Prosetin for ALS

(PRO-101 Trial)

Recruiting at2 trial locations

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1

Recruiting

Sponsor: ProJenX

Must be taking: Riluzole

Must not be taking: Investigational drugs

Disqualifiers: Dementia, Cancer, Seizures, others

Trial Summary

What is the purpose of this trial?

This trial tests a new drug called prosetin on healthy adults to ensure it is safe and understand how the body processes it.

Will I have to stop taking my current medications?

If you are taking riluzole or other standard ALS treatments, you must be on a stable dose for at least 30 days before the trial and continue it throughout the study. The protocol does not specify about other medications, so it's best to discuss with the trial team.

Research Team

Eligibility Criteria

Adults aged 18-65 with a BMI of 18.0 to 32.0, in good health as determined by medical exams, and willing to use contraception can join this trial. It's for healthy volunteers and those with ALS.

Inclusion Criteria

My BMI is between 18.0 and 32.0.

I am between 18 and 65 years old.

My overall health is good as confirmed by recent medical exams.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive multiple ascending doses of prosetin or placebo for 14 days

2 weeks

Daily visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension

Participants with ALS may opt into continuation of treatment with prosetin for up to 52 weeks

52 weeks

Treatment Details

Interventions

• placebo (Other)
• prosetin (Other)

Trial Overview The study is testing the safety of Prosetin, a potential treatment for ALS. Participants will receive either Prosetin or a placebo to compare effects.

Participant Groups

Group I: Part D - open-label administration of prosetin in participants with ALS Experimental Treatment 1 Intervention

Participants will be administered a once-daily dose of prosetin for up to 52 weeks.

Group II: Part C - multiple ascending doses of prosetin in participants with ALS Experimental Treatment 1 Intervention

Participants will be administered a once-daily dose of prosetin at multiple ascending dose levels for 14 days.

Group III: Part B - multiple ascending doses of prosetin Experimental Treatment 1 Intervention

Healthy volunteers were administered a once-daily dose of prosetin at 0.06 or 0.10 mg/kg for 14 days.

Group IV: Part A - single ascending doses of prosetin Experimental Treatment 1 Intervention

Healthy volunteers were administered a single dose of prosetin oral solution at 0.03, 0.06, 0.12, or 0.24 mg/kg.

Group V: Part A - single dose of placebo Placebo Group 1 Intervention

Healthy volunteers were administered a single dose of prosetin-matched placebo oral solution.

Group VI: Part B - multiple doses of placebo Placebo Group 1 Intervention

Healthy volunteers were administered a once-daily dose of prosetin-matched placebo for 14 days.

Group VII: Part C - multiple doses of placebo in participants with ALS Placebo Group 1 Intervention

Participants are administered a once-daily dose of prosetin-matched placebo for 14 days.

Who Is Running the Clinical Trial?

ProJenX

Lead Sponsor

Trials

1

Recruited

70+

Congressionally Directed Medical Research Programs

Collaborator

Trials

59

Recruited

10,600+

Colonel Mark G. Hartell

Congressionally Directed Medical Research Programs

Director, Congressionally Directed Medical Research Programs since 2022

PhD in Analytical Chemistry from Auburn University, MS in Biophysical Chemistry from Ohio State University, BS in Chemistry from State University of New York at Oswego

Dr. Sarah Goldman

Congressionally Directed Medical Research Programs

Chief Medical Officer since 2023

MD

Worldwide Clinical Trials

Collaborator

Trials

70

Recruited

15,800+

Peter Benton

Worldwide Clinical Trials

Chief Executive Officer since 2024

MBA in Finance and Strategy from The Wharton School, University of Pennsylvania; Bachelor's degree in Mechanical Engineering from Northeastern University

Dr. Michael F. Murphy

Worldwide Clinical Trials

Chief Medical Officer since 1997

MD and PhD in Pharmacology with training at Tulane University, Stanford University, and Mt. Sinai School of Medicine