden;Switzerland;United Kingdom | |
| 75 | EUCTR2004-000646-20-SE | 21/09/2004 | 19 March 2012 | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | A Hemoglobin Stabilization and Transfusion Reduction Efficacy and Safety Clinical Investigation, Randomized, Multi-Center, Double-Blind, Placebo-Controlled, Using Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients - TRIUMPH | paroxysmal nocturnal hemoglobinuria MedDRA version: 7.0 Level: PT Classification code 10034042 | Product Name: eculizumab Pharmaceutical Form: Solution for infusion INN or Proposed INN: eculizumab Current Sponsor code: h5G1.1-mAb Other descriptive name: Anti-C5 antibody Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Intravenous infusion Route of administration of the placebo: Intravenous use | ALEXION PHARMACEUTICALS INCORPORATED | Not Recruiting | Female: yes Male: yes | 75 | Ireland;Italy;Sweden | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 76 | NCT00145613 | June 2003 | 19 February 2015 | Haploidentical Stem Cell Transplant for Treatment Refractory Hematological Malignancies | Haploidentical Stem Cell Transplantation Utilizing T-Cell Depletion as Therapy for Patients With Refractory Hematological Malignancies | Acute Lymphoblastic Leukemia (ALL);Acute Myeloid Leukemia (AML);Secondary AML;Myelodysplastic Syndrome (MDS);Secondary MDS;Chronic Myeloid Leukemia;Juvenile Myelomonocytic Leukemia (JMML);Paroxysmal Nocturnal Hemoglobinuria (PNH);Lymphoma, Non-Hodgkin;Hodgkin Disease | Procedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodies | St. Jude Children's Research Hospital | Not recruiting | 2 Years | 21 Years | Both | 25 | Phase 2 | United States | |
| 77 | NCT00587054 | June 2001 | 16 December 2017 | 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 II Trial of Allogeneic T-Cell Depleted Hematopoietic Stem Cell Transplants From HLA Compatible, Related and Unrelated Donors After a Myeloablative Preparative Regimen With Hyperfractionated TBI, Thiotepa and Fludarabine For Treatment of Adult Patients (>18 Years) With Lymphohematopoietic Disorders | Allogeneic Stem Cell Transplant;Leukemia;Non-Hodgkins;Lymphoblastic Lymphoma;Myelodysplastic Syndrome;Paroxysmal Nocturnal Hemoglobinuria (PNH) | Drug: cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant | Memorial Sloan Kettering Cancer Center | National Cancer Institute (NCI) | Not recruiting | 18 Years | 55 Years | All | 129 | Phase 2 | United States |
| 78 | NCT00012376 | March 2001 | 19 February 2015 | Chemotherapy Plus Sargramostim in Treating Patients With Refractory Myeloid Cancer | Dose Finding Study of Bryostatin-1 and GM-CSF in Refractory Myeloid Malignancies | Accelerated Phase Chronic Myelogenous Leukemia;Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities;Adult Acute Myeloid Leukemia With Del(5q);Adult Acute Myeloid Leukemia With Inv(16)(p13;q22);Adult Acute Myeloid Leukemia With t(15;17)(q22;q12);Adult Acute Myeloid Leukemia With t(16;16)(p13;Adult Acute Myeloid Leukemia With t(8;21)(q22;Blastic Phase Chronic Myelogenous Leukemia;Chronic Myelomonocytic Leukemia;Chronic Phase Chronic Myelogenous Leukemia;Paroxysmal Nocturnal Hemoglobinuria;Previously Treated Myelodysplastic Syndromes;Recurrent Adult Acute Myeloid Leukemia;Refractory Anemia;Refractory Anemia With Ringed Sideroblasts;Relapsing Chronic Myelogenous Leukemia;Thrombocytopenia;Untreated Adult Acute Myeloid Leukemia | Drug: bryostatin 1;Biological: sargramostim;Other: laboratory biomarker analysis;Other: pharmacological study | National Cancer Institute (NCI) | Not recruiting | 18 Years | N/A | Both | 35 | Phase 1 | United States | |
| 79 | NCT00004143 | September 1999 | 19 October 2017 | Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes | Allogeneic Mixed Chimerism Stem Cell Transplantation Utilizing In Vivo and In Vitro Campath for Hemoglobinopathies and Bone Marrow Failure Syndromes | Sickle Cell Anemia;Severe Aplastic Anemia;Paroxysmal Nocturnal Hemoglobinuria (PNH);Pure Red Cell Aplasia | Drug: Campath, Chemo and/or TBI Allo SCT | David Rizzieri, MD | Not recruiting | 18 Years | N/A | All | 2 | Phase 2 | United States | |
| 80 | NCT00004464 | February 1996 | 28 January 2019 | Study of High Dose Cyclophosphamide in Patients With Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria | High Dose Cyclophosphamide for the Treatment of Severe Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria | Aplastic Anemia;Paroxysmal Hemoglobinuria, Nocturnal | Drug: cyclophosphamide;Drug: filgrastim | Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins | Not recruiting | N/A | 70 Years | All | 69 | Phase 2 | United States |