RecruitingPHASE2INTERVENTIONAL

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

This research study is investigating a new drug called dazucorilant for people living with Amyotrophic Lateral Sclerosis (ALS), also known as Motor Neurone Disease. The main goals are to check how safe the new drug is and how well it works to help patients. The study has two parts. The first part compares different doses of dazucorilant with a dummy treatment (placebo) over 24 weeks. Patients who complete this part then have the option to continue receiving the drug in an ‘open-label’ extension for up to 132 weeks, where everyone knows they are getting the active medication. The second part of the study focuses on finding the best way to introduce the medication and how well people tolerate it by gradually increasing the dose. This study is for adults over 18 years old.

At a glance

Status

Recruiting

Phase

PHASE2

Sponsor

Corcept Therapeutics

Enrolment target

279

Start

15 Nov 2022

Estimated completion

01 Nov 2027

Amyotrophic Lateral Sclerosis

What is this study about?

This study is exploring a new treatment called dazucorilant for people diagnosed with Amyotrophic Lateral Sclerosis (ALS). ALS is a serious condition that affects nerves in the brain and spinal cord, leading to muscle weakness over time. Researchers are keen to find new ways to help manage this condition and improve the lives of those affected.

The study has two main phases, designed to gather different types of information. In the first phase, some patients will receive dazucorilant, while others will get a placebo – a treatment that looks like the study drug but contains no active medication. This helps researchers understand if any improvements are genuinely due to dazucorilant or other factors. Patients who complete this initial phase will then have the option to continue taking dazucorilant in a long-term 'open-label' part of the study, where everyone knows they are receiving the active drug. This allows for a deeper look at its long-term effects and safety.

The second phase of the study focuses on how people tolerate dazucorilant and finding the best way to start taking it. Participants in this part will begin with a lower dose, which will be slowly increased until a target dose is reached, as long as it's comfortable for them. This careful approach helps ensure patient safety and comfort while determining the most effective way to use the medication. Ultimately, this study aims to build a comprehensive picture of dazucorilant's potential as a treatment for ALS.

Key takeaways

Tests a new drug, dazucorilant, for ALS.
Aims to check safety and how well it works.
Involves different doses and a dummy treatment (placebo) in some parts.
Includes long-term follow-up options for up to 2.5 years.
Participation is for adults aged 18 and over with ALS.
You can stop participating at any time.

Who may be eligible?

This study is looking for adults aged 18 and over who have been diagnosed with ALS, whether it runs in their family or appeared without a clear family history. To be considered, your disease progression needs to be within a certain range as estimated by doctors during screening. If you are already taking other approved ALS medications like riluzole or edaravone, you must be on a steady dose before joining the study.

There are certain situations that would prevent you from taking part. For example, if you have other serious neurological conditions not related to ALS, or have difficulty swallowing capsules, you won't be able to join. Your kidney function, blood platelet levels, and certain infections (like HIV or hepatitis B/C) will also be checked, as these could affect your eligibility. Women who are pregnant, planning to become pregnant, or breastfeeding cannot participate.

Also, if you're using breathing support like a non-invasive ventilator, a breathing tube, or oxygen supplementation, you wouldn't be able to join this study. For specific parts of the study, there might be additional rules, such as restrictions on a particular gene mutation (SOD1) or a specific combination therapy (sodium phenylbutyrate and taurursodiol).

Quick self-check

Are you 18 years or older?
Do you have a diagnosis of ALS (Motor Neurone Disease)?
Are you able to swallow capsules?
Are you not using non-invasive ventilation, a breathing tube, or oxygen support?
Are you not pregnant, planning to become pregnant, or breastfeeding?
Do you not have other serious neurological conditions unrelated to ALS?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, the study involves regular visits to the clinic for check-ups and assessments. You'll be given the study medication as capsules to take daily. In the first part of the study, you'd take either dazucorilant or a placebo for 24 weeks, without knowing which one it is. If you complete this, you might have the option to continue taking dazucorilant for up to 132 weeks (about 2 and a half years) in an 'open-label' part, where everyone receives the active drug. If you don't enter the open-label part, you'll still have a follow-up period of 132 weeks. In the second part of the study, you'll start with a lower dose of dazucorilant, and this dose will be gradually increased over time until you reach the target dose, provided it's well-tolerated. This part can also lead into a 52-week open-label extension. The total duration of your participation would vary depending on which part of the study you join and whether you continue into the extension phases.

Potential risks and benefits

Participating in any clinical trial involves both potential benefits and risks. You might benefit from receiving a new drug that could help manage your ALS symptoms, or you could simply gain more knowledge about your condition through regular monitoring. However, dazucorilant may cause side effects, some of which are not yet fully known. There's also a chance you might receive the placebo in the first part of the study, meaning you wouldn’t get the active drug. Your safety and well-being are a top priority, and you'll be closely monitored by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (35)

062
Phoenix, United States· Recruiting
278
San Francisco, United States· Recruiting
287
Neptune City, United States· Recruiting
353
New York, United States· Recruiting
108
Leuven, Belgium· Active not recruiting
425
Hamilton, Canada· Active not recruiting
273
Montreal, Canada· Active not recruiting
422
Bron, France· Active not recruiting
258
Lille, France· Active not recruiting
257
Limoges, France· Active not recruiting
261
Marseille, France· Active not recruiting
423
Montpellier, France· Active not recruiting

+23 more sites — see the official record for the full list.

Common questions

What is ALS?

ALS, or Amyotrophic Lateral Sclerosis, is a progressive condition that affects the nerves in the brain and spinal cord, leading to muscle weakness and wasting.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks exactly like the study drug but contains no active medication. It helps researchers compare the effects of the active drug.

Will I know if I'm getting the actual drug?

In the first part of the study, it's 'double-blind,' meaning neither you nor the doctors will know if you're getting dazucorilant or the placebo. In the 'open-label' parts, everyone knows they are receiving dazucorilant.

How long will the study last?

The initial treatment period is 24 weeks. Depending on which part of the study you join and your choice, you could be involved in an extension study for up to 132 weeks (about 2.5 years).

Can I stop participating if I want to?

Yes, your participation is voluntary, and you can withdraw from the study at any time without needing to give a reason, and it will not affect your future medical care.

How to find out more

Clinical Trial Lead

study652@corcept.com (650) 249-9965 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.