Evaluating prosetin for safety and pharmacokinetics in ALS patients and healthy volunteers

A Phase 1, Randomized, Double-Blind, Placebo-Controlled Dose Escalating Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending and Multiple Ascending Doses of Prosetin in Healthy Volunteers and Participants With Amyotrophic Lateral Sclerosis (ALS) With an Optional Open-Label Extended Treatment Period for ALS Participants Who Complete 14 Days of Blinded Treatment

This study is testing the safety and effects of a new drug called prosetin in people with ALS and healthy volunteers to see how it works in both groups.

Quick facts

• Phase: Phase 1
• Study type: Interventional
• Enrollment: 72 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: ProJenX (Industry-sponsored)
• Locations: 4 sites (Boston, Massachusetts and 3 other locations)
• Trial ID: NCT05279755 on ClinicalTrials.gov

What this trial studies

This study aims to assess the safety, tolerability, and pharmacokinetics of prosetin in both healthy volunteers and participants diagnosed with Amyotrophic Lateral Sclerosis (ALS). It consists of four parts, with the first two parts completed in healthy individuals, while the ongoing parts focus on ALS patients. Part C is a double-blind, placebo-controlled component that will evaluate the effects of prosetin on safety and biomarkers, and Part D offers an optional 52-week open-label extension for those who complete Part C. The study is designed to gather critical data on the drug's effects in a vulnerable population.

Who should consider this trial

Good fit: Ideal candidates for this study are adults aged 18 and older with a confirmed diagnosis of ALS and a Slow Vital Capacity greater than 50% predicted.

Not a fit: Patients with active dementia, other neurological diseases, or significant comorbid conditions that require active treatment may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to a new treatment option for patients with ALS, potentially improving their quality of life.

How similar studies have performed: While this approach is novel in the context of ALS, similar studies evaluating pharmacokinetics and safety of new treatments have shown promise in other conditions.

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 [e-mail] with any questions about eligibility criteria.

Where this trial is running

Boston, Massachusetts and 3 other locations

• Massachusetts General Hospital — Boston, Massachusetts, United States (Recruiting)
• Worldwide Clinical Trials Early Phase Services — San Antonio, Texas, United States (Completed)
• The Neuro - Montréal Neurological Institute-Hospital — Montréal, Quebec, Canada (Recruiting)
• University Medical Center Utrecht — Utrecht, Netherlands (Recruiting)

Study contacts

• Study coordinator: ProJenX Clinical Trials

Conditions Amyotrophic Lateral Sclerosis; ALS; Amyotrophic lateral sclerosis; MAP4K; prosetin