DDrare: Database of Drug Development for Rare Diseases

Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ABP 959; ABP 959 - biosimilar to eculizumab; ABP 959 - proposed biosimilar to eculizumab; ACH-0144471; ACH-0145228; ALN-62643; ALN-CC5; ALXN1102; ALXN1103; ALXN1210; ALXN2040 (ACH-0144471); ALXN2050; APL-2; Anti-C5 antibody; Anti-thymocyte globulin; BCD-148; BCX9930; BCX9930 hydrochloride; Bryostatin 1; C5 Inhibitor; C5 inh MAb, SKY59, RO/CH7092230; C5 inhibitor background therapy; COVERSIN; CROVALIMAB; Campath; Campath, Chemo and/or TBI Allo SCT; Coversin; Crovalimab; Cyclophosphamide; Cyclosporin; Cyclosporin A; Cyclosporin A+Glucocorticoids; Cyclosporine; Cytoreductive regimen followed by a CD34+E- selected allogeneic stem cell transplant; DANICOPAN; Danicopan; Danicopan (ALXN2040); Device: Miltenyi Biotec CliniMACS; Device: Miltenyi CliniMACS; Dextran; ECULIZUMAB; Eculizumab; Elizaria®; Fc- and CDR-modified humanised monoclonal antibody against C5; Filgrastim; Fludarabine; Fludarabine Phosphate; Fludarabine phosphate; G-CSF; Glucocorticoids; H5G1.1-mAb; Human monoclonal antibody directed against complement 5 protein; Iron Dextran; Iron sucrose; L04AA25; LFG316; LNP023; Levamisole; Levamisole+cyclosporin A+Glucocorticoids; Melphalan; Methotrexate; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Myeloablative Preparative Regimen; No; Other: Blood collection for measurement of eculizumab peak concentrations; Other: Laboratory Biomarker Analysis; Other: Standard of care (SOC); Other: laboratory biomarker analysis; Other: pharmacological study; Pegcetacoplan; Pegcetacoplan (APL-2); Phosphate; Procedure: Allogeneic stem cell transplantation; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Stem Cell Transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; RA101495; RA101495 sodium; RAVULIZUMAB; REGN3918; RO7112689; RO7112689/F01; RO7112689/F03-01; RO7112689/F03-03; RVA576; RVA576 (COVERSIN); RVA576 (Coversin); Radiation: Total-Body Irradiation; Radiation: total-body irradiation; Ravulizumab; Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; SB12 (proposed eculizumab biosimilar); SOLIRIS; Sargramostim; Sirolimus; Soliris; Soliris (eculizumab); Soliris 300; Soliris®; Sterile Normal Saline (0.9% NaCl); Sucrose; Synthetic 15-amino-acid macrocyclic peptide; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24; Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24 palmitoylated linker; Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker; Systematic chemotherapy and antibodies; Systemic chemotherapy and antibodies; Tacrolimus; ULTOMIRIS; Ultomiris; VARI; ZILUCOPLAN; Zilucoplan; Zilucoplan (RA101495);  LNP023 HYDROCHLORIDE SALT

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00587054 (ClinicalTrials.gov)	June 2001	21/12/2007	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)	Completed	18 Years	55 Years	All	129	Phase 2	United States

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NCT00587054
(ClinicalTrials.gov)

June 2001

21/12/2007

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)

Completed

18 Years

55 Years

All

129

Phase 2

United States