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A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
NCT04747431 · View on ClinicalTrials.gov ↗
Study 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
Conditions Studied
Interventions
- DRUG PBFT02
Study Locations (10)
Other
- Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG) — Minas Gerais
- Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP) — São Paulo
- Centro Hospitalar e Universitário de Coimbra — Coimbra
Michigan
- Michigan Alzheimer's Disease Center — Ann Arbor
Pennsylvania
- University of Pennsylvania — Philadelphia
Tennessee
- Vanderbilt University Medical Center — Nashville
Texas
- University of Texas at Houston — Houston
Victoria
- Eastern Health-Box Hill Hospital — Melbourne
Ontario
- University of Toronto, Toronto Western Hospital — Toronto
Quebec
- Montreal Neurological Institute-Hospital — Montreal
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 30 participants |
| Start Date | 2021-09-14 |
| Est. Completion | 2031-08 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04747431
The ClinicalTrials.gov registry entry for NCT04747431 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 30 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Passage Bio, which has 1 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 5 conditions, with Frontotemporal Dementia appearing as the primary indexed condition, and to 1 intervention — of which PBFT02 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04747431 reports 10 study locations spanning 8 distinct geographic areas — top geographies include Other, Michigan, Pennsylvania. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04747431 about?
NCT04747431 is a clinical study titled "A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes". 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...
What is the current status of trial NCT04747431?
This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 30 participants. The study started on 2021-09-14. Estimated completion is 2031-08.
What conditions does trial NCT04747431 study?
This clinical trial studies the following conditions: Frontotemporal Dementia, FTD, FTD-GRN, C9orf72, Dementia Frontotemporal. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04747431?
The interventions under investigation include: PBFT02 (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04747431?
This trial is sponsored by Passage Bio, which has 1 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04747431 being conducted?
This trial has 10 study locations across Michigan, Pennsylvania, Tennessee, Texas, Victoria. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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