Dazucorilant for ALS

(DAZALS Trial)

Not currently recruiting at 42 trial locations

Overseen By Clinical Trial Lead

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Corcept Therapeutics

Must not be taking: Glucocorticoids

Disqualifiers: Non-ALS neurologic disorder, HIV, others

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

Compensation: Varies

Number of Visits: Unknown

Duration: 24 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called dazucorilant for people with Amyotrophic Lateral Sclerosis (ALS), a condition affecting nerve cells in the brain and spinal cord, leading to muscle weakness. The trial aims to assess the safety and effectiveness of dazucorilant for those living with ALS. Participants will receive either 300 mg or 150 mg of dazucorilant or a placebo (a pill with no active drug) each day. The trial seeks individuals diagnosed with ALS who are currently stable on medications like riluzole or edaravone. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of people.

Do I have to stop taking my current medications for the trial?

If you are taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, you must be on a stable dose before joining the trial. The protocol does not specify about other medications, so it's best to discuss with the trial team.

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

Research shows that dazucorilant has been tested for safety in people with ALS. In earlier studies, most participants tolerated dazucorilant well. Some experienced side effects, but serious problems were rare. This treatment remains under investigation, so researchers closely monitor its safety. The current trial is in Phase 2, indicating that initial safety results are promising, but further research is necessary to fully understand its effects. ^{1 2 3 4 5}

Why are researchers excited about this study treatment for ALS?

Dazucorilant is unique because it offers a new approach to treating ALS by targeting the body's stress response system, specifically the glucocorticoid receptor. This mechanism is different from current treatments like riluzole and edaravone, which primarily aim to slow disease progression through different pathways. Researchers are excited about dazucorilant because it could potentially provide a more effective way to manage ALS symptoms by reducing inflammation and stress in nerve cells, which might not only slow the disease but also improve patients' quality of life. Additionally, dazucorilant is administered as a once-daily oral pill, making it convenient for patients compared to other treatment forms that may require more complex administration.

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

Research shows that dazucorilant could aid in treating Amyotrophic Lateral Sclerosis (ALS), even though it did not achieve its primary goal in a study. In this trial, participants may receive either 150 mg or 300 mg of dazucorilant, or a placebo. Notably, previous research indicated that patients lived longer, particularly in the 300 mg group, where no deaths occurred, compared to five deaths in the placebo group. The treatment also improved scores on a scale measuring daily activities. These findings suggest dazucorilant may help ALS patients live longer and maintain daily functions. ^{2 3 6 7 8}

Who Is on the Research Team?

Corcept Therapeutics Incorporated

Principal Investigator

Corcept Therapeutics Incorporated

Are You a Good Fit for This Trial?

This trial is for men and women over 18 with ALS, either sporadic or familial. Participants must be on a stable dose of riluzole/edaravone if taking them. Excluded are those with significant non-ALS neurological disorders, prior glucocorticoid modulator treatments, swallowing issues, recent systemic glucocorticoid use, need for diaphragm pacing systems or ventilation support, pregnant/breastfeeding women, and individuals with HIV or hepatitis B/C.

Inclusion Criteria

I have been on a consistent dose of riluzole and/or edaravone before screening.

I am 18 or older with ALS, either by itself or in my family.

Exclusion Criteria

Women who are pregnant, planning to become pregnant, or are breastfeeding

You currently need or may soon need a diaphragm pacing system.

I have a serious nerve disorder that is not ALS.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are randomized to receive either 300 mg or 150 mg of dazucorilant or placebo daily for a 24-week double-blind period

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment if they do not enter the open-label extension

132 weeks

Open-label extension

Participants may opt into continuation of treatment with 300 mg dazucorilant daily for long-term safety and efficacy assessment

132 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Dazucorilant
• Placebo

Trial Overview The study tests the safety and effectiveness of dazucorilant in ALS patients. It involves two different doses of dazucorilant (300 mg and 150 mg) compared to a placebo to see which works better at managing symptoms.

How Is the Trial Designed?

Group I: CORT113176 (Dazucorilant) 300 mg Experimental Treatment 1 Intervention

300 mg of dazucorilant will be administered once daily.

Group II: CORT113176 (Dazucorilant) 150 mg Experimental Treatment 1 Intervention

150 mg of dazucorilant will be administered once daily.

Group III: Placebo (matched to study drug) Placebo Group 1 Intervention

Placebo will be administered once daily.

Who Is Running the Clinical Trial?

Corcept Therapeutics

Lead Sponsor

Trials

72

Recruited

7,700+

Dr. Joseph K. Belanoff

Corcept Therapeutics

Chief Executive Officer since 2014

MD from Stanford University

Dr. William Guyer

Corcept Therapeutics

Chief Medical Officer since 2021

PharmD

Published Research Related to This Trial

In a trial involving 20 Iranian patients with amyotrophic lateral sclerosis (ALS), Edaravone was found to be safe, with no significant injection reactions or adverse events compared to controls.

However, the study did not demonstrate any significant efficacy of Edaravone in improving physical and functional status in ALS patients, indicating that more research is needed before it can be recommended for use outside Japan.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of edaravone in well-defined Iranian patients with amyotrophic lateral sclerosis: A parallel-group single-blind trial. Eishi-Oskouei, A., Basiri, K.[2023]

A multicenter study involving 324 ALS patients found that long-term intravenous edaravone therapy was feasible and generally well tolerated, with potential adverse effects occurring in 16% of patients, primarily infections and allergic reactions.

However, the study showed no significant difference in disease progression or survival outcomes between patients receiving edaravone and those on standard therapy alone, indicating that edaravone may not offer a meaningful clinical benefit in the long-term management of ALS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis. Witzel, S., Maier, A., Steinbach, R., et al.[2023]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05407324?cond=Amyotrophic%20Lateral%20Sclerosis&aggFilters=status:&viewType=Table&rank=3

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS). Detailed ...

2. ir.corcept.com ir.corcept.com/news-releases/news-release-details/corcept-presents-results-phase-2-study-dazucorilant-patients-0

Corcept Presents Results from Phase 2 Study of Dazucorilant ... Overall survival was a secondary endpoint. Although DAZALS did not meet its primary endpoint, patient survival significantly improved. At week ...

3. clinicaltrials.eu clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-dazucorilant-for-patients-with-amyotrophic-lateral-sclerosis-als/

Study on the Safety and Effectiveness of Dazucorilant for ... This clinical trial examines the safety and effectiveness of Dazucorilant (CORT113176) in treating patients with Amyotrophic Lateral ...

4. synapse.patsnap.com synapse.patsnap.com/article/corcept-reports-phase-2-dazucorilant-study-results-in-als-patients

Corcept Reports Phase 2 Dazucorilant Study Results in ... A significant element of the DAZALS trial was its primary endpoint, aimed at assessing the change in the ALS Functional Rating Scale-Revised ( ...

5. firstwordpharma.com firstwordpharma.com/story/5920991

Corcept's ALS drug trial disappoints, but zero deaths in ... Despite missing that goal, Corcept noted that there were no deaths in the higher 300-mg dazucorilant group, compared to five in the placebo arm.

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05407324

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

7. ir.corcept.com ir.corcept.com/news-releases/news-release-details/corcept-announces-results-phase-2-study-dazucorilant-patients

Corcept Announces Results From Phase 2 Study of ... The study's primary endpoint was to slow the decline in motor skills and other functional criteria, compared to placebo, as measured by the ALSFRS-R.

8. als.net als.net/als-trial-navigator/500042/

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).