Clinical Trials

A clinical trial is a research study conducted in humans with the goal of answering specific questions about new therapies, vaccines, diagnostic procedures, or new ways of using known treatments. Clinical trials are used to determine whether new drugs, diagnostics, or treatments are both safe and effective. Clinical trials are critically important for developing cures for conditions like FTD.

PROCLAIM Genetic Therapy Trial (Phase I/II)

Title: A PHASE 1/2 ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND EFFECTS ON PROGRANULIN LEVELS OF LY3884963 IN PATIENTS WITH FRONTO-TEMPORAL DEMENTIA WITH PROGRANULIN MUTATIONS (FTD-GRN) (sponsor: Prevail Therapeutics).

This clinical trial research study is being conducted to assess the safety, tolerability, and effects of an investigational genetic therapy product called LY3884963 on progranulin protein (PGRN) levels in blood and cerebrospinal fluid (CSF).

Description

LY3884963 will be administered as a single dose via injection into the fluid area at the base of the skull called the cisterna magna by a trained radiologist. The procedure will be performed with the patient under general anesthesia and using imaging guidance. This will involve a 2-night inpatient stay. Patients will be given medications to reduce any risk of inflammation before and after the injection procedure. Participation in this study will last for 5 years. The first year is more involved with a higher number of visits to monitor safety and tolerability (Dosing, Day 7, Day 14, Day 21, Month 1, Month 1.5, Month 2, Month 3, Month 6, Month 9, Year 1). Years 2-5 will involve a visit every 6 months to assess the effects of long-term exposure to LY3884963 on safety and efficacy outcomes. If you agree to join the study, you will be asked to complete the following research procedures at various time points over the duration of the study.

Eligibility

Symptomatic individuals who are confirmed carriers of a GRN mutation causative of FTD.

Study Activities

A one-time dose of LY3884963 injected into the fluid at the base of the skull by an interventional neuro-radiologist (one-time procedure) with a two-night inpatient stay
Magnetic Resonance Imaging (MRI) / Magnetic Resonance Angiography (MRA)
Lumbar Punctures (LP)
Electrocardiograms (ECG)
Vital signs (blood pressure, heart rate, etc.)
Urine tests for safety and drug screen
Viral shedding tests (urine, saliva, feces)
Physical and neurological examinations
Blood tests for safety and PGRN levels
Cognitive testing
Questionnaires about mood and everyday activities (for both patient and study partner)

For more information, please reach out to our clinical trials team:

Dahlia Kamel, Clinical Research Program and Operations Lead

Office: 215-662-6134, Cell: 919-302-1117

kamel.dahlia@pennmedicine.upenn.edu

Cara Joyce, Clinical Trials Coordinator

Office: 215-662-3205, Cell: 610-955-3124

cara.joyce@pennmedicine.upenn.edu

Quinn Hlava, Clinical Trials Coordinator

Office: 215-662-6162, Cell: 678-978-1415

quinn.hlava@pennmedicine.upenn.edu

Alex Tavani, Clinical Trials Coordinator

Office: 215-662-2060, Cell: 610-930-8224

alexandra.tavani@pennmedicine.upenn.edu

Anusha Patgiri, Study Team Coordinator

Office: 215-622-6122, Cell: 240-449-9334

anusha.patgiri@pennmedicine.upenn.edu

upliFT-D Genetic Therapy Trial (Phase I)

Title: 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 CISTERNA MAGNA (ICM) OF ADULT SUBJECTS WITH FRONTOTEMPORAL DEMENTIA (FTD) AND MUTATIONS IN THE PROGRANULIN GENE (GRN) (sponsor: Passage Bio).

This clinical trial research study is being conducted to assess the safety, tolerability, and effects of an investigational genetic therapy product called PBFT02 on progranulin protein (PGRN) levels in blood and cerebrospinal fluid (CSF).

Description

PBFT02 will be administered as a single dose via injection into the fluid area at the base of the skull called the cisterna magna by a trained radiologist. The procedure will be performed with the patient under general anesthesia and using imaging guidance. This will involve a 3-night inpatient stay. Patients will be given medications to reduce any risk of inflammation before and after the injection procedure. Participation in this study will last for 5 years. The first year is more involved with a higher number of visits to monitor safety and tolerability (Dosing, Day 7, Day 14, Day 21, Month 1, Month 2, Month 3, Month 6, Month 12). Years 2-5 will follow a more variable schedule of visits, ranging from 6 to 12 months apart, to assess the effects of long-term exposure to PBFT02 on safety and efficacy outcomes. If you agree to join the study, you will be asked to complete the following research procedures at various time points over the duration of the study.

Eligibility

Symptomatic individuals who are confirmed carriers of a GRN mutation causative of FTD.

Study Activities

A one-time dose of PBFT02 injected into the fluid at the base of the skull by an interventional neuro-radiologist (one-time dose procedure) with a three-night inpatient stay
Magnetic Resonance Imaging (MRI) / Magnetic Resonance Angiography (MRA)
Lumbar Punctures (LP)
Electrocardiograms (ECG)
Optical Coherence Tomography (OCT)
Nerve Conduction Study (NCS)
Vital signs (blood pressure, heart rate, etc.)
Urine tests for safety and drug screen
Viral shedding tests (urine, saliva, feces)
Physical and neurological examinations
Blood tests for safety and PGRN levels
Cognitive testing
Questionnaires about mood and everyday activities (for both patient and study partner)

Veri-T-001 Trial (Phase I)

Title: VERI-T: A PHASE 1, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND PRELIMINARY EFFICACY STUDY OF ORAL VERDIPERSTAT (BHV-3241) IN PATIENTS WITH SEMANTIC VARIANT PRIMARY PROGRESSIVE APHASIA (SVPPA) DUE TO FRONTOTEMPORAL LOBAR DEGENERATION WITH TDP-43 PATHOLOGY (FTLD-TDP) (sponsor: NIH).

This clinical trial research study is being conducted to assess the safety, tolerability, and effects of an investigational drug called Verdiperstat (also known as BHV-3241) on individuals with semantic variant primary progressive aphasia (svPPA) likely due to Frontotemporal Lobar Degeneration with TDP-43 Pathology (FTLD-TDP).

Description

Verdiperstat will be given as an oral tablet, taken daily. This is a placebo-controlled study, meaning that some participants will receive the study drug and some participants will receive placebo (tablets that contains no active Verdiperstat drug). Participation in this study will last for about 8 months, including a 24-week study drug treatment period with visits occurring every 4 weeks. The study will test the effects of Verdiperstat on your cerebrospinal fluid (CSF), blood proteins, brain magnetic resonance imaging (MRI), and cognitive function. If you agree to join the study, you will be asked to complete the following research procedures at various time points over the duration of the study.

Eligibility

Individuals with a clinical diagnosis of semantic variant primary progressive aphasia (svPPA).

Study Activities

Oral study drug administration (daily for 24 weeks)
Magnetic Resonance Imaging (MRI)
Lumbar Punctures (LP)
Electrocardiograms (ECG)
Vital signs (blood pressure, heart rate, etc.)
Urine tests for safety
Physical and neurological examinations
Blood tests for safety and biomarker levels
Cognitive testing
Questionnaires about mood and everyday activities (for both patient and study partner)

Clinical Trials

PROCLAIM Genetic Therapy Trial (Phase I/II)

​Title: A PHASE 1/2 ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND EFFECTS ON PROGRANULIN LEVELS OF LY3884963 IN PATIENTS WITH FRONTO-TEMPORAL DEMENTIA WITH PROGRANULIN MUTATIONS (FTD-GRN) (sponsor: Prevail Therapeutics).

This clinical trial research study is being conducted to assess the safety, tolerability, and effects of an investigational genetic therapy product called LY3884963 on progranulin protein (PGRN) levels in blood and cerebrospinal fluid (CSF).

Eligibility ​​ Symptomatic individuals who are confirmed carriers of a GRN mutation causative of FTD.

Study Activities ​​

A one-time dose of LY3884963 injected into the fluid at the base of the skull by an interventional neuro-radiologist (one-time procedure) with a two-night inpatient stay

Magnetic Resonance Imaging (MRI) / Magnetic Resonance Angiography (MRA)

Lumbar Punctures (LP)

Electrocardiograms (ECG)

Vital signs (blood pressure, heart rate, etc.)

Urine tests for safety and drug screen

Viral shedding tests (urine, saliva, feces)

Physical and neurological examinations

Blood tests for safety and PGRN levels

Cognitive testing

Questionnaires about mood and everyday activities (for both patient and study partner)

Subscribe to the Penn FTD Center Newsletter:

Title: A PHASE 1/2 ASCENDING DOSE STUDY TO EVALUATE THE SAFETY AND EFFECTS ON PROGRANULIN LEVELS OF LY3884963 IN PATIENTS WITH FRONTO-TEMPORAL DEMENTIA WITH PROGRANULIN MUTATIONS (FTD-GRN) (sponsor: Prevail Therapeutics).

Eligibility

Symptomatic individuals who are confirmed carriers of a GRN mutation causative of FTD.

Study Activities