AP-101 for ALS

Recruiting at 14 trial locations

Overseen By Study Director: AL-S Pharma SA

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: AL-S Pharma

Must be taking: Riluzole, Edaravone

Disqualifiers: Cognitive impairment, Severe cardiovascular, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 51 weeks

63 Participants Needed

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.

Do I need to stop my current medications for the AP-101 ALS trial?

If you are taking riluzole or edaravone, you must be on a stable dose and are expected to stay on the same dose throughout the study. The protocol does not specify about other medications, so it's best to discuss with the study team.

Research Team

Study Director

Principal Investigator

AL-S Pharma SA

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 don't use BiPAP/PAV for ALS symptoms more than 4 hours. I may use CPAP for other conditions.

I am using birth control as required.

Exclusion Criteria

I have undergone stem cell therapy.

I have not had SOD1 targeting treatment in the last 6 months.

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

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AP-101 or placebo administered by IV to evaluate safety, tolerability, PK, and PD

51 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• AP-101
• Placebo

Trial Overview 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.

Participant Groups

Group I: AP-101 Experimental Treatment 1 Intervention

AP-101 is administered by IV.

Group II: Placebo Placebo Group 1 Intervention

Placebo is administered by IV.

Who Is Running the Clinical Trial?

AL-S Pharma

Lead Sponsor

Trials

2

Recruited

80+