Health TrialsbyHealthPanel

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorCorcept Therapeutics

About this trial

The purpose of this 2-part study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male and female patients ≥18 years of age with sporadic or familial ALS. In Part 1, patients must have a risk of ALS progression characterized by a European Network for the Cure of ALS (ENCALS) risk profile score ≥ -6 and ≤ -3. In Part 2 patients must have a risk of ALS progression characterized by an Treatment Research Initiative to Cure ALS (TRICALS) risk profile score ≥ -7 and ≤ -3.

If taking riluzole, edaravone, and/or sodium phenylbutyrate and taurursodiol, must be on a stable dose prior to Screening. Sodium phenylbutyrate and taurursodiol are not permitted for patients enrolled in Part 2 of the study.

Part 2 only: Patients with a pathogenic mutation in superoxide dismutase 1 gene (SOD1) must not be receiving treatment with tofersen or eligible for treatment with tofersen if available. Patients who have received prior treatment with tofersen and discontinued due to safety and/or efficacy reasons prior to Screening are eligible.

Part 2 only: Use of ultra high-dose methylcobalamin for the treatment of ALS is permitted provided the patient has been on a stable dose for ≥11 weeks prior to the Day 1 visit.

Disqualifiers

History of a clinically significant non-ALS neurologic disorder

Inability to swallow capsules.

Blood platelet count <150,000/mm^3.

Renal impairment indicated by Estimated Glomerular Filtration Rate (eGFR) ≤30 mL/min/1.73 m². Part 2 only: Patients with a recent history of acute kidney injury should have returned to their baseline renal function (i.e, eGFR prior to acute kidney injury) prior to enrollment.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Dazucorilant 300 mg

    Drug

    300 mg of dazucorilant will be administered once daily in 4 capsules of 75 mg dazucorilant/capsule.

  • Dazucorilant 150 mg

    Drug

    Dazucorilant and placebo will be administered once daily in 4 capsules, 2 capsules with 75 mg dazucorilant/capsule, and 2 capsules of placebo equivalent.

  • Placebo

    Other intervention

    Placebo will be administered once daily in capsules of placebo equivalent.

  • Dazucorilant

    Drug

    Dazucorilant will be administered once daily in 75-mg capsules.

Treatment groups

279 Participants
are divided into 4 treatment groups
Group A: Part 1: CORT113176 (Dazucorilant) 300 mgExperimental treatment 1 intervention
Group B: Part 1: CORT113176 (Dazucorilant) 150 mgExperimental treatment 2 interventions
Group C: Part 1: Placebo (matched to study drug)Placebo comparator 1 intervention
Group D: Part 2: CORT113176 (Dazucorilant) 75 - 300 mg Dose-titrationExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.

This outcome measure is assessed in study Part 1.

Time frame
Baseline to Week 24
2

Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, AEs by severity, and deaths due to AEs

This outcome measure is assessed in study Part 1.

Time frame
Baseline to Week 24
3

Incidence of treatment-emergent AEs and SAEs

This outcome measure is assessed in study Part 2.

Time frame
Baseline up to Week 12
4

Incidence of treatment-emergent AEs leading to dose interruptions, dose reductions, and/or discontinuations of study drug

This outcome measure is assessed in study Part 2.

Time frame
Baseline up to Week 12

Secondary outcomes

1

Change from Baseline to Week 24 in muscle strength (assessed using hand-held dynamometer)

This outcome measure is assessed in study Part 1.

Time frame
Baseline to Week 24
2

Change from Baseline to Week 24 in Percent Slow Vital Capacity

This outcome measure is assessed in study Part 1.

Time frame
Baseline to Week 24
3

Change from Baseline to Week 24 in EuroQol-5 Dimensions-5 Levels (EQ-5D-5L)

This outcome measure is assessed in study Part 1.

Time frame
Baseline to Week 24
4

Time to Death

This outcome measure is assessed in study Part 1.

Time frame
From randomization date to the date of death from any cause up to 156 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Corcept Therapeutics

Lead sponsor

Source ClinicalTrials.gov
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