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
67	多発性嚢胞腎	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	EUCTR2012-000550-60-AT (EUCTR)	17/01/2014	27/11/2013	Pulsed oral sirolimus in autosomal dominant polycystic kidney disease	Pulsed oral sirolimus in autosomal dominant polycystic kidney disease - The Vienna RAP Study	Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and uncontrolled proliferation of innumerable epithelial-lined cysts that stem from renal tubular cells, which compress and/or destroy vital renal tissue with a gradual decline in renal function, and terminal kidney failure with the need for renal reaplacement therapy. As yet, other than supportive care there is no viable therapy. MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Trade Name: Rapamune 1mg tablets Product Name: Rapamune 1mg tablets INN or Proposed INN: SIROLIMUS	Medizinische Universität Wien, Klinische Abteilung für Nephrologie und Dialyse, Universitätsklinik für Innere Medizin 3	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	68	Phase 3	Austria

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

EUCTR2012-000550-60-AT
(EUCTR)

17/01/2014

27/11/2013

Pulsed oral sirolimus in autosomal dominant polycystic kidney disease

Pulsed oral sirolimus in autosomal dominant polycystic kidney disease - The Vienna RAP Study

Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and uncontrolled proliferation of innumerable epithelial-lined cysts that stem from renal tubular cells, which compress and/or destroy vital renal tissue with a gradual decline in renal function, and terminal kidney failure with the need for renal reaplacement therapy. As yet, other than supportive care there is no viable therapy.
MedDRA version: 20.0;Level: LLT;Classification code 10036046;Term: Polycystic kidney, autosomal dominant;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trade Name: Rapamune 1mg tablets
Product Name: Rapamune 1mg tablets
INN or Proposed INN: SIROLIMUS

Medizinische Universität Wien, Klinische Abteilung für Nephrologie und Dialyse, Universitätsklinik für Innere Medizin 3

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

Austria