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	EUCTR2019-003829-18-GB (EUCTR)	18/09/2020	05/02/2020	Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)	A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)	Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH) 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: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN Product Name: Danicopan (ALXN2040) Product Code: ACH-0144471 INN or Proposed INN: DANICOPAN Other descriptive name: DANICOPAN	Alexion Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	84	Phase 3	United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
2	EUCTR2017-000665-79-GB (EUCTR)	23/07/2018	27/04/2018	A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100	An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100	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]		Achillion Pharmaceuticals, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	12	Phase 2	New Zealand;Italy;United Kingdom;Korea, Republic of
3	EUCTR2016-003526-16-GB (EUCTR)	20/07/2018	23/04/2018	A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have poor Response to Eculizumab Monotherapy	A Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy	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]	Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471 Product Name: Danicopan Product Code: ALXN2040 (ACH-0144471) INN or Proposed INN: Not available Other descriptive name: ACH-0144471	Alexion Pharmaceuticals Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	14	Phase 2	United States;Italy;United Kingdom
4	NCT03472885 (ClinicalTrials.gov)	May 8, 2018	15/3/2018	A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab	A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Drug: ACH-0144471;Drug: Eculizumab	Alexion Pharmaceuticals	Achillion, a wholly owned subsidiary of Alexion	Active, not recruiting	18 Years	65 Years	All	12	Phase 2	United States
5	EUCTR2016-002652-25-GB (EUCTR)	02/02/2018	27/10/2017	A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)	A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Untreated 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]		Achillion Pharmaceuticals, Inc.	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 2	New Zealand;Italy;United Kingdom;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	NCT03181633 (ClinicalTrials.gov)	June 22, 2017	5/6/2017	A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100	Paroxysmal Nocturnal Hemoglobinuria	Drug: ACH-0144471	Alexion Pharmaceuticals	NULL	Active, not recruiting	18 Years	N/A	All	8	Phase 2	New Zealand
7	NCT03053102 (ClinicalTrials.gov)	March 31, 2017	1/2/2017	A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Drug: ACH-0144471	Alexion Pharmaceuticals	NULL	Completed	18 Years	N/A	All	10	Phase 2	Italy;Korea, Republic of;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

EUCTR2019-003829-18-GB
(EUCTR)

18/09/2020

05/02/2020

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)

A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)

Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
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: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN
Product Name: Danicopan (ALXN2040)
Product Code: ACH-0144471
INN or Proposed INN: DANICOPAN
Other descriptive name: DANICOPAN

Alexion Pharmaceuticals Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of

EUCTR2017-000665-79-GB
(EUCTR)

23/07/2018

27/04/2018

A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100

An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment with ACH-0144471 in Patients with PNH who Completed Clinical Study ACH471-100

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]

Achillion Pharmaceuticals, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

New Zealand;Italy;United Kingdom;Korea, Republic of

EUCTR2016-003526-16-GB
(EUCTR)

20/07/2018

23/04/2018

A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have poor Response to Eculizumab Monotherapy

A Phase 2 Open-label Study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

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]

Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471
Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471
Product Name: Danicopan
Product Code: ALXN2040 (ACH-0144471)
INN or Proposed INN: Not available
Other descriptive name: ACH-0144471

Alexion Pharmaceuticals Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Italy;United Kingdom

NCT03472885
(ClinicalTrials.gov)

May 8, 2018

15/3/2018

A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to Eculizumab

A Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab Monotherapy

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Drug: ACH-0144471;Drug: Eculizumab

Alexion Pharmaceuticals

Achillion, a wholly owned subsidiary of Alexion

Active, not recruiting

18 Years

65 Years

All

Phase 2

United States

EUCTR2016-002652-25-GB
(EUCTR)

02/02/2018

27/10/2017

A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Untreated 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]

Achillion Pharmaceuticals, Inc.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

New Zealand;Italy;United Kingdom;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

NCT03181633
(ClinicalTrials.gov)

June 22, 2017

5/6/2017

A Long-term Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

An Open-Label Study to Evaluate Efficacy and Safety of Long-term Treatment With ACH-0144471 in Patients Who Completed Clinical Study ACH471-100

Paroxysmal Nocturnal Hemoglobinuria

Drug: ACH-0144471

Alexion Pharmaceuticals

NULL

Active, not recruiting

18 Years

N/A

All

Phase 2

New Zealand

NCT03053102
(ClinicalTrials.gov)

March 31, 2017

1/2/2017

A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Drug: ACH-0144471

Alexion Pharmaceuticals

NULL

Completed

18 Years

N/A

All

Phase 2

Italy;Korea, Republic of;New Zealand;United Kingdom