All studies
RecruitingPHASE3INTERVENTIONAL

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

This important study is testing a new medication, Leriglitazone, for adult men diagnosed with cerebral adrenoleukodystrophy (cALD). This is a serious genetic condition that affects the brain and spinal cord. The main goal is to find out if Leriglitazone can help manage the condition and slow its progression. Researchers also want to understand if the drug is safe for people to use. Some participants will receive Leriglitazone, while others will receive a placebo (a dummy pill with no active drug) to fairly compare the effects. This is a Phase 3 study, meaning it's one of the final steps before a drug might be approved for wider use.

At a glance

Status
Recruiting
Phase
PHASE3
Sponsor
Minoryx Therapeutics, S.L.
Enrolment target
40
Start
12 Jul 2023
Estimated completion
01 May 2027

What is this study about?

This research study is focused on a genetic condition called cerebral adrenoleukodystrophy, often shortened to cALD. This condition primarily affects the brain and spinal cord, leading to a range of symptoms. Unfortunately, it tends to get worse over time. Scientists are always looking for new and better ways to help people living with cALD. This particular study is evaluating a new drug called Leriglitazone.

The main purpose of this study is to see if Leriglitazone can effectively help adult men who have cALD. This means looking at whether it can improve symptoms or slow down the disease's progression. Alongside this, the researchers will keep a very close eye on the participants to make sure the drug is safe and doesn't cause too many unwanted side effects.

By carefully comparing the results from people taking Leriglitazone with those taking a placebo (an inactive dummy pill), the researchers can get a clear picture of how well the new drug works. Finding effective treatments for cALD is incredibly important, as it could offer new hope and improve the quality of life for those affected.

Key takeaways

  • This study is testing a new drug for adult men with cerebral adrenoleukodystrophy (cALD).
  • It aims to see if the drug is safe and effective in slowing down the disease.
  • Participants will receive either the drug or a placebo.
  • Strict criteria determine who can participate.
  • Participation involves regular medical check-ups and taking the study medication.
  • You can stop participating at any time if you wish.

Who may be eligible?

This study is specifically looking for adult men aged 18 or older who have been diagnosed with a type of cALD that is currently getting worse, as seen on brain scans. You might be a good fit if a stem cell transplant (HSCT) isn't recommended for you, or if you prefer not to have one.

There are also some specific requirements based on your brain scan scores and your current physical and mental abilities. For example, your brain scan Loes score needs to be within a certain range. You should not have major problems with daily activities, though some issues like being in a wheelchair or bladder control problems are understood as part of the disease and won't stop you from joining.

However, you can't join if you've already had a stem cell transplant or certain gene therapies. People with known type 1 or type 2 diabetes, or allergies to similar medications, also can't take part. You also shouldn't be taking certain other medications or participating in another drug study at the same time.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you an adult male aged 18 or older?
  2. Do you have cerebral adrenoleukodystrophy (cALD) that is progressing?
  3. Has your doctor not recommended a stem cell transplant, or are you not keen on one?
  4. Do you NOT have type 1 or type 2 diabetes?
  5. Have you NOT had a stem cell transplant or gene therapy before?
  6. Are you NOT currently in another medical research study involving a drug?
Answer every question to see your result.

What does participation involve?

Taking part in this study would involve regular visits to the study clinic where you'll have various check-ups and assessments. You will be given either the study drug, Leriglitazone, or a placebo, which you'll take as instructed. The medical team will closely monitor your health, perform tests, and ask you about any symptoms or side effects you might experience. They'll likely do brain scans and other neurological assessments to track the progress of your condition and see how the treatment is working. The exact number of visits and the total length of your participation will be explained by the study team, but clinical trials typically involve follow-up over several months or even longer.

Potential risks and benefits

Participating in this study might offer you access to a new potential treatment for cALD before it's widely available. You'll also receive very close medical monitoring from experts in the field. However, there are potential risks, as with any medication. Leriglitazone might cause side effects, some of which could be serious, and it's not guaranteed to help your condition. The placebo arm means you might not receive the active drug. You are completely free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (13)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • Stanford University Medical Center
    Verified postcode
    Palo Alto, United States· Recruiting
  • Neuro Medicine Hospital / UF Health
    Verified postcode
    Gainesville, United States· Withdrawn
  • Kennedy Krieger Institute
    Verified postcode
    Baltimore, United States· Recruiting
  • Massachusetts General Hospital
    Verified postcode
    Boston, United States· Recruiting
  • University of Minnesota
    Verified postcode
    Minneota, United States· Recruiting
  • Health University of Utah
    Verified postcode
    Salt Lake City, United States· Withdrawn
  • Hospital Austral
    Verified postcode
    Buenos Aires, Argentina· Recruiting
  • Federal University of Sao Paulo
    Verified postcode
    São Paulo, Brazil· Recruiting
  • ICM La Pitie Salpetriere
    Verified postcode
    Paris, France· Recruiting
  • Klinik und Poliklinik für Neurologie-Leipzig
    Verified postcode
    Leipzig, Germany· Recruiting
  • Sir Ganga Ram Hospital
    Verified postcode
    New Delhi, India· Recruiting
  • Hospital 12 de Octubre
    Verified postcode
    Madrid, Spain· Recruiting

Common questions

What is cerebral adrenoleukodystrophy (cALD)?

It's a serious genetic condition that mainly affects the brain and spinal cord, causing problems with movement, thinking, and other body functions.

What is Leriglitazone?

Leriglitazone is a new medication being tested in this study to see if it can help slow down or manage cALD.

What does 'placebo' mean?

A placebo is a 'dummy pill' that looks exactly like the study drug but contains no active medicine. It helps researchers fairly compare the drug's effects.

Who can join this study?

This study is for adult men (18 and over) with cALD that is getting worse, and who meet specific health and medical history requirements.

Will I definitely get the new drug?

You might receive Leriglitazone or the placebo. This is decided by chance, like flipping a coin, to make the study fair.

How to find out more

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

Interested in taking part?

Register your interest

Share your details and the research team for "A Clinical Study to Assess the Efficacy and Safety of Lerigl…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

Discussion

Community discussion

Powered by our forum at community.patient.info. Please be respectful — this is not medical advice.