Dazucorilant for ALS

(DAZALS Trial)

Recruiting at 35 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

249 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing a new medication called dazucorilant to see if it can help people with ALS. ALS is a serious disease with few treatment options, so new treatments are needed. The medication might work by slowing down the disease or making symptoms less severe.

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.

How does the drug Dazucorilant differ from other ALS drugs?

Dazucorilant is unique because it is being explored as a potential treatment for ALS, whereas existing drugs like Riluzole and Edaravone have shown only modest benefits. Unlike these drugs, Dazucorilant may offer a novel approach, but its specific mechanism and effectiveness in ALS are still under investigation. ^{1 2 3 4 5}

Research Team

Grace Mann, PhD

Principal Investigator

Corcept Therapeutics

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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

Findings from Research

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Riluzole and edaravone: A tale of two amyotrophic lateral sclerosis drugs. [2019]

2. England pubmed.ncbi.nlm.nih.gov

Glatiramer acetate has no impact on disease progression in ALS at 40 mg/day: a double- blind, randomized, multicentre, placebo-controlled trial. [2019]

3. Iran 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. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Safety and Effectiveness of Long-term Intravenous Administration of Edaravone for Treatment of Patients With Amyotrophic Lateral Sclerosis. [2023]

5. England pubmed.ncbi.nlm.nih.gov

Edaravone for the treatment of amyotrophic lateral sclerosis. [2020]