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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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years (multiple visits)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years (multiple visits)
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 moreImagine-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)
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Who is running the clinical trial?
Passage Bio, Inc.Lead Sponsor
5 Previous Clinical Trials
187,079 Total Patients Enrolled
Mark Forman, MDStudy DirectorPassage Bio, Inc.
1 Previous Clinical Trials
15 Total Patients Enrolled
David Weinstein, MD, MMScStudy DirectorPassage Bio, Inc.
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have nerve damage in my hands or feet.My heart muscle is weak (high troponin levels).I have not taken miglustat in the last 48 hours and will not take it during the study.My infant is 1-11 months old with early signs of GM1 gangliosidosis noted before 6 months.My child is 6-24 months old, showed GM1 gangliosidosis symptoms between 6-18 months, and can sit up alone.My kidney function is low, with an eGFR under 30 mL/min.I do not have any significant thinking or memory problems not caused by my condition.I haven't had a vaccine 14 days before or won't have one 30 days after dosing.I have needed a machine to help me breathe due to my condition.I have had severe seizures or epilepsy that required hospital care in the last 30 days.I have no health conditions that make the ICM procedure risky for me.You have a medical condition that makes it unsafe for you to have an MRI or lumbar puncture.I don't have health issues that would make certain medical procedures risky.I have undergone gene therapy before.Your blood takes longer than usual to clot.I haven't used enzyme therapy or experimental treatments recently.Your platelet count is less than 100,000 per microliter.Your liver function tests show high levels of certain enzymes or bilirubin.I have GM1 gangliosidosis confirmed by genetic testing and enzyme deficiency.
Research Study Groups:
This trial has the following groups:- Group 1: Part 1: Dose III of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01
- Group 2: Part 1: Dose I of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01
- Group 3: Part 1: Dose II of Dose Escalation Cohorts designed to identify the optimal dose of PBGM01
- Group 4: Part 2: Expansion Cohort designed to confirm the safety and efficacy of PBGM01
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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