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A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis | Clinical Trials | Clareo Health

NOT_YET_RECRUITINGPHASE1

A Phase I Study of Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

NCT07479953•Tippi Mackenzie

View on ClinicalTrials.gov

Summary

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

Eligibility

Age

All ages

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•3\. Live fetuses at 28 0/7 weeks to 35 6/7 weeks gestation 4. Diagnosis of Type I or Type II GM1 in utero by genetic analyses performed on amniotic fluid, fetal blood, placental tissue, or other samples through chorionic villus sampling (CVS), amniocentesis, or cordocentesis.
•1. In the event that parents are identified as genetic carriers of Type I or Type II GM1, diagnostic testing for the fetus would be performed to confirm the diagnosis
•2. If the fetal genetic testing confirms known mutations, parental genetic testing would not be necessary to enroll the fetus.
•4. The case must be reviewed and accepted by the enrollment advisory board (EAB) based on available clinical data (including age of onset and disease severity of affected family members), clinical presentation, literature review, available case studies, and available research assays (in addition to molecular testing as above).
•1. Pregnant women age 18 years or older, carrying a live fetus at 28 0/7 weeks to 35 6/7 weeks gestation
•2. Identified through the above listed means to be carrying a fetus with GM1 Type I or II
•3. Ability to give written informed consent oneself and comply with the requirements of the study
•5\. Maternal anti-AAV9 antibodies \<1:50. 6. Consents to fetal autopsy in the event of fetal demise

Exclusion Criteria

•1\. Fetuses with a concurrent severe structural anomaly, pathogenic genetic diagnosis, or other condition that presents a high risk of fetal mortality.
•While all possible congenital or structural anomalies that may be exclusionary cannot be listed, the following will be hard exclusions:
•Cardiac anomaly requiring neonatal surgical intervention
•Esophageal or bowel atresia
•Sacrococcygeal teratomas
•Chromosomal anomalies (e.g. trisomies)
•Other severe genetic conditions that would impact survival early in life (e.g. muscular dystrophy)
•Placental malformation that would impact safety of prenatal intervention (e.g. placental accreta)
•Examples of minor issues that would not be exclusionary include minor genetic or structural anomalies that can be readily treated and would not impact long-term survival, such as:
•Hearing loss that could be treated with hearing aids
+12 more criteria

Key Dates

Start Date

June 1, 2026

Primary Completion Date

June 1, 2038

Completion Date

June 1, 2055

Last Updated

March 18, 2026

Enrollment

ESTIMATED participants

Conditions

GM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2

Interventions

Gene Transfer with an AAV9 Vector Expressing Human ß-galactosidase

DRUG

Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis

NCT04713475

GM1 GangliosidosisGM1 Gangliosidosis, Type I+2 more

View study

TERMINATEDPHASE4

Synergistic Enteral Regimen for Treatment of the Gangliosidoses

NCT02030015

GM1 GangliosidosesGM2 Gangliosidoses+2 more

View study

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: March 18, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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