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	EUCTR2016-004129-18-LT (EUCTR)	15/03/2019	29/01/2019	An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;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 INN or Proposed INN: rVA576 Other descriptive name: COVERSIN	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India
2	NCT03588026 (ClinicalTrials.gov)	June 7, 2018	1/6/2018	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	NULL	Completed	18 Years	N/A	All	9	Phase 3	Kazakhstan;Lithuania;Sri Lanka
3	EUCTR2017-003847-39-LT (EUCTR)	12/03/2018	23/01/2018	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: 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 Code: rVA576 INN or Proposed INN: NA	Akari Therapeutics Plc.	NULL	Not Recruiting			Female: yes Male: yes	30	Phase 3	Ecuador;Turkey;Romania;Lithuania;Peru;Kazakhstan;Sri Lanka
4	EUCTR2016-004129-18-NL (EUCTR)	14/02/2018	16/11/2017	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: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;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 INN or Proposed INN: COVERSIN Other descriptive name: COVERSIN	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	Argentina;Poland;Lithuania;Netherlands;United Kingdom
5	EUCTR2016-004129-18-GB (EUCTR)	17/03/2017	21/12/2016	An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;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 INN or Proposed INN: rVA576 Other descriptive name: rVA576	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;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	NCT03829449 (ClinicalTrials.gov)	March 13, 2017	20/12/2018	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	NULL	Recruiting	18 Years	N/A	All	50	Phase 3	Poland
7	EUCTR2016-004129-18-PL (EUCTR)	07/03/2017	18/01/2017	An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study	CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study	Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS) MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders MedDRA version: 21.1;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: rVA576 (Coversin) Product Code: rVA576 INN or Proposed INN: rVA576 (COVERSIN) Other descriptive name: rVA576 (COVERSIN)	Akari Therapeutics Plc	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 3	France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom
8	EUCTR2015-003778-34-NL (EUCTR)	13/01/2016	10/12/2015	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 INN or Proposed INN: Not applicable Other descriptive name: coversin	Volution Immuno Pharmaceuticals (UK) Ltd	NULL	Not Recruiting			Female: yes Male: yes	6	Phase 2	Netherlands

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

EUCTR2016-004129-18-LT
(EUCTR)

15/03/2019

29/01/2019

An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS)
MedDRA version: 21.1;Level: PT;Classification code 10018932;Term: Haemolytic uraemic syndrome;System Organ Class: 10005329 - Blood and lymphatic system disorders
MedDRA version: 21.1;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
INN or Proposed INN: rVA576
Other descriptive name: COVERSIN

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom;India

NCT03588026
(ClinicalTrials.gov)

June 7, 2018

1/6/2018

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

NULL

Completed

18 Years

N/A

All

Phase 3

Kazakhstan;Lithuania;Sri Lanka

EUCTR2017-003847-39-LT
(EUCTR)

12/03/2018

23/01/2018

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: 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 Code: rVA576
INN or Proposed INN: NA

Akari Therapeutics Plc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Ecuador;Turkey;Romania;Lithuania;Peru;Kazakhstan;Sri Lanka

EUCTR2016-004129-18-NL
(EUCTR)

14/02/2018

16/11/2017

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

Product Name: Coversin
Product Code: rVA576
INN or Proposed INN: COVERSIN
Other descriptive name: COVERSIN

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

Argentina;Poland;Lithuania;Netherlands;United Kingdom

EUCTR2016-004129-18-GB
(EUCTR)

17/03/2017

21/12/2016

An extension study on rVA576 (Coversin) for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Product Code: rVA576
INN or Proposed INN: rVA576
Other descriptive name: rVA576

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;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

NCT03829449
(ClinicalTrials.gov)

March 13, 2017

20/12/2018

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

NULL

Recruiting

18 Years

N/A

All

Phase 3

Poland

EUCTR2016-004129-18-PL
(EUCTR)

07/03/2017

18/01/2017

An extension study on Coversin for Patients with PNH and aHUS who received benefit from their initial study

CONSERVE: rVA576 (Coversin) Long Term Safety and Efficacy Surveillance Study

Product Name: rVA576 (Coversin)
Product Code: rVA576
INN or Proposed INN: rVA576 (COVERSIN)
Other descriptive name: rVA576 (COVERSIN)

Akari Therapeutics Plc

NULL

Not Recruiting

Female: yes
Male: yes

Phase 3

France;Argentina;Poland;Lithuania;Kazakhstan;Netherlands;Germany;Sri Lanka;United Kingdom

EUCTR2015-003778-34-NL
(EUCTR)

13/01/2016

10/12/2015

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
INN or Proposed INN: Not applicable
Other descriptive name: coversin

Volution Immuno Pharmaceuticals (UK) Ltd

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

Netherlands