DDrare: Database of Drug Development for Rare Diseases

Searched query = "Aniridia"
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.

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	NCT04117880 (ClinicalTrials.gov)	December 31, 2018	19/9/2019	A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia	Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study	Aniridia	Drug: Ataluren	PTC Therapeutics	NULL	Withdrawn	2 Years	N/A	All	0	Phase 2	NULL
2	NCT02647359 (ClinicalTrials.gov)	January 31, 2016	29/12/2015	Study of Ataluren in Participants With Nonsense Mutation Aniridia	STAR: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia	Aniridia	Drug: Ataluren;Drug: Placebo	PTC Therapeutics	NULL	Active, not recruiting	2 Years	N/A	All	39	Phase 2	United States;Canada
3	JPRN-UMIN000002948	2013/04/01	29/12/2009	Cultivated epithelial sheet transplantation for corneal epithelial stem cell deficiency		a) Steven's Johnson Syndromeb) Ocular cicatricial pemphigoidc) Chemical/ Thermal Burns of the Cornead) Gelatinous Drop Like Dystrophye) Salzmann Corneal Degenerationf) Congenital Aniridia	Corneal epithelial sheet transplantation Cultivated eptihealial sheet transplantation will be perfored in the surgical department of Keio University Hospital. Simultaneous lamellar keratoplasty will be allowed. Surgical procedure involves removal of all pannus and abnormal epithelial tissue, the use of 0.04% mitomycin C to prevent fibrosis, and transplantation of the cultivated sheet.	Department of Ophthalmology, Keio University School of Medicine	NULL	Complete: follow-up complete	20years-old	Not applicable	Male and Female	5	Not selected	Japan

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

NCT04117880
(ClinicalTrials.gov)

December 31, 2018

19/9/2019

A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

Nonsense Mutation Aniridia: An Ataluren (PTC124) Phase 2 Extension Study

Aniridia

Drug: Ataluren

PTC Therapeutics

NULL

Withdrawn

2 Years

N/A

All

Phase 2

NULL

NCT02647359
(ClinicalTrials.gov)

January 31, 2016

29/12/2015

Study of Ataluren in Participants With Nonsense Mutation Aniridia

STAR: A Phase 2, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study of the Safety and Efficacy of Ataluren (PTC124) for the Treatment of Nonsense Mutation Aniridia

Aniridia

Drug: Ataluren;Drug: Placebo

PTC Therapeutics

NULL

Active, not recruiting

2 Years

N/A

All

Phase 2

United States;Canada

JPRN-UMIN000002948

2013/04/01

29/12/2009

Cultivated epithelial sheet transplantation for corneal epithelial stem cell deficiency

a) Steven's Johnson Syndromeb) Ocular cicatricial pemphigoidc) Chemical/ Thermal Burns of the Cornead) Gelatinous Drop Like Dystrophye) Salzmann Corneal Degenerationf) Congenital Aniridia

Corneal epithelial sheet transplantation
Cultivated eptihealial sheet transplantation will be perfored in the surgical department of Keio University Hospital. Simultaneous lamellar keratoplasty will be allowed.
Surgical procedure involves removal of all pannus and abnormal epithelial tissue, the use of 0.04% mitomycin C to prevent fibrosis, and transplantation of the cultivated sheet.

Department of Ophthalmology, Keio University School of Medicine

NULL

Complete: follow-up complete

20years-old

Not applicable

Male and Female

Not selected

Japan