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Gene Therapy

Gene Therapy for Gangliosidosis (Imagine-1 Trial)

Phase 1 & 2
Waitlist Available
Research Sponsored by Passage Bio, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Early onset infantile (Type 1) must be ≥1 month and <12 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started before 6 months of age with specific minimum developmental milestones remaining.
Late onset infantile (Type 2a) must be ≥6 months and ≤24 months of age at enrollment and have signs and/or symptoms of GM1 gangliosidosis that started between 6 and 18 months of age with specific minimum developmental milestones remaining including the ability to sit independently at screening as defined by the WHO Multicenter Growth Reference Study (WHO-MGRS) criteria of being able to sit up unsupported with head erect for at least 10 seconds.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years (multiple visits)
Awards & highlights

Imagine-1 Trial Summary

This trial is testing a gene therapy for GM1 gangliosidosis, a disease that affects the nervous system. The goal is to see if it is safe and effective in patients with early or late onset infantile GM1 gangliosidosis.

Who is the study for?
This trial is for children with GM1 gangliosidosis, a genetic disorder. Infants (Type 1) must be 1-12 months old with symptoms starting before 6 months and meet certain developmental milestones. Older infants (Type 2a), aged 6-24 months, should show symptoms between 6-18 months and can sit independently. Participants cannot have had recent vaccines, gene therapy, or certain medical conditions that could affect the study.Check my eligibility
What is being tested?
The trial tests PBGM01, a gene therapy aimed at delivering a functional GLB1 gene to brain and peripheral tissues of kids with Type 1 or Type 2a GM1 gangliosidosis. It's designed in two parts to evaluate safety, tolerability and effectiveness of this treatment.See study design
What are the potential side effects?
While specific side effects are not listed here, potential risks may include reactions related to gene therapy such as immune response to the introduced genes or complications from delivery methods like injection into the spine.

Imagine-1 Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
My infant is 1-11 months old with early signs of GM1 gangliosidosis noted before 6 months.
Select...
My child is 6-24 months old, showed GM1 gangliosidosis symptoms between 6-18 months, and can sit up alone.
Select...
I have GM1 gangliosidosis confirmed by genetic testing and enzyme deficiency.

Imagine-1 Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years (multiple visits)
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years (multiple visits) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Change from Baseline in Developmental Milestones as Assessed by the Bayley Scale of Infant and Toddler Development, Third Edition
Number of Participants with Treatment Related AEs and SAEs as Characterized by CTCAEv5.0
Secondary outcome measures
Change from Baseline in Developmental Milestones as Assessed by the Vineland Adaptive Behavior Scale-II
Change in Baseline in Biomarkers of Beta-Galactosidase Activity in Blood and CSF
Change in Baseline in Biomarkers of Beta-Galactosidase Substrates in Blood and CSF
+4 more

Imagine-1 Trial Design

4Treatment groups
Experimental Treatment
Group I: Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01Experimental Treatment1 Intervention
Confirmatory Cohorts: Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1) Assigned Intervention: PBGM01 Single dose of PBGM01, via intra cisterna magna Dose to be determined
Group II: Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01Experimental Treatment1 Intervention
Assigned Intervention: PBGM01 Dose III: 2.2 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1)
Group III: Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01Experimental Treatment1 Intervention
Assigned Intervention: PBGM01 Dose II: 1.1 x 10^11 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1)
Group IV: Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01Experimental Treatment1 Intervention
Assigned Intervention: PBGM01 Dose I: 3.3 x 10^10 GC/g estimated brain weight, single dose of PBGM01 via intra cisterna magna in Late Onset Infantile GM1 Gangliosidosis (Type 2a) and Early Onset Infantile GM1 Gangliosidosis (Type 1)

Find a Location

Who is running the clinical trial?

Passage Bio, Inc.Lead Sponsor
5 Previous Clinical Trials
187,079 Total Patients Enrolled
David Weinstein, MD, MMScStudy DirectorPassage Bio, Inc.
Samiah Al-Zaidy, MDStudy DirectorPassage Bio, Inc.
1 Previous Clinical Trials
24 Total Patients Enrolled

Media Library

PBGM01 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04713475 — Phase 1 & 2
GM1 Gangliosidosis Research Study Groups: Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01, Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01
GM1 Gangliosidosis Clinical Trial 2023: PBGM01 Highlights & Side Effects. Trial Name: NCT04713475 — Phase 1 & 2
PBGM01 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04713475 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the current enrollment size of this study?

"To fulfill the requirement of this trial, 20 suitable participants must be recruited. Those interested in participating can do so from University of Minnesota at Minneapolis, British Columbia and BC Children's Hospital located in Vancouver or New jersey."

Answered by AI

Is this research accessible to persons aged 75 and older?

"According to the prerequisites of this medical trial, the appropriate age range for applicants is between 4 months and 3 years. 6 trials are available for those under 18years old, while 2 options exist for individuals over 65."

Answered by AI

Is this experiment currently accessible to participants?

"Confirming the details on clinicaltrials.gov, this medical study is still seeking participants since it was first presented to the public on March 17th 2021 and recently updated November 11th 2022."

Answered by AI

Am I eligible to partake in this clinical research project?

"This clinical trial seeks to recruit 20 patients between four and thirty-six months of age who present with gangliosidosis, GM1. To be eligible for inclusion in the study, they must demonstrate two mutations in their GLB1 gene as well as a laboratory deficiency of beta-galactosidase enzyme. Furthermore, those within the early onset infantile (Type 1) cohort should have had signs or symptoms before six months old while those in late onset infantile (Type 2a) group will manifest after 6–18 months old and both cohorts must still possess specific developmental milestones."

Answered by AI

How widely deployed is this trial across various locations?

"Presently, 7 medical centres are enrolling for this trial in locations such as Minneapolis, Vancouver and Toronto. To reduce travel demands, it is prudent to pick the closest site when signing up."

Answered by AI

What objectives does this research endeavor seek to fulfill?

"The primary aim of this medical trial, which will be followed over a 5 year period with multiple visits, is to ascertain the number of participants who display clinically noteworthy laboratory abnormalities as judged by hematology, chemistry and coagulation tests. Other objectives include gauging changes in quality of life using PedsQL-IS scales, estimating how much ventilator-free survival differs from natural history data and tracking any modifications to developmental milestones through Vineland Adaptive Behavior Scale-II examination."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis
2021. "Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04713475.
~9 spots leftby Feb 2026
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