Mark Forman, MD, PhD, Chief Medical Officer at Passage Bio, discusses the global phase 1/2 upliFT-D clinical trial evaluating PBFT02, an adeno-associated virus (AAV) delivery gene therapy for patients with frontotemporal dementia (FTD) with granulin (GRN) mutations.
FTD is a disorder that affects the frontal and temporal lobes of the brain, areas that control personality, executive function, and language. FTD is a form of early onset dementia and currently has no approved disease-modifying therapies. In approximately 5–10% of individuals with FTD, the disease occurs because of mutations in the GRN gene. These mutations cause a deficiency of progranulin that helps regulate cellular processes.
Recently, Passage Bio announced that the first patient has been dosed in the global phase 1/2 upliFT-D clinical trial evaluating PBFT02. As Dr. Forman explains, PBFT02 is an AAV delivery gene therapy for the treatment of patients with FTD with GRN mutations. The upliFT-D trial is a dose-escalation study in which two doses will be sequentially evaluated in two cohorts, with a possible third cohort. Inclusion criteria for the trial include that patients:
- Be documented as a pathogenic GRN mutation carrier
- Have a clinical diagnosis of FTD
- Be between the ages of 35-75
- Have a reliable informant / caregiver who personally speaks with or sees the subject at least weekly
- Be living in the community (i.e., not in a nursing home; assisted living may be permitted at the discretion of the investigator).
Enrolment for the trial is currently ongoing. The primary endpoint of the study is to evaluate the safety and tolerability of PBFT02. Secondary endpoints include disease biomarkers and clinical outcome measures. upliFT-D is a two-year study with a three-year safety extension.
As Dr. Forman mentions, genetic testing for FTD with GRN mutations has been lacking. To enhance recruitment for the upliFT-D trial and increase genetic testing for this disease, Passage Bio has collaborated with InformedDNA to provide no-cost genetic counseling and testing for adults who have been diagnosed by their physicians with FTD. To learn more about this, visit informeddna.com/passagebio-ftd/
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