A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF… (NCT04747431) | Clinical Trial Compass
RecruitingPhase 1/2
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
United States, Australia30 participantsStarted 2021-09-14
Plain-language summary
PBFT02 is a gene therapy for frontotemporal dementia intended to deliver a functional copy of the GRN gene to the brain. This study will assess the safety, tolerability and efficacy of this treatment in patients with frontotemporal dementia and mutations in the granulin precursor (GRN) or chromosome 9 open reading frame 72 (C9ORF72) genes
Who can participate
Age range35 Years – 75 Years
SexALL
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Inclusion criteria
✓. Documented to be a pathogenic carrier of GRN or C9orf72 mutation
✓. Clinical diagnosis of frontotemporal dementia
✓. Have a reliable informant / caregiver (and back-up informant / caregiver) who personally speaks with or sees the subject at least weekly
✓. Living in the community (i.e., not in a nursing home); assisted living may be permitted at the discretion of the investigator
Exclusion criteria
✕. Classification of the GRN mutation as "not pathogenic," "likely benign variant," "benign variant," or "pathogenic nature unclear" (FTD- GRN Cohorts 1-3) or C9orf72 HRE length ≤ 30 (FTD-C9orf72 Cohorts 4-5).
✕. Previous treatment with any gene therapy. Any other therapies with the potential to alter PGRN levels must be washed out for at least 5 half-lives prior to entry into this study
✕. Known presence of a structural brain lesion (eg, tumor, cortical infarct) that could reasonably explain symptoms in a symptomatic subject
✕. Known presence of an AD-causing mutation in PSEN1, PSEN2 or APP based on genetic testing history (if performed)
✕. Previous history of Korsakoff encephalopathy, severe alcohol or substance dependence (within 5 years of onset of dementia), except where onset of increased alcohol consumption occurs at the time of FTD disease onset
What they're measuring
1
Number of Participants with Treatment-Related AEs and SAEs
Timeframe: Up to 5 years (multiple visits)
2
Change in Nerve Conduction Velocity and Amplitude from Baseline on Nerve Conduction Studies
Timeframe: From baseline to 5 years (multiple visits)
3
Change in Cellular and Humoral Response Against the Vector and Transgene in Serum
Timeframe: From baseline to 5 years (multiple visits)