Trials / Recruiting
RecruitingNCT04747431
A Study of PBFT02 in Participants With FTD and Mutations in the Granulin Precursor (GRN) or C9ORF72 Genes
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
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 30 (estimated)
- Sponsor
- Passage Bio, Inc. · Industry
- Sex
- All
- Age
- 35 Years – 75 Years
- Healthy volunteers
- Not accepted
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
Detailed 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 participants. This is a 5-year study, with a 2-year main study, followed by a 3-year safety extension.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | PBFT02 | PBFT02 |
Timeline
- Start date
- 2021-09-14
- Primary completion
- 2028-08-01
- Completion
- 2031-08-01
- First posted
- 2021-02-10
- Last updated
- 2025-12-31
Locations
10 sites across 5 countries: United States, Australia, Brazil, Canada, Portugal
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT04747431. Inclusion in this directory is not an endorsement.