Actively Recruiting
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Led by Passage Bio, Inc. · Updated on 2025-12-31
30
Participants Needed
10
Research Sites
515 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Documented pathogenic carrier of GRN or C9orf72 mutation
- Clinical diagnosis of frontotemporal dementia
- Have a reliable informant or caregiver who personally sees or speaks with the participant at least weekly
- Living in the community; assisted living may be allowed at investigator's discretion
You will not qualify if you...
- GRN mutation classified as not pathogenic, likely benign, benign, or unclear; C9orf72 repeat length = 30 (specific to cohorts)
- Prior treatment with any gene therapy or therapies affecting progranulin levels without washout
- Homozygous GRN or C9orf72 mutation carrier
- Rosen-modified Hachinski Ischemic Scale score > 7
- Structural brain lesion explaining symptoms
- Known Alzheimer's disease-causing mutation
- History of Korsakoff encephalopathy or severe substance dependence within 5 years
- Untreated vitamin B12 deficiency
- Untreated hypothyroidism
- Severe kidney impairment (eGFR �b1 30 ml/min)
- Elevated liver enzymes or bilirubin
- Respiratory failure needing supplemental oxygen or ventilation
- Inability to provide consent or lack of authorized caregiver
- Contraindications to MRI, lumbar puncture, or cisterna magna injection
- Medical or laboratory conditions increasing risk from procedures
- Immunocompromised status
- Peripheral axonal sensory neuropathy
- Recent vaccine within 14 days
- Positive tests for HIV, HTLV, Hepatitis B/C, or tuberculosis
- Malignant neoplasia or hereditary cancer syndrome
- Other diseases causing cognitive impairment unrelated to GRN or C9orf72
- Recent suicidal ideation
- Positive pregnancy test or unwillingness to use birth control (women)
- Breastfeeding women
- Unwillingness to use double-barrier contraception or abstinence (men)
- Any condition increasing risk or interfering with study
- Acute illness requiring hospitalization within 30 days
- Coagulation test failures
- Recent use of anticoagulants
- Hypersensitivity to corticosteroids or PBFT02
- Increased risk of thrombosis
- Hypersensitivity to apixaban
- Additional criteria for FTD-C9orf72 cohorts related to ALS severity and treatments
AI-Screening
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Trial Site Locations
Total: 10 locations
1
Michigan Alzheimer's Disease Center
Ann Arbor, Michigan, United States, 48105
Actively Recruiting
2
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
3
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232
Actively Recruiting
4
University of Texas at Houston
Houston, Texas, United States, 77030
Actively Recruiting
5
Eastern Health-Box Hill Hospital
Melbourne, Victoria, Australia, 3128
Actively Recruiting
6
Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
Minas Gerais, Brazil
Actively Recruiting
7
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
São Paulo, Brazil
Actively Recruiting
8
University of Toronto, Toronto Western Hospital
Toronto, Ontario, Canada, M5T 2S8
Actively Recruiting
9
Montreal Neurological Institute-Hospital
Montreal, Quebec, Canada, H3A 2B4
Actively Recruiting
10
Centro Hospitalar e Universitário de Coimbra
Coimbra, Portugal
Actively Recruiting
Research Team
P
Patient/Family Inquiries
CONTACT
P
Physician Inquiries
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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