Darifenacin for ALS

Recruiting at 2 trial locations

Overseen By Gabriele Riva, PhD

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: McGill University

Must be taking: Riluzole, Edaravone, Albrioza

Must not be taking: Anticonvulsants, Antibiotics, Antifungals, others

Disqualifiers: Glaucoma, Liver disease, Neurodegenerative diseases, others

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 22 weeks

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the safety and effects of Darifenacin for people with ALS, a condition that affects nerves and muscles, leading to severe disability. Researchers hope Darifenacin will improve nerve connections and muscle function in ALS patients. Participants will receive either the drug or a placebo (a non-active pill) to compare results. Those diagnosed with ALS within the last three years and who can swallow tablets might be suitable for this study. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

If you are taking standard ALS medications like Riluzole, Edaravone, or Albrioza, you must be on a stable dose before starting the trial and continue that dose during the study. If you are taking certain other medications, like strong CYP3A4 inducers or inhibitors, or antimuscarinic medications, you may need to stop them before joining the trial.

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

Research has shown that Darifenacin is FDA-approved for treating overactive bladder, indicating its general safety for that condition. This study examines how well individuals with ALS, a progressive nerve disease, can tolerate Darifenacin. Past studies reported that participants tolerated the treatment well, experiencing mild side effects such as dry mouth and constipation. This ongoing study aims to confirm these results specifically for ALS patients. ^{1 2 3 4 5}

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

Unlike the standard ALS treatments, which often include drugs like riluzole and edaravone that primarily aim to slow down disease progression, darifenacin offers a unique approach by potentially addressing symptoms more directly. Darifenacin is traditionally used to manage overactive bladder by blocking specific receptors that control muscle contractions. Researchers are excited about the possibility that this drug might help manage ALS symptoms by reducing unwanted muscle activity, offering a new way to improve quality of life for patients. This different mechanism of action sets darifenacin apart, providing hope for a novel method of symptom management in ALS.

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

Research has shown that Darifenacin, which participants in this trial may receive, might help treat ALS by targeting specific parts of the body. This drug may aid certain cells, called glial cells, in repairing themselves. These cells play a crucial role in maintaining healthy nerve connections. In studies with mice that had ALS, these cells began to repair when given Darifenacin, suggesting it might also help protect nerve connections in humans. Although more research is needed, these findings offer hope that Darifenacin could improve muscle function and quality of life for those with ALS. ^{1 2 5 6 7}

Who Is on the Research Team?

Oliver Blanchard, MD

Principal Investigator

McGill University - Montreal Neurological Institute

Are You a Good Fit for This Trial?

This trial is for people aged 18-85 with ALS, a progressive neurological disorder. Participants will be taking Darifenacin or a placebo to see if it can help maintain nerve and muscle connections by influencing certain cells at the neuromuscular junction.

Inclusion Criteria

I have been diagnosed with ALS.

My ALS symptoms started less than 3 years ago.

I have been on a consistent treatment plan for ALS before joining the study.

Exclusion Criteria

History of drug and/or alcohol abuse

History of hepatitis C virus antibody or acute/prolonged hepatitis B & C

Currently enrolled in another clinical trial with an Investigational Product

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Titration

Participants receive a daily dose of either a placebo or 7.5 mg darifenacin for two weeks

2 weeks

1 visit (in-person)

Treatment

Participants receive a daily dose of 15 mg darifenacin or placebo for 22 weeks

22 weeks

Regular intervals (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Darifenacin

Trial Overview The study tests Darifenacin's safety and effects in ALS patients. It involves taking a daily dose of this medication for six months, starting with 7.5 mg and increasing to 15 mg after two weeks, compared to a placebo.

How Is the Trial Designed?

Group I: Darifenacin Treatment Experimental Treatment 1 Intervention

Patients in the Treatment arm will receive a daily dose of 1 or 2 darifenacin extended-release tablets.

Group II: Placebo Placebo Group 1 Intervention

Patients in the Placebo arm will receive a daily dose of 1 or 2 placebo tablets.

Darifenacin is already approved in United States, European Union for the following indications:

Approved in United States as Enablex for:

• Overactive bladder with symptoms of urge urinary incontinence, urgency and frequency

Approved in European Union as Emselex for:

• Overactive bladder with symptoms of urge urinary incontinence, urgency and frequency

Who Is Running the Clinical Trial?

McGill University

Lead Sponsor

Trials

421

Recruited

1,017,000+

Oliver Blanchard

Lead Sponsor

Université de Montréal

Collaborator

Trials

223

Recruited

104,000+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9798295/

Identification of an immune-related gene prognostic index for ... Identification of an immune-related gene prognostic index for predicting prognosis, immunotherapeutic efficacy, and candidate drugs in amyotrophic lateral ...

2. als.org als.org/blog/four-trials-selected-als-associations-clinical-trials-awards-program

Four Trials Selected For The ALS Association's Clinical ... A $997,000 grant will support research led by Richard Robitaille at Universite de Montreal to study the effects of Darifenacin on the connection ...

3. app.trialscreen.org app.trialscreen.org/trials/phase-2-to-assess-safety-tolerability-pharmacology-darifenacin-patients-trial-nct06249867

A Phase 2 study of Darifenacin in ALS A Phase 2 study of Darifenacin in ALS. A study to evaluate the safety and effects of the drug Darifenacin in patients with Amyotrophic Lateral Sclerosis (ALS).

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06249867

NCT06249867 | A Study to Assess the Safety, Tolerability ... The purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS.

5. ctv.veeva.com ctv.veeva.com/study/a-study-to-assess-the-safety-tolerability-and-pharmacology-of-darifenacin-in-patients-with-als

A Study to Assess the Safety, Tolerability, and Pharmacology ... The purpose of the current Phase 2 trial is therefore to test the safety, tolerability, and pharmacology of Darifenacin in patients with ALS.

6. trialx.com trialx.com/clinical-trials/listings/302523/a-study-to-assess-the-safety-tolerability-and-pharmacology-of-darifenacin-in-patients-with-als/?&radius=50

A Study to Assess the Safety, Tolerability, and ... The trial will evaluate the effects of this medication on several outcome measures including patient safety, physical and neurological function, ...

7. clinconnect.io clinconnect.io/trials/NCT06249867

A Study to Assess the Safety, Tolerability, and - ClinConnect This clinical trial is studying a medication called Darifenacin to see if it is safe and tolerable for people with Amyotrophic Lateral Sclerosis (ALS), a ...