A Phase 1 interventional study of prosetin and placebo in Amyotrophic Lateral Sclerosis, sponsored by ProJenX. Active, not recruiting at 4 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-27.
Sponsored by ProJenX · Phase 1, Interventional, and Treatment
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.
981 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 283 are open to participants now.
This study's planned enrollment of 72 is above the median of 36 across 667 interventional studies indexed under Amyotrophic Lateral Sclerosis.
Browse Amyotrophic Lateral Sclerosis studies →This is the only study on the registry with ProJenX as lead sponsor.
Counted across the registry records on this site, refreshed daily.
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
Key Exclusion Criteria - Part C
Key Inclusion Criteria- Part D
Participants who meet all of the following criteria may be included in Part D of the study:
Key Exclusion Criteria- Part D
NOTE: Other protocol-defined Inclusion/Exclusion Criteria may apply. Please contact trials@projenx.com with any questions about eligibility criteria.
Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution.
Drug: placebo
Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg.
Drug: prosetin
Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days.
Drug: placebo
Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days.
Drug: prosetin
Participants are administered a once-daily dose of prosetin-matched placebo for 14 days.
Drug: placebo
Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days.
Drug: prosetin
Participants will be administered a once-daily dose of prosetin for up to 52 weeks.
Drug: prosetin
oral solution
oral solution
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
Parts A, B, C, and D: Maximum Observed Concentration (Cmax) of Prosetin in Plasma
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: Time to Reach Maximum Observed Concentration (Tmax) of Prosetin in Plasma
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: Area Under the Concentration-Time Curve (AUC) of Prosetin in Plasma
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: Apparent Terminal Elimination Half-life (t1/2) of Prosetin in Plasma
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 and D: Measure of Concentration of Prosetin in CSF
Time frame: Part A: Day 1; Part D: Up to 48 weeks
Plan to share: No
This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Amyotrophic Lateral Sclerosis→