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
28	全身性アミロイドーシス	1

告示番号

疾患名（ページ内リンク）

臨床試験数

全身性アミロイドーシス

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	NCT02627820 (ClinicalTrials.gov)	January 2016	19/11/2015	The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart	An 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis)	Amyloidosis	Drug: Isis 420915/GSK 299872	Brigham and Women's Hospital	GlaxoSmithKline;Ionis Pharmaceuticals, Inc.	Withdrawn	50 Years	90 Years	Both	0	Phase 2	NULL

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

NCT02627820
(ClinicalTrials.gov)

January 2016

19/11/2015

The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart

An 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis)

Amyloidosis

Drug: Isis 420915/GSK 299872

Brigham and Women's Hospital

GlaxoSmithKline;Ionis Pharmaceuticals, Inc.

Withdrawn

50 Years

90 Years

Both

Phase 2

NULL