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	EUCTR2016-003522-16-FI (EUCTR)	20/06/2017	27/12/2016	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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	Hungary;Canada;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
3	EUCTR2016-003523-34-DK (EUCTR)	01/05/2017	14/02/2017	Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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 Haemoglobinuria (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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	28	Phase 2	United States;Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom
4	EUCTR2016-003522-16-DK (EUCTR)	01/05/2017	14/02/2017	Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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 Haemoglobinuria (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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	Hungary;Canada;Finland;Australia;Denmark;Germany;New Zealand;United Kingdom
5	EUCTR2016-003522-16-HU (EUCTR)	17/03/2017	31/01/2017	Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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 Haemoglobinuria (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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	Hungary;Finland;Denmark;Australia;Germany;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
6	EUCTR2016-003523-34-HU (EUCTR)	17/03/2017	31/01/2017	Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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 Haemoglobinuria (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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24	Ra Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	28	Phase 2	United States;Hungary;Finland;Denmark;Australia;Germany;New Zealand;United Kingdom
7	EUCTR2016-003522-16-GB (EUCTR)	15/02/2017	03/01/2017	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 INN or Proposed INN: RA101495 sodium Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24	Ra Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	20	Phase 2	Hungary;Canada;Finland;Denmark;Australia;Germany;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

EUCTR2016-003522-16-FI
(EUCTR)

20/06/2017

27/12/2016

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
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

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

EUCTR2016-003523-34-DK
(EUCTR)

01/05/2017

14/02/2017

Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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 Haemoglobinuria (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
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

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

EUCTR2016-003522-16-DK
(EUCTR)

01/05/2017

14/02/2017

Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (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

Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

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

EUCTR2016-003522-16-HU
(EUCTR)

17/03/2017

31/01/2017

Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)

Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Hungary;Finland;Denmark;Australia;Germany;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

EUCTR2016-003523-34-HU
(EUCTR)

17/03/2017

31/01/2017

Extension Study of RA101495 in Paroxysmal Nocturnal Haemoglobinuria (PNH)

A Multicenter, Open-label, Uncontrolled, Extension Study of RA101495 in Subjects with Paroxysmal Nocturnal Hemoglobinuria Who Have Completed a RA101495 Clinical Study

Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24

Ra Pharmaceuticals, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

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

EUCTR2016-003522-16-GB
(EUCTR)

15/02/2017

03/01/2017

Study of RA101495 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

Product Name: RA101495
Product Code: RA101495
INN or Proposed INN: RA101495 sodium
Other descriptive name: Synthetic 15-amino-acid macrocyclic peptide acylated with a ethyleneglycol24

Ra Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

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