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Gene Therapy

Gene Therapy for Frontotemporal Dementia (ASPIRE-FTD Trial)

Phase 1 & 2
Recruiting
Research Sponsored by AviadoBio Ltd
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Male or female, 30 to 75 years of age
Carriers of a pathogenic GRN mutation
Must not have
Previous treatment with any gene or cell therapy
Clinically significant abnormality on MRI at Screening considered to be a contraindication to Intrathalamic infusion
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 26 week initial, 5-year total follow-up period
Awards & highlights
No Placebo-Only Group

Summary

"This trial aims to test a new gene therapy product called AVB-101 for treating Frontotemporal Dementia with Progranulin Mutations (FTD-GRN). FTD-GR

Who is the study for?
This trial is for adults aged 30-75 with Frontotemporal Dementia (FTD) due to a specific genetic mutation (GRN). Participants must have certain symptoms, be able to consent, and have a study partner. They need enough thalamic volume in the brain and should commit to the study's procedures and schedule.
What is being tested?
The trial tests AVB-101, a gene therapy delivered once directly into the brain. It aims to see if this treatment is safe for FTD-GRN patients, can restore normal protein levels associated with dementia, and potentially halt or slow disease progression.
What are the potential side effects?
Since AVB-101 is an investigational gene therapy product being tested for safety, potential side effects are not fully known but may include reactions at the injection site or immune responses.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I am between 30 and 75 years old.
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I carry a harmful GRN gene mutation.
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I have been diagnosed with possible bvFTD or PPA.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I have not had gene or cell therapy before.
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My MRI shows I can't have treatments directly into my brain.
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My MRI shows brain shrinkage that affects surgery plans.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~26 week initial, 5-year total follow-up period
This trial's timeline: 3 weeks for screening, Varies for treatment, and 26 week initial, 5-year total follow-up period for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Change from baseline in brain structure
Change from baseline in the Mini-Mental State Examination (MMSE)
Incidence of treatment emergent suicidal ideation or behavior
+2 more
Secondary study objectives
Change from baseline in AAV9 immunogenicity in CSF
Change from baseline in AAV9 immunogenicity in blood
Change from baseline in CDR + NACC FTLD-SB score
+9 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Group I: Cohort 2 (dose 2)Experimental Treatment2 Interventions
Escalated dose, delivered as a one-time only, intrathalamic administration.
Group II: Cohort 1 (dose 1)Experimental Treatment2 Interventions
Initial dose, delivered as a one-time only, intrathalamic administration.

Find a Location

Who is running the clinical trial?

AviadoBio LtdLead Sponsor
~4 spots leftby Apr 2026
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