a study to evaluate the safety and pharmacokinetics of single and multiple doses of prosetin in healthy volunteers and participants with als

Conditions: Amyotrophic Lateral Sclerosis

Summary: the primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with als.

pro-101 is a four-part study. parts a and b, which respectively evaluated the safety, tolerability, and pk of single and multiple ascending doses of prosetin in 48 healthy volunteers, have been completed. parts c and d, which are ongoing, will evaluate the effects of prosetin on safety, tolerability, pk, and biomarkers in 24 participants with als. part c is a double-blind, placebo-controlled, multiple ascending dose component of the study, and part d is an optional 52-week open-label extension available to als participants who complete 14 days of dosing in part c.

Eligibility criteria:
pro-101, parts a and b, were completed in healthy volunteers. pro-101, parts c and d are ongoing in participants with als. key eligibility criteria are summarized below: key inclusion criteria - part c * adults ≥18 years of age * diagnosis of als based on the gold coast diagnostic criteria * slow vital capacity (svc) \>50% predicted * if being concomitantly treated with riluzole and/or locally approved standard of care treatments, the participant must be on a stable dose for at least 30 days prior to screening and throughout the study * in the opinion of the investigator, participant is able to swallow liquid in order to ingest the study medication. key exclusion criteria - part c * active dementia, neurologic diseases other than als, or psychiatric illness that in the opinion of the investigator would affect participation in the current study. * significant history or clinical manifestation of comorbid disease in any organ system that currently requires active treatment or is likely to require treatment during the study. * any episodes of vertigo in the previous 12 months prior to screening. * any medical history of seizures, or any clinically significant eeg finding at screening or at day -1. * a diagnosis of cancer or evidence of continued disease within five years before screening. protocol-specified exceptions may be considered with approval from the sponsor's medical monitor. * participation in any other investigational study drug trial in which receipt of an investigational study drug occurred within 30 days prior to the first dose of study medication. * prior exposure to any stem cell or gene therapies (investigational or off-label) for the treatment of als. key inclusion criteria- part d participants who meet all of the following criteria may be included in part d of the study: * participants must have completed 14 days of blinded treatment in part c. * participants taking approved als standard-of-care medications must remain on stable doses through day 28 of open-label treatment. * in the judgment of the investigator, the participant's participation in the open-label portion of the study is medically appropriate key exclusion criteria- part d * treatment with any other investigational drug or device throughout the duration of the study is excluded, with the exception of any covid-19 vaccine or treatment with an emergency use authorization. note: other protocol-defined inclusion/exclusion criteria may apply. please contact trials@projenx.com with any questions about eligibility criteria.

Keywords: als amyotrophic lateral sclerosis map4k prosetin