Phase 2 Motor Neurone Disease Trial, Recruiting NCT05407324 Sponsor: Corcept Therapeutics Condition: Motor Neurone Disease
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Phase 2 Motor Neurone Disease Trial, Recruiting

NCT05407324
Recruiting Phase 2

Who may be able to join

AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI

Who might be able to join this trial:

  • Adults aged 18 or older who have been diagnosed with ALS (either inherited or not inherited from a family member).
  • In Part 1 of the study: people whose ALS is expected to progress at a certain rate, measured by a specific scoring system called the ENCALS risk profile (score between -6 and -3) (confirm with trial site).
  • In Part 2 of the study: people whose ALS is expected to progress at a certain rate, measured by a different scoring system called the TRICALS risk profile (score between -7 and -3) (confirm with trial site).
  • People already taking riluzole, edaravone, and/or sodium phenylbutyrate with taurursodiol, as long as the dose has been stable for a set period before the study begins (note: sodium phenylbutyrate with taurursodiol is not allowed in Part 2).
  • In Part 2 only: people with a specific genetic mutation (in the SOD1 gene) who are not currently receiving or eligible to receive a drug called tofersen, or who previously tried tofersen but stopped due to safety or effectiveness concerns.
  • In Part 2 only: people taking a very high dose of a vitamin B12 treatment (methylcobalamin) for ALS, as long as the dose has been stable for at least 11 weeks before the study starts.

Who may not be able to join:

  • People who have a significant neurological condition other than ALS.
  • People who are unable to swallow capsules.
  • People with a low blood platelet count (below 150,000/mm³), which can affect how the blood clots.
  • People with poor kidney function, based on a specific kidney test result (eGFR of 30 or below); in Part 2, people who recently had a sudden decline in kidney function must have recovered to their normal kidney function before joining.
  • People who have HIV, or an active or ongoing hepatitis B or hepatitis C infection (in Part 2, this also applies to people with a known history of these conditions).
  • Women who are pregnant, planning to become pregnant, or currently breastfeeding.
  • People who are already using breathing support devices, such as a non-invasive breathing machine, a ventilator connected through the throat, or any form of supplemental oxygen.
  • People who are currently being treated for cancer (with some exceptions for certain early-stage or well-controlled skin or cervical cancers), or who have a cancer history where life expectancy is less than 2 years.
  • People who currently need or are expected to need a diaphragm pacing system (a device that helps the breathing muscle work).
  • People who have previously been treated with a type of drug called a glucocorticoid receptor modulator or antagonist (confirm with trial site).
  • People who have recently used certain steroid medications (including tablets, inhalers, or strong skin creams/ointments) within a time period based on how long that specific steroid stays in the body; people who stop steroids should have another treatment option available if their condition gets worse during the study.

Important: Always verify eligibility with the trial site directly before applying.

Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.

This is a simplified plain English summary of the eligibility criteria. Full criteria are set by the trial investigators and may include additional requirements not shown here. Never self-exclude from a trial based on this summary. Contact the trial site directly to confirm your eligibility.
Last synced 23 July 2026
Phase 2: approximately ~30% of drugs entering this phase reach regulatory approval, based on published industry-wide historical data. This is not specific to this trial.
Phase success rates shown are historical industry-wide averages based on published data. They are not a prediction for this specific trial or your individual situation.

Contact this trial

Principal Investigator: Sophia Majeed, PharmD, PhD, Corcept Therapeutics Incorporated

Phone: (650) 249-9965

Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.

GP referral letter

Print a one-page summary to share with your doctor.

Trial details

Status
Recruiting
Phase
Phase 2
Sponsor
Corcept Therapeutics
Registry
ClinicalTrials.gov
Start date
15 November 2022
Est. completion
1 October 2026

Where this trial is recruiting

🇧🇪 Belgium 🇨🇦 Canada 🇫🇷 France 🇩🇪 Germany 🇮🇪 Ireland 🇳🇱 Netherlands 🇵🇱 Poland 🇪🇸 Spain 🇬🇧 United Kingdom 🇺🇸 United States

Primary endpoints

Change from Baseline to Week 24 in the ALS Functional Rating Scale-Revised (ALSFRS-R) total score.; Incidence of Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, AEs by severity, and deaths due to AEs; Incidence of treatment-emergent AEs and SAEs; Incidence of treatment-emergent AEs leading to dose interruptions, dose reductions, and/or discontinuations of study drug

Can't join this trial?

Expanded access pathways

If this trial is not available to you, other access pathways may exist. In Australia, the TGA Special Access Scheme allows access to unapproved therapeutic goods for individual patients.

TGA Special Access Scheme information

Find other recruiting trials on ClinicalTrials.gov

Data last synced from ClinicalTrials.gov: 23 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.

Trial recruitment status can change without notice between our nightly data updates. Always contact the trial site directly to confirm current recruitment status before making any decisions or travel arrangements.

View original record on ClinicalTrials.gov