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
Status: Recruiting
Location: See all (4) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:
• 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.
⁃ 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
Locations
United States
Massachusetts
Massachusetts General Hospital
RECRUITING
Boston
Texas
Worldwide Clinical Trials Early Phase Services
COMPLETED
San Antonio
Other Locations
Canada
The Neuro - Montréal Neurological Institute-Hospital
RECRUITING
Montreal
Netherlands
University Medical Center Utrecht
RECRUITING
Utrecht
Contact Information
Primary
ProJenX Clinical Trials
trials@projenx.com
(917) 423-6476
Time Frame
Start Date:2022-02-26
Estimated Completion Date:2026-10-31
Participants
Target number of participants:72
Treatments
Placebo_comparator: Part A - single dose of placebo
Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution.
Experimental: Part A - single ascending doses of prosetin
Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg.
Placebo_comparator: Part B - multiple doses of placebo
Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days.
Experimental: Part B - multiple ascending doses of prosetin
Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days.
Placebo_comparator: Part C - multiple doses of placebo in participants with ALS
Participants are administered a once-daily dose of prosetin-matched placebo for 14 days.
Experimental: Part C - multiple ascending doses of prosetin in participants with ALS
Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days.
Experimental: Part D - open-label administration of prosetin in participants with ALS
Participants will be administered a once-daily dose of prosetin for up to 52 weeks.