for people ages 30-80 (full criteria)
at San Francisco, California and other locations
study started
estimated completion



Study PRV-FTD101 is a Phase 1/2, multi-center, open-label ascending dose, first-in-human study that will evaluate the safety and effect of intra-cisternal PR006 administration on progranulin protein (PGRN) levels in patients with frontotemporal dementia with progranulin mutations (FTD-GRN). Three escalating dose (low dose, medium dose and high dose) cohorts are planned. The duration of the study is 5 years. During the first year, patients with be evaluated for the effect of PR006 on safety, tolerability, immunogenicity, biomarkers, and efficacy. Patients will follow up for an additional 4 years to monitor safety and changes on selected biomarkers and clinical outcomes.

Official Title

A Phase 1/2 Ascending Dose Study to Evaluate the Safety and Effects on Progranulin Levels of PR006A in Patients With Fronto-Temporal Dementia With Progranulin Mutations (FTD-GRN)


Frontotemporal Dementia Fronto-Temporal Dementia Progranulin Mutations FTD-GRN Gene Therapy Dementia Gene Therapy AAV9 Dementia Aphasia, Primary Progressive Pick Disease of the Brain Sirolimus Prednisone Methylprednisolone Methylprednisolone Acetate Methylprednisolone Hemisuccinate Prednisolone Prednisolone acetate Prednisolone hemisuccinate Prednisolone phosphate


You can join if…

Open to people ages 30-80

  • Body weight range of ≥40 kg (88 lbs) to ≤110 kg (242 lb) and a BMI of 18 to 34 kg/m2.
  • Has symptomatic frontotemporal dementia (FTD) per investigator assessment.
  • Stable use of background medications at least 8 weeks prior to investigational product dosing.
  • Carrier of a pathogenic GRN (progranulin gene) mutation.
  • Negative screening test for Mycobacterium tuberculosis (MTB) or documented negative MTB test within 1 year prior to screening.
  • Age- and gender-appropriate cancer screenings are up-to-date.
  • Patient and/or patient's legally authorized representative has the ability to understand the purpose and risks of the study, and provide written informed consent and authorization to use protected health information.
  • Patient has a reliable study partner/informant (e.g. family member, friend) willing and able to participate in the study as a source of information on the patient's health status and cognitive and functional abilities..
  • Patient is not dependent on a walker or wheelchair.
  • Patient is living in the community (i.e. not in nursing home); some levels of assisted living may be permitted at the discretion of the investigator.
  • Pneumococcal pneumonia and shingles vaccines are required within 10 years of screening (allowed to be performed during screening but must be given at least 4 weeks prior to administration of immunosuppressant at the start of the study).

You CAN'T join if...

  • Diagnosis of a significant CNS (central nervous system) disease other than frontotemporal dementia (FTD) that may cause FTD symptoms or confound study objectives.
  • Brain magnetic resonance image (MRI) / magnetic resonance angiography (MRA) showing clinically significant abnormality considered to prevent intracisternal injection.
  • Hypersensitivity or contraindications to corticosteroid and/or sirolimus use.
  • Clinical evidence of peripheral symmetric sensory polyneuropathy (stable sensory mononeuropathies and radiculopathies are not exclusionary).
  • Concomitant disease or condition within 6 months of screening that could interfere with, or treatment of which might interfere with, the conduct of the study or that would, in the opinion of the investigator, pose an unacceptable safety risk to the patient or interfere with the patient's ability to comply with study procedures
  • Clinically significant laboratory test result abnormalities assessed at screening.
  • Participation within 3 months prior to screening in another therapeutic investigational drug or device study with purported disease-modifying effects on FTD, unless it can be documented that the patient received placebo only.
  • Any type of prior gene or cell therapy.
  • Immunizations (live vaccines) in the 4 weeks prior to screening. Pneumococcal vaccine and shingles vaccine administration is allowed during screening.
  • Use of blood thinners in the 2 weeks prior to screening, or anticipated use of blood thinners during the study. Antiplatelet therapies may be acceptable.
  • Contraindications or intolerance to imaging methods (MRI, CT) and intolerance to contrast agents.
  • Contraindications to general anesthesia or deep sedation.

Other protocol-defined inclusion/exclusion criteria may apply


  • UCSF Memory and Aging Center, 350 Parnassus Ave, Suite 706 not yet accepting patients
    San Francisco California 94117 United States
  • Mayo Clinic, 4500 San Pablo Road, not yet accepting patients
    Jacksonville Florida 32224 United States


accepting new patients
Start Date
Completion Date
Prevail Therapeutics
Phase 1/2
Study Type
Last Updated