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
3	脊髄性筋萎縮症	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	EUCTR2018-004383-65-NL (EUCTR)	30/10/2019	03/04/2019	A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy	Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study	Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850 MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Product Name: SRK-015 INN or Proposed INN: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY	Scholar Rock, Inc.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	55	Phase 2	United States;Canada;Spain;Germany;Netherlands;Italy

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

EUCTR2018-004383-65-NL
(EUCTR)

30/10/2019

03/04/2019

A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy

Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study

Later Onset Spinal Muscular Atrophy (SMA)
MedDRA version: 20.0;Level: LLT;Classification code 10079415;Term: Spinal muscular atrophy type III;System Organ Class: 100000004850
MedDRA version: 20.0;Classification code 10079416;Term: Spinal muscular atrophy type II;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY
Product Name: SRK-015
INN or Proposed INN: SRK-015
Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY

Scholar Rock, Inc.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;Canada;Spain;Germany;Netherlands;Italy