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	EUCTR2019-004665-40-NL (EUCTR)	20/11/2020	09/11/2020	Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment	A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody	Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;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 Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab	Novartis Pharma AG	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	91	Phase 3	United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of
2	EUCTR2019-004665-40-HU (EUCTR)	17/11/2020	23/09/2020	Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment	A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody	Paroxysmal Nocturnal Hemoglobinuria (PNH) MedDRA version: 21.1;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 Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Product Name: LNP023 Product Code: LNP023 INN or Proposed INN: not yet established Other descriptive name: LNP023 HYDROCHLORIDE SALT Trade Name: Soliris Product Name: Eculizumab INN or Proposed INN: ECULIZUMAB Trade Name: Ultomiris Product Name: Ravulizumab INN or Proposed INN: RAVULIZUMAB Other descriptive name: ravulizumab	Novartis Pharma AG	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	91	Phase 3	United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of
3	EUCTR2019-003440-74-GB (EUCTR)	14/05/2020	17/03/2020	Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab	Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401	Paroxysmal nocturnal hemoglobinuria (PNH) MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Trade Name: ULTOMIRIS Product Name: ULTOMIRIS Product Code: ALXN1210 INN or Proposed INN: RAVULIZUMAB	Alexion Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	20	Phase 4	United Kingdom
4	EUCTR2019-001453-10-ES (EUCTR)	16/09/2019	09/08/2019	A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria	A MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA	Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Trade Name: Soliris 300 Product Name: ECULIZUMAB INN or Proposed INN: ECULIZUMAB Trade Name: ULTOMIRIS Product Name: Ultomiris INN or Proposed INN: RAVULIZUMAB Other descriptive name: Ultomiris	Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd	NULL	Not Recruiting			Female: yes Male: yes	200	Phase 4	United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of
5	EUCTR2017-002370-39-SE (EUCTR)	27/03/2019	30/11/2018	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: 21.1;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 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab Trade Name: ULTOMIRIS Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5 Other descriptive name: Ravulizumab	Alexion Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	105	Phase 3	United States;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Sweden;Korea, Republic of
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	EUCTR2017-002820-26-GB (EUCTR)	24/11/2017	26/10/2017	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: 21.1;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]	Trade Name: Ultomiris Product Name: Ravulizumab Product Code: ALXN1210 INN or Proposed INN: Ravulizumab Other descriptive name: ALXN1210	Alexion Pharmaceuticals Incorporated	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	13	Phase 3	United States;France;Russian Federation;Norway;Netherlands;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

EUCTR2019-004665-40-NL
(EUCTR)

20/11/2020

09/11/2020

Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment

A randomized, multicenter, active-comparator controlled, open-label trial to evaluate efficacy and safety of oral, twice daily LNP023 in adult patients with PNH and residual anemia, despite treatment with an intravenous anti-C5 antibody

Paroxysmal Nocturnal Hemoglobinuria (PNH)
MedDRA version: 21.1;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 Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name: LNP023 HYDROCHLORIDE SALT
Product Name: LNP023
Product Code: LNP023
INN or Proposed INN: not yet established
Other descriptive name: LNP023 HYDROCHLORIDE SALT
Trade Name: Soliris
Product Name: Eculizumab
INN or Proposed INN: ECULIZUMAB
Trade Name: Ultomiris
Product Name: Ravulizumab
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: ravulizumab

Novartis Pharma AG

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Czechia;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Canada;Brazil;Netherlands;Germany;Japan;Korea, Republic of

EUCTR2019-004665-40-HU
(EUCTR)

17/11/2020

23/09/2020

Study of efficacy and safety of twice daily oral LNP023 in adult PNH patients with residual anemia despite anti-C5 antibody treatment

Novartis Pharma AG

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Taiwan;Spain;Italy;United Kingdom;France;Hungary;Czech Republic;Canada;Brazil;Germany;Netherlands;Japan;Korea, Republic of

EUCTR2019-003440-74-GB
(EUCTR)

14/05/2020

17/03/2020

Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab

Phase 4, Single-Arm Study of Ravulizumab in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with High-Dose Eculizumab - ALXN1210-PNH-401

Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Trade Name: ULTOMIRIS
Product Name: ULTOMIRIS
Product Code: ALXN1210
INN or Proposed INN: RAVULIZUMAB

Alexion Pharmaceuticals Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 4

United Kingdom

EUCTR2019-001453-10-ES
(EUCTR)

16/09/2019

09/08/2019

A Low-Interventional Study Documenting the Efficacy, Health-Related Quality of Life, and Safety of Standard-Of-Care Treatment with Eculizumab or Treatment with Ravulizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria

A MULTICENTER, LOW-INTERVENTIONAL STUDY DOCUMENTING THE EFFICACY, HEALTH-RELATED QUALITY OF LIFE, AND SAFETY OF STANDARD-OF-CARE TREATMENT WITH ECULIZUMAB OR TREATMENT WITH RAVULIZUMAB IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Paroxysmal nocturnal hemoglobinuria (PNH) that is treated with either eculizumab or ravulizumab as per local label
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Trade Name: Soliris 300
Product Name: ECULIZUMAB
INN or Proposed INN: ECULIZUMAB
Trade Name: ULTOMIRIS
Product Name: Ultomiris
INN or Proposed INN: RAVULIZUMAB
Other descriptive name: Ultomiris

Roche Farma S. A. U. que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche Ltd

NULL

Not Recruiting

Female: yes
Male: yes

200

Phase 4

United States;Taiwan;Greece;Spain;Turkey;Israel;Colombia;United Kingdom;Italy;France;Czech Republic;Hungary;Canada;Argentina;Poland;Brazil;Romania;Netherlands;Germany;Japan;Sweden;Korea, Republic of

EUCTR2017-002370-39-SE
(EUCTR)

27/03/2019

30/11/2018

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: 21.1;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
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab
Trade Name: ULTOMIRIS
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Fc- and CDR-modified humanised monoclonal antibody against C5
Other descriptive name: Ravulizumab

Alexion Pharmaceuticals, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

105

Phase 3

United States;Finland;Spain;Turkey;Austria;Russian Federation;United Kingdom;Italy;France;Czech Republic;Canada;Belgium;Brazil;Australia;Germany;Netherlands;Sweden;Korea, Republic of

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

EUCTR2017-002820-26-GB
(EUCTR)

24/11/2017

26/10/2017

A Phase 3, Open-Label Study of ALXN1210 in Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Trade Name: Ultomiris
Product Name: Ravulizumab
Product Code: ALXN1210
INN or Proposed INN: Ravulizumab
Other descriptive name: ALXN1210

Alexion Pharmaceuticals Incorporated

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;France;Russian Federation;Norway;Netherlands;United Kingdom