Davunetide (AL-108) in Predicted Tauopathies - Pilot Study

What Was Tested

The trial (phase 1) is built around Davunetide.

The primary objective of the study is to obtain preliminary safety and tolerability data with davunetide (NAP, AL-108) in patients with a tauopathy (frontotemporal lobar degeneration \[FTLD\] with predicted tau pathology, corticobasal degeneration syndrome \[CBS\] or progressive supranuclear palsy \[PSP\]).

The secondary objectives of this study are to obtain preliminary data on short term changes (at 12 weeks) in a variety of clinical, functional and biomarker measurements from baseline, including cerebrospinal fluid (CSF) tau levels, eye movements, and brain MRI measurements.

It had two arms (groups): davunetide (Al-108, NAP) nasal spray (experimental) and Placebo nasal spray (placebo comparator).

The registry classes the interventions as drug.

Who Took Part

12 actual participants took part.

It was open to people of any sex aged from 40 years up to 85 years.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: A probable tauopathy defined as:. Probable or possible progressive supranuclear palsy (PSP) defined as:. At least a 12-month history of:. Postural instability or falls during the first 3 years that symptoms are present and. Age at symptom onset ≥ 40 years by history; and.

The full record lists 36 inclusion and 31 exclusion criteria; among the exclusions: insufficient fluency in local language to complete neuropsychological and functional assessments; A diagnosis of Amyotrophic Lateral Sclerosis or other motor neuron disease; any of the following:.

How the Study Was Run

It was run as a randomized, parallel-group and quadruple-blind study.

Because people in this Davunetide study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Neither the participants nor the staff measuring them knew who was on Davunetide, so expectations could not easily colour the readings.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2010-01 with completion listed for 2012-12.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Researchers chose this as the main measure: Safety evaluations will be performed by recording clinical adverse events at each study visit. Clinical laboratory, ECGs, physical examinations will be conducted (time frame: 12 weeks).

It also tracked 12 secondary outcomes, for example PSP Rating Scale, Clinician's Global Impression (CGI-ds), Schwab and England Activities of Daily Living scale (SEADL), MRI brain ventricular volume, Repeatable Battery for the Assessment of Neuropsychological Status (RBANS) and Unified Parkinson's Disease Rating Scale (UPDRS).

What This Means in Plain Language

Put simply, this is a phase 1, randomized trial of Davunetide in 12 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Davunetide compound page, all human trials.