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	EUCTR2016-004184-39-IT (EUCTR)	30/05/2017	06/02/2018	A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy.	AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY - A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067	Spinal Muscular Atrophy (SMA) MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]	Product Name: RO7034067 Product Code: RO7034067/F06 + solvente (RO Other descriptive name: RO7034067 Product Name: RO7034067 Product Code: RO7034067/F07 + solvente (RO Other descriptive name: RO7034067	F. HOFFMANN - LA ROCHE LTD.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	24	Phase 2	United States;United Kingdom;Switzerland;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

EUCTR2016-004184-39-IT
(EUCTR)

30/05/2017

06/02/2018

A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067 in adult and pediatric patients with spinal muscular atrophy.

AN OPEN-LABEL STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, AND PHARMACOKINETICS/PHARMACODYNAMICS OF RO7034067 IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY - A study to investigate the safety, tolerability, and pharmacokinetics/pharmacodynamics of RO7034067

Spinal Muscular Atrophy (SMA)
MedDRA version: 20.1;Level: PT;Classification code 10041582;Term: Spinal muscular atrophy;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Product Name: RO7034067
Product Code: RO7034067/F06 + solvente (RO
Other descriptive name: RO7034067
Product Name: RO7034067
Product Code: RO7034067/F07 + solvente (RO
Other descriptive name: RO7034067

F. HOFFMANN - LA ROCHE LTD.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

United States;United Kingdom;Switzerland;Italy