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A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗
Trial flagged as At Risk
Primary completion moved at least 2 years later Jan 1, 2028 → Jan 1, 2030
See other at-risk trials from National Human Genome Research Institute (NHGRI)

Record History

The 50 most recent of 189 record versions are accounted for below. See every version on ClinicalTrials.gov ↗

  1. Interventions: AAV9-GLB1 (Biological), Abdominal ultrasound (Procedure), Audiology assessment with ABR (Diagnostic Test), Bone density scan (DEXA)… to AAV9-GLB1 (Biological), Abdominal ultrasound (Procedure), Audiology assessment with ABR (Diagnostic Test), Bone density scan (DEXA)… Eligibility criteria expanded, about 10 words · Secondary endpoints: details revised at 2 of 7 entries · Study description shortened, about 30 words Show 3 more changesShow less
    • Eligibility criteria expanded, about 10 words
    • Secondary endpoints: details revised at 2 of 7 entries
    • Study description shortened, about 30 words
  2. Re-verified, no change to tracked fieldsv188
  3. Primary and study completion pushed from Jan 1, 2028 to Jan 1, 2030v187
  4. 6 versions Re-verified since Aug 2, 2025, no change to tracked fieldsv181-v186
  5. Enrollment target: 45 to 54 Secondary endpoints: 6 to 7 · Study description revised · Eligibility criteria expanded, about 1 wordv180 Show 3 more changesShow less
    • Secondary endpoints: 6 to 7
    • Study description revised
    • Eligibility criteria expanded, about 1 word

    See this version in full on ClinicalTrials.gov ↗

Show 40 earlier versionsHide 40 earlier versions
  1. Eligibility criteria revised Study description expanded, about 11 wordsv179 Show 1 more changeShow less
    • Study description expanded, about 11 words

    See this version in full on ClinicalTrials.gov ↗

  2. 7 versions Re-verified since Jun 11, 2024, no change to tracked fieldsv172-v178
  3. Trial status: Active, Not Recruiting to Recruiting Enrollment target: 17 to 45 · Site details revisedv171 Show 2 more changesShow less
    • Enrollment target: 17 to 45
    • Site details revised
  4. Enrollment target: 16 to 17v170
  5. Re-verified, no change to tracked fieldsv169
  6. Study Status, Study Description, Eligibilityv168
  7. 2 versions Re-verified since Mar 16, 2023, no change to tracked fieldsv166-v167
  8. Primary completion date: Apr 1, 2024 to Jan 1, 2028 Trial status: Recruiting to Active, Not Recruiting · Enrollment target: estimated 45 to confirmed 16 · Completion date: Apr 30, 2026 to Jan 1, 2028v165 Show 3 more changesShow less
    • Trial status: Recruiting to Active, Not Recruiting
    • Enrollment target: estimated 45 to confirmed 16
    • Completion date: Apr 30, 2026 to Jan 1, 2028
  9. 2 versions Re-verified since Jan 14, 2023, no change to tracked fieldsv163-v164
  10. Primary completion date: Dec 31, 2027 to Apr 1, 2024 Completion date: Dec 31, 2027 to Apr 30, 2026v162 Show 1 more changeShow less
    • Completion date: Dec 31, 2027 to Apr 30, 2026
  11. Interventions: AAV9-GLB1 (Biological), Audiology assessmentwith ABR (Diagnostic Test), Bone density scan (DEXA (Diagnostic Test), Brain MRI/MRS/fMRI… to AAV9-GLB1 (Biological), Abdominal ultrasound (Procedure), Audiology assessment with ABR (Diagnostic Test), Bone density scan (DEXA)…v161
  12. 21 versions Re-verified since Jun 2, 2022, no change to tracked fieldsv140-v160
  • 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.
Background: GM1 gangliosidosis is a disorder that destroys nerve cells. It is fatal. There is no treatment. People with GM1 are deficient in a certain enzyme. A gene therapy may help the body make this enzyme. This could improve GM1 symptoms. Objective: To test if a gene therapy helps Type I and Type II GM1 gangliosidosis symptoms. Eligibility: Type I subjects will be male and female \>= 6 months \<= 12 months of age at the time of full ICF signing. Type II subjects will be male and female \> 12 months old and \< 12 years old at the time of full ICF signing. Design: Participants will be screened with their medical history and a phone survey. Participants will stay at NIH for 8-10 weeks. Participants will have baseline tests: Blood, urine, and heart tests Hearing tests Ultrasound of abdomen EEG: Sticky patches on the participant s head will measure brain function. Lumbar puncture: A needle will be stuck into the participant s spine to remove fluid. MRI scans, bone x-rays, and bone scans: Participants will lie in a machine that takes pictures of the body IQ tests Neurology exams Central line placement Skin biopsy: A small piece of the participant s skin will be removed. Speech tests Participants will have an x-ray while swallowing food. Participants will take drugs by mouth and IV. This will get their immune system ready for therapy. Participants will get the gene therapy by IV. They may stay at NIH for a week to watch for side effects. Participants will have visits 3 and 6 months after treatment. Then visits will be every 6 months for 2 years. Then they will have a visit at 3 years. Visits will take 4-5 days. Participants will return to NIH once a year for 2 years for tests in an extension study....
Trial Details
NCT Number NCT03952637
Lead Sponsor National Human Genome Research Institute (NHGRI)
Collaborators: Sio Gene Therapies
Conditions Lysosomal Diseases, Gangliosidosis, GM1
Enrollment 54 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov