NCT05898620View on ClinicalTrials.gov

A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

14

Participants

Timeline

Start Date

June 13, 2023

Primary Completion Date

October 31, 2029

Study Completion Date

October 31, 2029

Conditions
Rett Syndrome
Interventions
GENETIC

NGN-401

NGN-401 is a non-replicating, recombinant AAV9 carrying a full length human MECP2 transgene.

Trial Locations (8)

10467

RECRUITING

Montefiore Medical Center, New York

60612

RECRUITING

Rush University Medical Center, Chicago

77030

RECRUITING

Texas Children's Hospital, Houston

80045

RECRUITING

Children's Hospital Colorado, Aurora

02115

RECRUITING

Boston Children's Hospital, Boston

Unknown

ACTIVE_NOT_RECRUITING

The Children's Hospital at Westmead, Sydney

EH16 4TJ

RECRUITING

Royal Hospital for Children and Young People, Edinburgh

M13 9WL

RECRUITING

Manchester University NHS Foundation Trust, Manchester

Sponsors

Lead Sponsor

Neurogene Inc.
All Listed Sponsors
lead

Neurogene Inc.

INDUSTRY

NCT05898620 - A Novel, Regulated Gene Therapy (NGN-401) Study for Females With Rett Syndrome | Biotech Hunter | Biotech Hunter