72 Participants Needed

Prosetin for ALS

(PRO-101 Trial)

Recruiting at 3 trial locations
PC
ID
Overseen ByIngela Danielsson, MD, PhD, MBA
Age: 18+
Sex: Any
Trial Phase: Phase 1
Sponsor: ProJenX
Must be taking: Riluzole

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests prosetin, a new treatment for amyotrophic lateral sclerosis (ALS), a disease affecting nerves and muscles. The main goal is to assess the safety and tolerability of prosetin in both healthy volunteers and people with ALS. The trial includes several parts, administering single and multiple doses of prosetin to different groups. People with ALS, diagnosed using specific criteria and able to manage daily tasks like swallowing liquids, might be suitable candidates. Participants must not have other significant health issues or have participated in other drug trials recently. As a Phase 1 trial, this research focuses on understanding how prosetin works in people, offering participants the chance to be among the first to receive this new treatment.

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.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that prosetin was safe in earlier studies. Healthy volunteers tolerated both single and multiple doses well compared to a placebo, with no major safety issues reported. Prosetin is also considered safe for long-term use, suggesting suitability for extended treatment. These results support further testing in people with ALS. However, since this trial is in an early phase, it will focus more on assessing safety and tolerance.12345

Why do researchers think this study treatment might be promising for ALS?

Prosetin is unique because it offers a fresh approach to treating ALS (amyotrophic lateral sclerosis). Unlike traditional treatments like riluzole and edaravone, which primarily focus on slowing disease progression, prosetin targets a novel mechanism. It acts on the cellular stress response system, aiming to protect neurons from the damage caused by ALS. Researchers are excited about prosetin's potential to not only slow down the disease but also improve the quality of life for patients by preserving motor function longer.

What evidence suggests that prosetin might be an effective treatment for ALS?

Research suggests that prosetin might help treat ALS by targeting a key process involved in the disease. ALS, or amyotrophic lateral sclerosis, causes nerve cells to break down, leading to loss of muscle control. Prosetin aims to protect these nerve cells from damage. Early studies have shown that prosetin can reduce ALS symptoms and protect nerve cells. In this trial, participants with ALS will receive either multiple ascending doses of prosetin or a placebo to evaluate its effectiveness. Although more research is needed, these findings offer hope that prosetin could be a promising treatment for people with ALS.35678

Are You a Good Fit for This Trial?

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.
My overall health is good as confirmed by recent medical exams.
I agree to use birth control as required.

Timeline for a Trial Participant

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

What Are the Treatments Tested in This Trial?

Interventions

  • placebo
  • prosetin
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.
How Is the Trial Designed?
7Treatment groups
Experimental Treatment
Placebo Group
Group I: Part D - open-label administration of prosetin in participants with ALSExperimental Treatment1 Intervention
Group II: Part C - multiple ascending doses of prosetin in participants with ALSExperimental Treatment1 Intervention
Group III: Part B - multiple ascending doses of prosetinExperimental Treatment1 Intervention
Group IV: Part A - single ascending doses of prosetinExperimental Treatment1 Intervention
Group V: Part A - single dose of placeboPlacebo Group1 Intervention
Group VI: Part B - multiple doses of placeboPlacebo Group1 Intervention
Group VII: Part C - multiple doses of placebo in participants with ALSPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

ProJenX

Lead Sponsor

Trials
1
Recruited
70+

Congressionally Directed Medical Research Programs

Collaborator

Trials
59
Recruited
10,600+

Worldwide Clinical Trials

Collaborator

Trials
70
Recruited
15,800+

Citations

NCT05279755 | A Study to Evaluate the Safety and ...The primary purpose of this study is to evaluate the safety and tolerability of prosetin in healthy volunteers and participants with ALS. Detailed Description.
2.projenx.comprojenx.com/?p=383
ProJenX Announces Removal of Partial Clinical Hold for ...The United States Food and Drug Administration (FDA) has removed a partial clinical hold on Study PRO-101, a hybrid Phase 1 clinical trial evaluating prosetin.
Genetic and Mechanistic Insights Inform Amyotrophic ...Real-world evidence of riluzole effectiveness in treating amyotrophic lateral sclerosis. Amyotroph Lateral Scler Frontotemporal Degener.
FDA Removes Partial Clinical Hold on Phase 1 Trial ...The phase 1 study, dubbed PRO-101, assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in both patients with ALS and healthy ...
Phase 1 trial of prosetin to create digital twins of ALS patientsA treatment goal is to ease ALS symptoms. The AI-based model will draw on data collected from patients at trial entry to forecast outcomes for ...
ProJenX Receives European Authorization for First Study ...Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co-founders at Columbia University for the treatment of ALS ...
2022 Amyotrophic Lateral Sclerosis HighlightIt should be noted that prosetin is orally bioavailable, metabolically stable, CNS-penetrant, and well-tolerated in long-term administration ...
NCT05279755 | A Study to Evaluate the Safety and ...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- ...
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