A Study to Evaluate PR006 in Patients with Frontotemporal Dementia with Progranulin Mutations (FTD-GRN)


About this study

The purpose of this study is to 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. 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

  • Men or women aged 30 to 80 years (inclusive), at the time of informed consent.
  • Body weight range of ≥ 40 kg (88 lb) to ≤ 110 kg (242 lb).
  • BMI of 18 to 34 kg/m^2.
  • 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.
  • Women of non-childbearing potential must be either surgically sterile or postmenopausal.
  • Men and women of childbearing potential must use a highly effective method of contraception for the duration of the study.
  • Men must agree to abstain from sperm donation for the duration of the study.
  • Women must agree to abstain from egg donation for the duration of the study.
  • Women of childbearing potential cannot be pregnant or lactating/breastfeeding, and must have a negative serum pregnancy test at screening.
  • Patient is not dependent on 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.

Exclusion Criteria:

  • 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.
  • Contraindications to corticosteroid use or uncontrolled hypertension.
  • 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 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.
  • Positive urine test for drugs of abuse (including opiates, benzodiazepines, amphetamines, cocaine, barbiturates and phencyclidine) without prescription at screening and day -1.
  • Other protocol-defined inclusion/exclusion criteria may apply.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location Status Contact

Jacksonville, Fla.

Mayo Clinic principal investigator

Neill Graff Radford, M.D.

Contact us for the latest status

Contact information:

Heather Cissel


More information


Publications are currently not available

Study Results Summary

Not yet available

Supplemental Study Information

Not yet available


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