Clinical Trials

ELIGIBILITY: Symptomatic or pre-symptomatic individuals who are confirmed carriers of a GRN mutation causative of FTD

BRIEF DESCRIPTION: This clinical trial research study is being conducted to test the safety and tolerability (i.e., possible side effects) of an investigational drug called AL001, and what effects this drug has on progranulin protein (PGRN) levels in blood. The participation will last for about 96 weeks and involves a monthly visit to Penn. This is a placebo-controlled trial, so some participants will receive a water infusion without the active ingredient. Neither the study team nor the participant will know who is receiving the drug or the placebo. This is followed by a 2-year Open label extension, where all participants will receive the drug. 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: 

STUDY ACTIVITIES:
•       IV Drug or Placebo Infusion (Once every 28 days) 
•       Magnetic Resonance Imaging (MRI) 
•       Optional Lumbar Punctures (LP) 
•       Electrocardiogram (ECG) 
•       Vital signs (blood pressure, heart rate, etc.) 
•       Urine test for safety and drug screen 
•       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)

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

BRIEF DESCRIPTION: This clinical trial research study is being conducted to assess the safety and tolerability of an investigational genetic therapy product called PR006A on progranulin protein (PGRN) levels in blood and cerebrospinal fluid (CSF). PR006A will be administered as a single dose injection into the fluid area around 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 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 PR006A 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: 

STUDY ACTIVITIES
•       A one-time dose of PR006A 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) 
•       Electrocardiogram (ECG) 
•       Vital signs (blood pressure, heart rate, etc.) 
•       Urine test for safety and drug screen 
•       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)

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

BRIEF DESCRIPTION: This clinical trial research study is being conducted to test the safety and tolerability of an investigational gene therapy called PBFT02 on progranulin (PGRN) protein levels in plasma and cerebrospinal fluid (CSF). PBFT02 will be administered as a single dose via injection into 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 involves 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. If you agree to join the study, these are the research procedures which occur at various time points over the duration of the study: 
 

STUDY ACTIVITIES:
•       A one-time dose of PBFT02 to the fluid area at the base of the skull injected 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) 
•       Electrocardiogram (ECG) 
•       Optical Coherence Tomography (OCT) 
•       Nerve Conduction Study (NCS) 
•       Vital signs (blood pressure, heart rate, etc.) 
•       Urine test for safety and drug screen 
•       Viral shedding tests (urine, saliva, feces) 
•       Physical and neurological examinations 
•       Blood tests for safety and PGRN levels 
•       Neurocognitive testing 
•       Questionnaires about mood and everyday activities (for both patient and study partner)

For information about clinical trials (interventional research studies) at the Penn FTD Center, please reach out to our clinical trials team: