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
Overview
- Phase
- Phase 1
- Intervention
- placebo
- Conditions
- Amyotrophic Lateral Sclerosis
- Sponsor
- ProJenX
- Enrollment
- 72
- Locations
- 4
- Primary Endpoint
- Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Status
- Recruiting
- Last Updated
- last year
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Part A - single dose of placebo
Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution.
Intervention: placebo
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.
Intervention: prosetin
Part B - multiple doses of placebo
Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days.
Intervention: placebo
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.
Intervention: prosetin
Part C - multiple doses of placebo in participants with ALS
Participants are administered a once-daily dose of prosetin-matched placebo for 14 days.
Intervention: placebo
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.
Intervention: prosetin
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.
Intervention: prosetin
Outcomes
Primary Outcomes
Parts A, B, C, D: Number of Participants Experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: 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
Time Frame: Part C: Up to 28 days; Part D: Up to 54 weeks
Secondary Outcomes
- 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)