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 frontotemporal degeneration (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 may be eligible to participate in this study. More extensive eligibility criteria is reviewed during pre-screening and screening for this trial.

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:

Quinn Hlava, Senior Clinical Trials Coordinator

Office: 215-662-6162

Cell: 267-283-5267

quinn.hlava@pennmedicine.upenn.edu

Alex Tavani, Clinical Trials Coordinator

Office: 215-662-2060

Cell: 267-253-4856

alexandra.tavani@pennmedicine.upenn.edu

Riya Chaturvedi, Clinical Trials Coordinator

Riya.chaturvedi@pennmedicine.upenn.edu

For more information about the clinical research program at the Penn FTD Center, please reach out to our Clinical Research Program and Operations Lead, Assistant Director, Dahlia Kamel, via email at kamel.dahlia@pennmedicine.upenn.edu or via phone at 267-275-3948.

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 PARTICIPANTS WITH FRONTOTEMPORAL DEMENTIA (FTD) AND MUTATIONS IN THE GRANULIN PRECURSOR (GRN) OR CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) GENES (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 or c9orf72 mutation causative of FTD may be eligible to participate in this study. More extensive eligibility criteria is reviewed during pre-screening and screening for this trial.

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: Peter Ljubenkov, MD).

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) may be eligible to participate in this study. More extensive eligibility criteria is reviewed during pre-screening and screening for this trial.

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)

DNLI-H-0001 (Phase I/II)

Title: A Phase 1/2, Multicenter, Randomized, Placebo-Controlled, Double-Blind Single Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of DNL593 in Healthy Participants and Participants with Frontotemporal Dementia Followed by an Open-Label Extension (sponsor: Denali Therapeutics Inc).

This clinical trial research study is being conducted to assess the safety, tolerability, and effects of protein-replacement therapy using an investigational product called DNL593 in participants with frontotemporal dementia caused by a GRN mutation (FTD-GRN).

Description

DNL593 will be administered as an IV infusion approximately every 4 weeks. This is a placebo-controlled study, meaning that some participants will receive the study drug, and some participants will receive a placebo (contains no active study drug). There are 13 visits total over the course of approximately 6 months (24 weeks), with an optional open label extension period. This study will test the effects of DNL593 on cerebrospinal fluid (CSF), blood proteins, brain magnetic resonance imaging (MRI), and cognitive function.

Eligibility

Participants of nonchildbearing potential with FTD-GRN, aged ≥ 18 to ≤ 80 years who have a reliable study partner may be eligible to participate in this study. More extensive eligibility criteria is reviewed during pre-screening and screening for this trial.

Study Activities

Monthly IV Infusion of DNL593 or placebo
Magnetic Resonance Imaging (MRI)
Lumbar Punctures (LP)
Electrocardiograms (ECG)
Vital signs (blood pressure, heart rate, etc.)
Urine tests for safety and drug screening
Neurological and Physical Examinations
Cognitive Tests
Blood tests (including tests for HIV and Hepatitis)
Questionnaires about mood (including thoughts on suicide), behavior, and everyday activities

CRVO24-NFD-701 (Phase IIa)

Title: A Phase 2a Clinical Study of the P38 Alpha Kinase Inhibitor Neflamapimod in Patients with Primary Progressive Aphasia (PPA) (Sponsor: CervoMed Inc.)

This clinical trial research study is being conducted to assess the safey and effects of an investigational drug called Neflamapimod on individuals with nonfluent variant primary progressive aphasis (nfvPPA).

Description

Neflamapimod will be given as an oral capsule, taken three times a day. The first 24 weeks of this study will be the open-label portion, where all participants receive the study drug. After 24 weeks of open-label treatment, the study will continue to a randomized, double-blind period, meaning that some participants will continue to receive the study drug and some will receive a placebo (capsules that contain no active Neflamapimod drug) for 12 weeks. Participation in this study will last for about 10 months, with visits occurring every 6 weeks. The study will test the effects of Neflamapimod on cognitive function, blood proteins, and PET imaging (optional). 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 nonfluent variant primary progressive aphasia (nfvPPA).

Study Activities

Oral study drug administration (three times a day for 36 weeks)
Electrocardiograms (ECG)
Vital signs (blood pressure, heart rate, etc.)
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)
Optional PET imaging

Clinical Trials

CRVO24-NFD-701 (Phase IIa)

​Title: A Phase 2a Clinical Study of the P38 Alpha Kinase Inhibitor Neflamapimod in Patients with Primary Progressive Aphasia (PPA) (Sponsor: CervoMed Inc.)

This clinical trial research study is being conducted to assess the safey and effects of an investigational drug called Neflamapimod on individuals with nonfluent variant primary progressive aphasis (nfvPPA).

Eligibility

Study Activities ​​

Subscribe to the Penn FTD Center Newsletter:

Title: A Phase 2a Clinical Study of the P38 Alpha Kinase Inhibitor Neflamapimod in Patients with Primary Progressive Aphasia (PPA) (Sponsor: CervoMed Inc.)

Study Activities