AP-101 for ALS

No longer recruiting at 16 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AP-101 for individuals with ALS (Amyotrophic Lateral Sclerosis), a condition that affects nerve cells and causes muscle weakness. The goal is to assess the safety and tolerability of AP-101 compared to a placebo, which contains no active medication. Participants will receive these treatments through an IV, delivering medication directly into the bloodstream. The trial seeks individuals who have experienced ALS symptoms for less than two years and have muscle weakness. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group.

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.

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

Research has shown that AP-101 was well-tolerated in earlier studies. In one study involving individuals with sporadic ALS and a specific gene mutation (SOD1-ALS), AP-101 met its main safety goals, and no major safety issues emerged. Another study found no harmful effects that prevented dose escalation. The most common side effects were mild. These findings suggest that AP-101 is generally safe for humans. ^{1 2 3 4 5}

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

Unlike the standard treatments for ALS, which often include medications like riluzole and edaravone, AP-101 is administered via IV and represents a novel approach. Researchers are excited about AP-101 because it potentially offers a new mechanism of action that targets specific pathways involved in ALS progression, which are not addressed by current therapies. This innovative focus provides hope for improved outcomes and a new avenue for treatment in the fight against this challenging condition.

What evidence suggests that AP-101 might be an effective treatment for ALS?

Research has shown that AP-101, which participants in this trial may receive, may help treat ALS (Amyotrophic Lateral Sclerosis). In previous studies, AP-101 proved to be safe and well-tolerated by patients. Some patients with both common and inherited forms of ALS experienced significant improvements. The treatment targets a protein called SOD1, linked to ALS, and this may help slow the disease. These findings suggest that AP-101 could benefit some people with ALS. ^{1 2 5 6 7}

Who Is on the Research Team?

Study Director

Principal Investigator

AL-S Pharma SA

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. neurimmune.com neurimmune.com/news/al-s-pharma-announces-positive-topline-results-from-phase-2-study-of-ap-101-for-the-treatment-of-amyotrophic-lateral-sclerosis-als

AL-S Pharma announces positive topline results from ... Phase 2 of AP-101 in sporadic ALS and mutant SOD1-ALS met its primary safety and tolerability endpoint. Clinically meaningful changes in ...

2. neurologylive.com neurologylive.com/view/ap-101-meets-primary-end-point-phase-2-study-sod1-als

AP-101 Meets Primary End Point in Phase 2 Study ... AL-S Pharma's AP-101 shows promising safety and efficacy in ALS patients, paving the way for future treatments targeting misfolded SOD1 ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03981536

A Study to Evaluate AP-101 in Familial and Sporadic ... A Study to Evaluate AP-101 in Familial and Sporadic Amyotrophic Lateral Sclerosis (ALS). ClinicalTrials.gov ID NCT03981536. Sponsor AL-S Pharma. Information ...

4. prnewswire.com prnewswire.com/news-releases/al-s-pharma-announces-positive-topline-results-from-phase-2-study-of-ap-101-for-the-treatment-of-amyotrophic-lateral-sclerosis-als-302545686.html

AL-S Pharma announces positive topline results from ... Phase 2 of AP-101 in sporadic ALS and mutant SOD1-ALS met its primary safety and tolerability endpoint; Clinically meaningful changes in ...

5. alsnewstoday.com alsnewstoday.com/news/ap-101-holds-promise-als-topline-data-show/

AP-101 holds promise for some with ALS, topline data show A year of treatment with AP-101 led to positive results for patients in a Phase 2 clinical trial, topline data showed.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05039099

NCT05039099 | A Study to Evaluate, Safety, Tolerability ... The purpose of this study is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.

7. neurology.org neurology.org/doi/10.1212/WNL.96.15_supplement.4484

A Phase 1, Multicenter, Open Label, Single-Ascending ... To date, no dose-limiting toxicities effects or any safety and tolerability concerns related to AP-101 have been observed. The most common ...