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
Passage Bio, Inc.
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
Description
PBFT02 is an adeno-associated viral vector serotype 1 carrying GRN, the gene encoding for human progranulin, formulated as a solution for injection into the cisterna magna. This is a global interventional, multicenter, open-label, single-arm study of PBFT02 delivered as a one-time dose administered into the cisterna magna to participants with FTD-GRN or C9orf72. Participants aged ≥ 35 and ≤ 75 years with early symptomatic FTD-GRN or with symptomatic FTD-C9orf72 may be enrolled into the study. PBFT02 will be studied in three cohorts of FTD-GRN participants and two cohorts of FTD-C9orf72 partic…
Eligibility
- Age range
- 35–75 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Documented to be a pathogenic carrier of GRN or C9orf72 mutation 2. Clinical diagnosis of frontotemporal dementia 3. Have a reliable informant / caregiver (and back-up informant / caregiver) who personally speaks with or sees the subject at least weekly 4. Living in the community (i.e., not in a nursing home); assisted living may be permitted at the discretion of the investigator Exclusion Criteria: 1. 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…
Interventions
- DrugPBFT02
PBFT02
Locations (10)
- Michigan Alzheimer's Disease CenterAnn Arbor, Michigan
- University of PennsylvaniaPhiladelphia, Pennsylvania
- Vanderbilt University Medical CenterNashville, Tennessee
- University of Texas at HoustonHouston, Texas
- Eastern Health-Box Hill HospitalMelbourne, Victoria
- Hospital das Clinicas da Universidade Federal de Minas Gerais (UFMG)Minas Gerais