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Gene Therapy
Gene Therapy for Frontotemporal Dementia (upliFT-D Trial)
Phase 1 & 2
Recruiting
Research Sponsored by Passage Bio, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Documented to be a pathogenic GRN mutation carrier
Clinical diagnosis of frontotemporal dementia
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years (multiple visits)
Awards & highlights
upliFT-D Trial Summary
This trial will test a gene therapy for frontotemporal dementia, which is a degenerative brain disease. The therapy involves delivering a functional copy of the GRN gene to the brain, and the trial will assess the safety, tolerability, and efficacy of the treatment.
Who is the study for?
This trial is for individuals with a clinical diagnosis of frontotemporal dementia who carry a specific pathogenic GRN mutation. They must have a caregiver, live outside of nursing homes (with some exceptions), and be able to give full consent. People with severe kidney issues, respiratory failure requiring oxygen, certain infections or recent vaccinations, untreated hypothyroidism or vitamin B12 deficiency, history of significant suicidal ideation or substance dependence, recent hospitalization due to acute illness, known mutations causing Alzheimer's disease, immunocompromised status or hypersensitivity to the study drug are excluded.Check my eligibility
What is being tested?
The trial is testing PBFT02 gene therapy designed to deliver a functional copy of the GRN gene directly into the brain. It aims to evaluate how safe and effective this treatment is for those affected by frontotemporal dementia due to progranulin gene mutations.See study design
What are the potential side effects?
Potential side effects may include reactions related to gene therapy such as immune responses against the introduced genes or delivery system components. There could also be complications from procedures required for administration like lumbar puncture and MRI.
upliFT-D Trial Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I carry a mutation in the GRN gene.
Select...
I have been diagnosed with frontotemporal dementia.
upliFT-D Trial Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 5 years (multiple visits)
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years (multiple visits)
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Assess Humoral Response Against the Vector and Transgene in CSF
Assess Humoral Response Against the Vector and Transgene in Serum
Change in Nerve Conduction Amplitude from Baseline on Nerve Conduction Studies
+2 moreSecondary outcome measures
Change from baseline in FTD clinical domains as assessed by the Clinical Dementia Rating National Alzheimer's Coordinating Center frontotemporal lobar degeneration domains (CDR Plus NACC FTLD)
Change from baseline in neurocognitive and other assessments
Change in Activities of Daily Living Scales
+7 moreupliFT-D Trial Design
3Treatment groups
Experimental Treatment
Group I: Optional Cohort 3Experimental Treatment1 Intervention
Drug: PBFT02 Dose 3: 2.2 x 10^11 GC/g* Single dose of PBFT02, via intra cisterna magna
*GC/g: gene copy per gram of estimated brain weight
Group II: Cohort 2Experimental Treatment1 Intervention
Drug: PBFT02 Dose 2: 1.1 x 10^11 GC/g* Single dose of PBFT02, via intra cisterna magna
*GC/g: gene copy per gram of estimated brain weight
Group III: Cohort 1Experimental Treatment1 Intervention
Drug: PBFT02 Dose 1: 3.3 x 10^10 GC/g* Single dose of PBFT02, via intra cisterna magna
*GC/g: gene copy per gram of estimated brain weight
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Who is running the clinical trial?
Passage Bio, Inc.Lead Sponsor
5 Previous Clinical Trials
187,090 Total Patients Enrolled
Mark Forman, MDStudy DirectorPassage Bio, Inc.
Gary Romano, MD, PhDStudy DirectorPassage Bio, Inc.
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I carry two copies of a GRN gene mutation.I have no health issues preventing me from undergoing the ICM procedure.I am allergic or cannot use corticosteroids due to health reasons.Your blood platelet count is higher than 100,000 per microliter.You have had a bad reaction to PBFT02 or similar medicines before.I carry a mutation in the GRN gene.I have a genetic mutation linked to Alzheimer's disease.You have a history of severe alcohol or substance dependence within 5 years of the start of memory loss, except if the increased alcohol use started when the memory loss began.You have untreated low thyroid hormone levels.I have been diagnosed with frontotemporal dementia.Your blood clotting test result (INR) should be less than 1.5.I have used anticoagulants recently or might need them during the study. Antiplatelet therapies are okay for me.I agree to use double-barrier contraception or abstain from sex for 90 days after my last dose.My kidney function is severely reduced.Your liver enzymes (ALT or AST) or bilirubin levels are higher than normal.I have had serious thoughts about suicide in the last 6 months.I have cancer other than skin cancer or a genetic cancer syndrome.I do not have any health conditions that could affect my thinking or memory.I have tested positive for HIV, HTLV-1/2, Hepatitis B/C, or tuberculosis within the last year.I need extra oxygen to breathe properly.My immune system is weakened.I don't have health issues that would make certain medical procedures risky.I have nerve damage in my hands or feet that affects my sense of touch.Your blood doesn't meet the specific requirements for the study's coagulation test.You have a score higher than 7 on the Rosen-modified Hachinski Ischemic Scale.I have a brain condition that explains my symptoms.I have not had gene therapy or treatments affecting PGRN levels without a proper washout period.I am not pregnant, willing to take pregnancy tests, and will use birth control or abstain from sex.I have never treated my vitamin B12 deficiency.My GRN mutation is not clearly harmful.Your blood takes less than 40 seconds to clot.You should not have any conditions that make it unsafe for you to have an MRI or lumbar puncture, such as a local infection or a history of blood clotting issues.I have a caregiver who checks on me at least once a week.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 1
- Group 2: Cohort 2
- Group 3: Optional Cohort 3
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What is the maximum number of people who can be enrolled in this clinical trial?
"The sponsor, Passage Bio, Inc., needs to enroll 6 individuals that fit the bill in order to commence this study. The clinical trial will take place at University of California at San Francisco and University of Toronto."
Answered by AI
In how many different medical facilities is this research project being conducted today?
"To make things more convenient for participants, this trial is enrolling at 5 sites which are situated in close proximity to large metropolitan areas. The cities with clinical trial sites include San Francisco, Toronto, New york and 5 others."
Answered by AI
Are we currently enrolling individuals for this experiment?
"The correct answer is that this trial, as indicated by information on clinicaltrials.gov, is actively looking for subjects. This study was first posted on September 14th 2021 and has since been updated April 26th of this year. They are expecting to have 6 patients total at 5 locations."
Answered by AI
Who would be an ideal candidate for this test group?
"This study is only for patients with semantic dementia that are between 35 and 75 years old. They will be one of six people taking part in the clinical trial."
Answered by AI
What are the aims of this research project?
"This trial's primary outcome is the number of participants with clinically significant laboratory abnormalities, which will be assessed over a period of approximately 5 years. Secondary outcomes include change in survival, change in concentration of biomarker of disease progression in CSF, and change in brain anatomy as assessed by MRI."
Answered by AI
Is this trial open to patients who are under the age of 80 years old?
"As this clinical trial's eligibility criteria state, the minimum age requirement for participation is 35 while the maximum is 75."
Answered by AI
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