PROCLAIM

Phase 1/2 clinical trial of LY3884963 in patients with frontotemporal dementia with progranulin mutations

Conditions

Frontotemporal dementia

Sponsor

Prevail Therapeutics

Clinical Trial

Phase II

Status

Currently recruiting

Drug

LY3884963

Countries

Belgium, France, United Kingdom

Information about the drug that will be tested in the study

Administration

The drug will be administered via an intra-cisternal injection (insertion of a needle into the cisterna magna, a large space located at the base of the brain).

Is the drug already on the market for another medical condition?

Will all participants receive the same drug?

Participants will be selected by chance to receive one of the following options:

An intra-cisternal injection of LY3884963 (low or medium dose)
An intravenous injection of methylprednisolone every 2 weeks in the first 3 months (corticosteroid used to treat inflammation or immune reactions).ct)

If needed and at the clinical team discretion, participants may receive optional oral tablets of sirolimus (a medicine that reduces the activity of the immune system) and prednisone (corticosteroid used to decrease inflammation).

Information about participating in the trial

What are the researchers trying to find out?

The purpose of the study is to evaluate the safety and effects on progranulin levels of LY3884963 in people with frontotemporal dementia with progranulin mutations.

How long will the treatment last?

5 years
During the first year, participants will be evaluated for the effect of the investigational drug. Participants will be followed up for an additional 4 years

What your involvement will entail?

During the study, participants will be asked to complete some laboratory/biological tests (i.e. blood) to evaluate the emergent adverse effects (unfavourable signs, symptoms or diseases temporally associated with the use of the drug tested in the study)
Participants will be asked to perform lumbar punctures (CSF) and brain scans (MRI)
Participants will complete tests that will cognition, global function and memory (CDR, NACC FTLD).

Further information on the procedures, tests and number of visits can be obtained from the study team.

Who can participate in this study?

Who can participate in the study?

To take part in the study, participants must:

Be between 30 and 85 years old
Have a body weight range of ≥40 kg to ≤110 kg
Have symptomatic frontotemporal dementia
Have a score between 0.5 and 15 on the test CDR plus NACC FTLD sum of boxes
Carry a pathogenic progranulin gene (GRN) mutation
Have a study partner who has a sufficient contact with the participant and is willing to participate in study procedures throughout the study duration
Not be dependent on a walker or wheelchair.

Who cannot participate in the study?

People cannot take part in the study if they have or have experienced:

Diagnosis of a significant central nervous system disease other than frontotemporal dementia
Any disease or condition that could, in the opinion of the investigator, interfere with the safety, tolerability and/or study assessments
Clinically significant laboratory test result abnormalities
Participation within 3 months prior to screening in another therapeutic investigational drug or device study
History of cancer
Contraindications or intolerance to brains scans (e.g., claustrophobia)
Contraindications to general anaesthesia or deep sedation
Positive urine test for drugs of abuse.

The above list is not exhaustive. It includes the most common conditions and diseases that might exclude people from the study.