AR1001 for Alzheimer's Disease

Recruiting at 94 trial locations

Overseen By Marshall Nash, MD

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: AriBio Co., Ltd.

Must be taking: Cholinesterase inhibitors, Memantine

Must not be taking: PDE-5 inhibitors, CYP3A4 inhibitors

Disqualifiers: Pregnancy, Delirium, Schizophrenia, Cancer, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

1150 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called AR1001 (also known as Mirodenafil) to determine its effectiveness in improving memory and thinking skills in people with early Alzheimer's disease. Participants will receive either AR1001 or a placebo (a pill with no active ingredient) for 52 weeks, with an option to continue AR1001 for another 52 weeks. The trial seeks individuals who have experienced mild memory or cognitive issues related to Alzheimer's in the past five years and have a partner to assist in monitoring their progress. As a Phase 3 trial, this study represents the final step before potential FDA approval, offering participants a chance to contribute to a treatment that could soon become widely available.

Will I have to stop taking my current medications?

The trial requires that participants stop taking certain medications, such as potent inhibitors of CYP3A4, at least 14 days before starting the study. If you are on an oral cholinesterase inhibitor or memantine, your dose must be stable for at least 3 months before the trial and remain unchanged during the study.

Is there any evidence suggesting that AR1001 is likely to be safe for humans?

Research has shown that AR1001 underwent safety testing in earlier studies. In one study involving 210 people with mild to moderate Alzheimer's disease, AR1001 was generally well-tolerated. About 82% of participants completed the 26-week treatment. Both the 10 mg and 30 mg doses produced similar safety outcomes, indicating similar types of side effects.

Although specific side effects were not listed, "well-tolerated" implies that any side effects were manageable or not severe enough to cause most participants to stop treatment. This research suggests that AR1001 might be safe for individuals with early Alzheimer's disease. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Alzheimer's?

Unlike the standard treatments for Alzheimer's, which typically focus on managing symptoms with cholinesterase inhibitors or memantine, AR1001 is believed to work differently by targeting neurodegenerative processes at a cellular level. Researchers are excited about AR1001 because it potentially enhances cellular energy production and supports neuron survival, which could slow the progression of the disease. This unique mechanism offers hope for a more direct intervention in the disease process rather than just symptom management.

What evidence suggests that AR1001 might be an effective treatment for Alzheimer's?

Research has shown that AR1001, which participants in this trial may receive, may help treat Alzheimer's disease. In earlier studies, AR1001, a drug that can improve blood flow and brain signals, helped people with mild to moderate Alzheimer's think and remember better. Those who took AR1001 showed improved memory and thinking skills compared to those who did not. Additionally, AR1001 reduced harmful proteins in the brain, like amyloid-β and phosphorylated tau, which are linked to Alzheimer's. These early findings suggest that AR1001 could be a useful option for slowing down Alzheimer's symptoms. ^{1 2 3 4 6}

Who Is on the Research Team?

Sharon J Sha

Principal Investigator

Stanford University

James Rock

Principal Investigator

AriBio Co., Ltd.

Are You a Good Fit for This Trial?

Adults aged 55-90 with early Alzheimer's Disease (AD), confirmed by specific cognitive tests and biomarkers, can join this trial. They need a reliable caregiver to assist and must not have other dementia causes or severe health issues like recent heart problems, uncontrolled seizures, or certain infections.

Inclusion Criteria

My MRI ruled out other causes for my dementia.

Participants who have one (or more) identified adult study partners(s) who, in the opinion of the Investigator, has sufficient contact with and knowledge about the participant as to be able to report knowledgably about the participant's cognition, function, behavior, and safety, and compliance with the protocol. The informant/care partner must be available by phone to provide information to the Investigator and study staff about the participant as well as agree to attend in-person clinic visits that require partner input for scale completion. The informant/care partner must be literate and provide informed consent and should be available for the duration of the study. The same informant/care partner is required to be consistent across all study visits except under rare, unavoidable circumstances (e.g., unexpected informant health crisis) that are approved by the Investigator and Sponsor

I have mild memory loss or mild dementia due to Alzheimer's disease.

Exclusion Criteria

I haven't had cancer or a malignant tumor in the last 5 years, except for certain types.

Participants who in the opinion of the Investigator have an inadequately treated thyroid disorder

I am not taking, or can stop taking, drugs that strongly affect liver enzyme CYP3A4 for 2 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive AR1001 30 mg or placebo daily for 52 weeks

52 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

52 weeks

Extension

Eligible participants receive AR1001 30 mg daily for an additional 52 weeks

52 weeks

What Are the Treatments Tested in This Trial?

Interventions

• AR1001
• Placebo

Trial Overview The study is testing AR1001 against a placebo in people with early AD. It's a Phase 3 trial where participants are randomly assigned to either the new drug or an inactive substance without knowing which one they receive.

How Is the Trial Designed?

Group I: Group A - Active Comparator Active Control 1 Intervention

Active, AR1001 30 mg QD will be administered daily for 52 weeks during the Treatment Phase of the study. In the Extension Phase, all eligible participants who choose to participate will receive AR1001 30 mg QD for 52 weeks.

Group II: Group B - Placebo Comparator Placebo Group 1 Intervention

Placebo QD will be administered daily for 52 weeks during the Treatment Phase of the study. In the Extension Phase, all eligible participants who choose to participate will receive AR1001 30 mg QD for 52 weeks.

Who Is Running the Clinical Trial?

AriBio Co., Ltd.

Lead Sponsor

Trials

3

Recruited

1,400+

Citations

1. neurologylive.com neurologylive.com/view/phase-2-trial-data-support-ar1001-potential-alzheimer-disease-monotherapy

Phase 2 Trial Data Support AR1001 as Potential Alzheimer ... AR1001, a PDE5 inhibitor, shows potential cognitive and biomarker benefits in mild to moderate Alzheimer's disease, particularly as monotherapy.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12501329/

A phase 2 randomized, placebo-controlled study on the ... A total of 210 participants were enrolled and 82% completed 26 weeks of treatment. AR1001 10 mg and 30 mg were well-tolerated with a similar safety profile ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT03625622

NCT03625622 | Efficacy and Safety of 26-Week Treatment ... Based on the preclinical results, AR1001 could be an effective treatment option with a mechanism of action that has not been explored for AD indication.

4. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/40912996/

A phase 2 randomized, placebo-controlled study on the ... Background: AR1001 is a phosphodiesterase-5 inhibitor that produces improved cognitive performance and reduces amyloid-β and phosphorylated tau ...

5. alzdiscovery.org alzdiscovery.org/uploads/cognitive_vitality_media/Mirodenafil_%28drug%29.pdf

Mirodenafil No studies have tested whether mirodenafil prevents age-related cognitive decline or dementia. Observational research linking the use of other ...

6. sciencedirect.com sciencedirect.com/science/article/pii/S2274580725002791

A phase 2 randomized, placebo-controlled study on the ... A total of 210 participants were enrolled and 82% completed 26 weeks of treatment. AR1001 10 mg and 30 mg were well-tolerated with a similar safety profile ...