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

Study acronym: Imagine-1
StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

See every version on ClinicalTrials.gov ↗

  1. Primary completion date: Feb 2026 to Feb 2028v24
  2. Lead sponsor: Passage Bio, Inc. to Gemma Biotherapeutics
    • Study sites: 9 to 7
    v23
  3. Re-verified, no change to tracked fieldsv22
  4. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised at 9 of 9 sites
    v21
  5. Site details revised at 1 of 9 sitesv20
Show 18 earlier versionsHide 18 earlier versions
  1. Site details revised at 4 of 9 sitesv19
  2. Trial status: Active, Not Recruiting to Recruiting
    • Site details revised at 9 of 9 sites
    v18
  3. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised at 9 of 9 sites
    v17
  4. Trial status: Active, Not Recruiting to Recruitingv16 Show 8 more changesShow less
    • Primary endpoints: 5 to 2
    • Enrollment target: 20 to 26
    • Eligibility criteria expanded, about 113 words
    • Secondary endpoints: 11 to 7
    • Study description shortened, about 61 words
    • Site details revised at 9 of 9 sites
    • Study title revised
    • Study arms: 2 to 4
  5. Trial status: Recruiting to Active, Not Recruiting
    • Site details revised at 9 of 9 sites
    v15
  6. Study sites: 10 to 9v14
  7. Site details revised at 1 of 10 sitesv13
  8. Site details revised at 1 of 10 sitesv12
  9. Site details revised at 1 of 10 sitesv11
  10. Re-verified, no change to tracked fieldsv10
  11. Study sites revised (10)v9
  12. Site details revised at 1 of 10 sitesv8
  13. Site details revised at 1 of 10 sitesv7
  14. Start date changed from estimate of Feb 2021 to Mar 17, 2021
    • Site details revised at 1 of 10 sites
    v6
  15. Trial status: Not Yet Recruiting to Recruiting
    • Site details revised at 10 of 10 sites
    v5
  16. Study sites: 9 to 10v4
  17. Start date: Jan 2021 to Feb 2021
    • Site contacts updated at 5 of 9 sites
    v3
  18. Study sites: 7 to 9
    • Eligibility criteria revised
    • Study description revised
    v2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis
Trial Details
NCT Number NCT04713475
Lead Sponsor Gemma Biotherapeutics
Conditions
(from sponsor)		 GM1 Gangliosidosis, GM1 Gangliosidosis, Type I, GM1 Gangliosidosis, Type 2, Beta-Galactosidase-1 (GLB1) Deficiency
Related terms Gangliosidosis, GM1, Lysosomal Storage Diseases, Rare Diseases
Enrollment 26 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov