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	2

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	EUCTR2020-000501-93-AT (EUCTR)	27/11/2020	08/09/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)	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: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride Product Name: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;Austria;South Africa;United Kingdom
2	EUCTR2020-000501-93-GB (EUCTR)	29/09/2020	09/07/2020	A long term study of BCX9930 in patients with PNH	A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)	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: BCX9930 Product Code: BCX9930 INN or Proposed INN: BCX9930 Other descriptive name: BCX9930 hydrochloride	BioCryst Pharmaceuticals Inc	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	200	Phase 2	European Union;South Africa;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

EUCTR2020-000501-93-AT
(EUCTR)

27/11/2020

08/09/2020

A long term study of BCX9930 in patients with PNH

A Phase 2, Open-Label Study to Evaluate the Long-term Safety of Oral BCX9930 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)

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: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride
Product Name: BCX9930
Product Code: BCX9930
INN or Proposed INN: BCX9930
Other descriptive name: BCX9930 hydrochloride

BioCryst Pharmaceuticals Inc

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

200

Phase 2

European Union;Austria;South Africa;United Kingdom

EUCTR2020-000501-93-GB
(EUCTR)

29/09/2020

09/07/2020