A Phase 1b Open-Label, Multicenter, Dose-Escalation Study to Assess the Safety, Tolerability, and Pharmacodynamic Effects of a Single Dose of PBFT02 Delivered Into the ICM of Adults With FTD and Mutations in the GRN or C9ORF72 Genes
Who is this study for? Adults with frontotemporal dementia and mutations in the progranulin gene
What treatments are being studied? PBFT02
Status: Recruiting
Location: See all (7) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Eligibility
Participation Requirements
Sex: All
Minimum Age: 35
Maximum Age: 75
Healthy Volunteers: f
View:
• 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
Locations
United States
Pennsylvania
University of Pennsylvania
RECRUITING
Philadelphia
Texas
University of Texas at Houston
RECRUITING
Houston
Other Locations
Brazil
Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)
RECRUITING
Minas Gerais
Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo (HCFMUSP)
RECRUITING
São Paulo
Canada
Montreal Neurological Institute-Hospital
RECRUITING
Montreal
University of Toronto, Toronto Western Hospital
RECRUITING
Toronto
Portugal
Centro Hospitalar e Universitário de Coimbra
RECRUITING
Coimbra
Contact Information
Primary
Patient/Family Inquiries
patientservices@passagebio.com
267-866-0113
Backup
Physician Inquiries
medinfo@passagebio.com
Time Frame
Start Date: 2021-09-14
Estimated Completion Date: 2031-08
Participants
Target number of participants: 25
Treatments
Experimental: Cohort 1
Drug: PBFT02 Dose 1; Single dose of PBFT02, via intra cisterna magna~\*GC/g: gene copy per gram of estimated brain weight
Experimental: Cohort 2, 3, 4 and 5
Drug: PBFT02 Dose 1 or 2; Single dose of PBFT02, via intra cisterna magna~\*GC/g: gene copy per gram of estimated brain weight
Related Therapeutic Areas
Sponsors
Leads: Passage Bio, Inc.