62. Paroxysmal nocturnal hemoglobinuria
202 clinical trials,   120 drugs   (DrugBank: 25 drugs),   15 drug target genes,   90 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
7 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2019-003829-18-GB
(EUCTR)
18/09/202005/02/2020Danicopan 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
84Phase 3United States;Taiwan;Finland;Thailand;Spain;Austria;Switzerland;United Kingdom;Italy;France;Canada;Poland;Brazil;Malaysia;Australia;Denmark;Netherlands;Germany;Japan;Sweden;Korea, Republic of
2EUCTR2017-000665-79-GB
(EUCTR)
23/07/201827/04/2018A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who Completed Clinical Study ACH471-100An 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.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
12Phase 2New Zealand;Italy;United Kingdom;Korea, Republic of
3EUCTR2016-003526-16-GB
(EUCTR)
20/07/201823/04/2018A treatment study of ACH-0144471 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have poor Response to Eculizumab MonotherapyA 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
14Phase 2United States;Italy;United Kingdom
4NCT03472885
(ClinicalTrials.gov)
May 8, 201815/3/2018A Treatment Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Inadequate Response to EculizumabA Phase 2 Open-label Study of ACH-0144471 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have an Inadequate Response to Eculizumab MonotherapyParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ACH-0144471;Drug: EculizumabAlexion PharmaceuticalsAchillion, a wholly owned subsidiary of AlexionActive, not recruiting18 Years65 YearsAll12Phase 2United States
5EUCTR2016-002652-25-GB
(EUCTR)
02/02/201827/10/2017A 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.NULLNot Recruiting Female: yes
Male: yes
12Phase 2New Zealand;Italy;United Kingdom;Korea, Republic of
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03181633
(ClinicalTrials.gov)
June 22, 20175/6/2017A 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 ACH471-100Paroxysmal Nocturnal HemoglobinuriaDrug: ACH-0144471Alexion PharmaceuticalsNULLActive, not recruiting18 YearsN/AAll8Phase 2New Zealand
7NCT03053102
(ClinicalTrials.gov)
March 31, 20171/2/2017A 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-0144471Alexion PharmaceuticalsNULLCompleted18 YearsN/AAll10Phase 2Italy;Korea, Republic of;New Zealand;United Kingdom