Frontotemporal Dementia (FTD) upliFT-D Clinical Trial

Information for healthcare providers

About our clinical trial

Our investigational gene therapy product candidate, PBFT02, uses an adeno-associated virus serotype 1 (AAV1) viral vector to deliver a functional GRN gene, encoding progranulin (PGRN), to a patient’s cells. The vector is delivered, with CT guidance, directly to the cerebrospinal fluid by a single injection to the cisterna magna (ICM injection).^1-3

The goal of this vector and delivery approach is to provide elevated levels of PGRN to the central nervous system (CNS) to overcome the PGRN deficiency seen in GRN mutation carriers, and to increase PGRN levels in C9orf72 mutation carriers with FTD. upliFT-D will assess the safety, tolerability, and efficacy of this treatment in patients with FTD caused by GRN mutation (FTD-GRN) or C9orf72 mutations (FTD-C9orf72).^1-4

Understand more about an FTD diagnosis, Passage Bio clinical trial options, and the FTD patient/care partner community.

Download the FTD patient brochure

Gain insight into the disease progression of FTD, the role of progranulin, and the therapeutic potential of PBFT02.

Download the FTD brochure for healthcare providers

Inclusion criteria¹

Aged ≥35 and
≤75 years
Clinical diagnosis of early symptomatic FTD
Documented to have pathogenic GRN or C9orf72 mutations
Have a reliable informant/caregiver who personally speaks with or sees the patient at least weekly
Living in the community (ie, not in a nursing home); assisted living may be permitted at the discretion of the investigator

EXCLUSION criteria

Study design^1,3

upliFT-D is a Phase 1/2, global, multi-center, open-label, clinical trial of PBFT02 administered by a one-time ICM injection in patients with FTD-GRN or FTD-C9orf72. This trial will assess the safety, tolerability, and efficacy of PBFT02 in patients with either genetic form of FTD.¹

In patients with FTD-GRN, the upliFT-D clinical trial will enroll patients across 3 cohorts¹
In patients with FTD-C9orf72, the clinical trial will enroll patients across 2 cohorts¹
This is a 5-year study, with a 2-year main study, followed by a 3-year safety extension¹

FTD-GRN

COHORT 1

Dose 1
COHORT 2

Dose 1 or 2
COHORT 3

Dose 1 or 2

FTD-C9orf72

COHORT 4

Dose 2
COHORT 5

Dose 1 or 2

Completed
Independent Data Monitoring Committee (IDMC) review

Promising emerging data

Interim results from the trial demonstrated durable, elevated CSF PGRN levels and early evidence of reduction in plasma NfL levels, a disease progression biomarker, compared to published natural history data.

See the latest interim results

Genetic testing available at no cost

Genetic testing programs are available to help you confirm FTD-GRN or FTD-C9orf72.

Visit InformedDNA to refer your US-based patients to no-cost genetic testing and counseling*^† InformedDNA

*InformedDNA no-cost genetic testing and counseling are only available to US residents.

^†While the FTD sponsored testing programs are sponsored by Passage Bio, no personal identifying information of individuals participating in these genetic testing programs will be shared with the company.

Is your patient right for the upliFT-D trial?

Have you confirmed your patient has FTD-GRN
or FTD-C9orf72 via genetic testing?

Refer patient to a sponsored
genetic testing program.Did the results confirm FTD-GRN or FTD-C9orf72?

Refer patient to a trial
site location for further eligibility assessment

Patient is ineligible for
the upliFT-D trial

How to refer a patient to the upliFT-D trial

Referring a patient to the nearest trial site can be coordinated by contacting the trial team directly to assess qualified patients for further eligibility
Please note that all trial-related costs, including travel, will be covered by Passage Bio

For more inquiries on the upliFT-D trial, email
[email protected]
Click here for more information on active trial sites

References: 1. A study of PBFT02 in patients with frontotemporal dementia and progranulin mutations (FTD-GRN) (upliFT-D). ClinicalTrials.gov identifier: NCT04747431. Updated January 24, 2025. Accessed January 28, 2025. https://clinicaltrials.gov/study/NCT04747431 2. Perez BA, Shutterly A, Chan YK, Byrne BJ, Corti M. Management of neuroinflammatory responses to AAV-mediated gene therapies for neurodegenerative diseases. Brain Sci. 2020;10(2):119. doi:10.3390/brainsci10020119 3. Data on file. Passage Bio; Philadelphia, PA. 4. Anderson KM, McNeil E, Weinstein DA, Romano G. Gene therapy for treatment of monogenic neurodegenerative diseases. Neurol Reviews. 2021;(suppl):S1-S7.

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