MedTrace Logo

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

NCT07479953 · Status: NOT_YET_RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2026-04-13

No results posted yet for this study

Summary

This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.

Conditions

  • GM1 Gangliosidoses
  • GM1 Gangliosidosis, Type I
  • GM1 Gangliosidosis, Type 2

Interventions

DRUG

Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase

Prenatal administration of an AAV9 Vector Expressing Human ß-galactosidase

Sponsors & Collaborators

  • Tippi Mackenzie

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-06-01
Primary Completion
2038-06-01
Completion
2055-06-01
FDA Drug
Yes

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07479953 on ClinicalTrials.gov