A Study to Evaluate the Safety and Pharmacokinetics of Single and Multiple Doses of Prosetin in Healthy Volunteers and Participants With ALS
- Registration Number
- NCT05279755
- Lead Sponsor
- ProJenX
- Brief Summary
The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 72
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A - single dose of placebo placebo Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution. Part A - single ascending doses of prosetin prosetin Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg. Part B - multiple doses of placebo placebo Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days. Part B - multiple ascending doses of prosetin prosetin Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days. Part C - multiple doses of placebo in participants with ALS placebo Participants are administered a once-daily dose of prosetin-matched placebo for 14 days. Part C - multiple ascending doses of prosetin in participants with ALS prosetin Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days. Part D - open-label administration of prosetin in participants with ALS prosetin Participants will be administered a once-daily dose of prosetin for up to 52 weeks.
- Primary Outcome Measures
Name Time Method Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs) Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, D: Number of Participants with Clinically Significant Laboratory Test Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, D: Number of Participants with Clinically Significant Vital Signs Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, and D: Number of Participants with Clinically Significant Electrocardiogram (ECG) Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, and D: Number of Participants with Clinically Significant Physical Examination Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, and D: Number of Participants with Clinically Significant Neurological Examination Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts A, B, C, and D: Number of Participants with Clinically Significant Ophthalmic Examination Abnormalities Part A: Up to 28 days; Part B: Up to 42 days; Part C: Up to 28 days; Part D: Up to 54 weeks Parts C and D: Number of Participants with Clinically Significant Electroencephalogram (EEG) Abnormalities Part C: Up to 28 days; Part D: Up to 54 weeks
- Secondary Outcome Measures
Name Time Method Parts A, B, C, and D: Maximum Observed Concentration (Cmax) of Prosetin in Plasma Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks Parts A, B, C, and D: Time to Reach Maximum Observed Concentration (Tmax) of Prosetin in Plasma Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks Parts A, B, C, and D: Area Under the Concentration-Time Curve (AUC) of Prosetin in Plasma Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks Parts A, B, C, and D: Apparent Terminal Elimination Half-life (t1/2) of Prosetin in Plasma Part A: Up to 28 days Part B: Up to 42 days Part C: Up to 28 days Part D: Up to 54 weeks Parts A and D: Measure of Concentration of Prosetin in CSF Part A: Day 1; Part D: Up to 48 weeks
Trial Locations
- Locations (4)
Massachusetts General Hospital
🇺🇸Boston, Massachusetts, United States
The Neuro - Montréal Neurological Institute-Hospital
🇨🇦Montréal, Quebec, Canada
University Medical Center Utrecht
🇳🇱Utrecht, Netherlands
Worldwide Clinical Trials Early Phase Services
🇺🇸San Antonio, Texas, United States