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	NCT00528268 (ClinicalTrials.gov)	July 2007	10/9/2007	Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy	Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy	Spinal Muscular Atrophy	Drug: Sodium phenylbutyrate (NaPB)	University of Utah	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)	Completed	N/A	6 Months	All	14	Phase 1;Phase 2	United States

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

July 2007

10/9/2007

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

Spinal Muscular Atrophy

Drug: Sodium phenylbutyrate (NaPB)

University of Utah

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Completed

N/A

6 Months

All

Phase 1;Phase 2

United States