DDrare: Database of Drug Development for Rare Diseases

Searched query = "Distal myopathy", "Distal muscular dystrophy", "Miyoshi myopathy", "Distal dysferlinopathy", "DMRV/GNE myopathy", "Oculopharyngodistal myopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = ACENEURAMIC ACID; Aceneuramic Acid; Aceneuramic Acid Extended-Release; Aceneuramic Acid Extended-Release Tablets; Aceneuramic acid; Aceneuramic acid extended-release (Ace-ER); Common Name: Sialic Acid; Deflazacort; INN: aceneuramic acid; N-acetylneuraminic acid; NPC-09; SA; Sialic acid; Sialic acid (INN: aceneuramic acid); Sialic acid, N-acetylneuraminic acid; UX001

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	NCT00527228 (ClinicalTrials.gov)	September 2003	6/9/2007	Deflazacort in Dysferlinopathies	Deflazacort in Dysferlinopathies (LGMD2B/MM) - a Double Blind, Placebo-controlled Clinical Study	LGMD2B;Miyoshi Myopathy;Dysferlinopathy	Drug: deflazacort;Drug: placebo	Ludwig-Maximilians - University of Munich	NULL	Completed	18 Years	N/A	Both	25	Phase 2;Phase 3	Germany

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

NCT00527228
(ClinicalTrials.gov)

September 2003

6/9/2007

Deflazacort in Dysferlinopathies

Deflazacort in Dysferlinopathies (LGMD2B/MM) - a Double Blind, Placebo-controlled Clinical Study

LGMD2B;Miyoshi Myopathy;Dysferlinopathy

Drug: deflazacort;Drug: placebo

Ludwig-Maximilians - University of Munich

NULL

Completed

18 Years

N/A

Both

Phase 2;Phase 3

Germany