DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
62	発作性夜間ヘモグロビン尿症	1

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	NCT04170023 (ClinicalTrials.gov)	December 16, 2019	6/11/2019	Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)	A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and With an Approved C5 Inhibitor as Background Therapy	Paroxysmal Nocturnal Hemoglobinuria (PNH)	Drug: ALXN2050;Drug: C5 inhibitor background therapy	Alexion Pharmaceuticals	NULL	Recruiting	18 Years	N/A	All	26	Phase 2	United States;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

NCT04170023
(ClinicalTrials.gov)

December 16, 2019

6/11/2019

Proof of Concept Study of ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH)

A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and With an Approved C5 Inhibitor as Background Therapy

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Drug: ALXN2050;Drug: C5 inhibitor background therapy

Alexion Pharmaceuticals

NULL

Recruiting

18 Years

N/A

All

Phase 2

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