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	EUCTR2018-003956-19-FI (EUCTR)	13/02/2019	28/01/2019	Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)	Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria - Phase 3 Study in Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria Patients	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 Name: Zilucoplan Product Code: RA101495 INN or Proposed INN: ZILUCOPLAN Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	40	Phase 3	United States;Hong Kong;Taiwan;Finland;Spain;Turkey;Russian Federation;Italy;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Malaysia;Belgium;Brazil;Australia;Georgia;Germany;New Zealand;Korea, Republic of
2	NCT03225287 (ClinicalTrials.gov)	July 17, 2017	17/7/2017	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	NULL	Active, not recruiting	18 Years	N/A	All	28	Phase 2	United States;Australia;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom
3	NCT03030183 (ClinicalTrials.gov)	April 17, 2017	20/1/2017	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	NULL	Completed	18 Years	N/A	All	3	Phase 2	United States
4	NCT03078582 (ClinicalTrials.gov)	March 8, 2017	8/3/2017	Phase 2 Safety and Efficacy Study of Zilucoplan (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: Zilucoplan (RA101495)	Ra Pharmaceuticals	NULL	Completed	18 Years	N/A	All	26	Phase 2	Australia;Canada;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom

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

EUCTR2018-003956-19-FI
(EUCTR)

13/02/2019

28/01/2019

Study of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Phase 3, Multicenter, Open-Label, Single-Arm Study to Confirm the Safety and Efficacy of Zilucoplan in Treatment-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria - Phase 3 Study in Treatment-Naive Paroxysmal Nocturnal Hemoglobinuria Patients

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 Name: Zilucoplan
Product Code: RA101495
INN or Proposed INN: ZILUCOPLAN
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

United States;Hong Kong;Taiwan;Finland;Spain;Turkey;Russian Federation;Italy;United Kingdom;Czech Republic;Mexico;Canada;Argentina;Malaysia;Belgium;Brazil;Australia;Georgia;Germany;New Zealand;Korea, Republic of

NCT03225287
(ClinicalTrials.gov)

July 17, 2017

17/7/2017

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

NULL

Active, not recruiting

18 Years

N/A

All

Phase 2

United States;Australia;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom

NCT03030183
(ClinicalTrials.gov)

April 17, 2017

20/1/2017

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

NULL

Completed

18 Years

N/A

All

Phase 2

United States

NCT03078582
(ClinicalTrials.gov)

March 8, 2017

8/3/2017

Phase 2 Safety and Efficacy Study of Zilucoplan (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: Zilucoplan (RA101495)

Ra Pharmaceuticals

NULL

Completed

18 Years

N/A

All

Phase 2

Australia;Canada;Denmark;Finland;Germany;Hungary;New Zealand;United Kingdom