AP-101 for ALS

Recruiting in Palo Alto (17 mi)

+14 other locations

Age: 18+

Sex: Any

Travel: May be covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: AL-S Pharma

Prior Safety Data

Trial Summary

What is the purpose of this trial?This trial is testing a new treatment called AP-101 to see if it is safe and well-tolerated. It focuses on people with familial and sporadic ALS, a condition that affects nerve cells and muscle control. Researchers want to understand how AP-101 moves through the body and its effects on ALS symptoms.

Eligibility Criteria

This trial is for adults with familial or sporadic ALS who meet specific clinical criteria, including normal lab results and a slow vital capacity of at least 50% predicted values. Participants must not be pregnant, nursing, or have other neuromuscular conditions. They should not have had certain treatments like stem cell therapy recently and must agree to use contraception.

Inclusion Criteria

I have been diagnosed with ALS based on specific criteria and show progressive motor impairment.

I have a confirmed SOD1 mutation linked to my familial ALS.

Exclusion Criteria

I have undergone stem cell therapy.

I use a breathing machine for my ALS symptoms more than 4 hours daily.

I do not have severe heart, blood, kidney, brain diseases, lung problems, or mental health issues.

I have had a tracheostomy for my ALS symptoms.

I have conditions that cause muscle weakness.

Participant Groups

The study tests the safety and effects of AP-101 on people with ALS compared to a placebo. It looks at how the body processes the drug (pharmacokinetics) and its impact on disease markers (pharmacodynamics). Some participants may continue in an open-label extension based on investigator judgment.

2Treatment groups

Experimental Treatment

Placebo Group

Group I: AP-101Experimental Treatment1 Intervention

AP-101 is administered by IV.

Group II: PlaceboPlacebo Group1 Intervention

Placebo is administered by IV.