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
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.
- Are you an adult male aged 18 or older?
- Do you have cerebral adrenoleukodystrophy (cALD) that is progressing?
- Has your doctor not recommended a stem cell transplant, or are you not keen on one?
- Do you NOT have type 1 or type 2 diabetes?
- Have you NOT had a stem cell transplant or gene therapy before?
- Are you NOT currently in another medical research study involving a drug?
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
Locations (13)
- Stanford University Medical CenterVerified postcodePalo Alto, United States· Recruiting
- Neuro Medicine Hospital / UF HealthVerified postcodeGainesville, United States· Withdrawn
- Kennedy Krieger InstituteVerified postcodeBaltimore, United States· Recruiting
- Massachusetts General HospitalVerified postcodeBoston, United States· Recruiting
- University of MinnesotaVerified postcodeMinneota, United States· Recruiting
- Health University of UtahVerified postcodeSalt Lake City, United States· Withdrawn
- Hospital AustralVerified postcodeBuenos Aires, Argentina· Recruiting
- Federal University of Sao PauloVerified postcodeSão Paulo, Brazil· Recruiting
- ICM La Pitie SalpetriereVerified postcodeParis, France· Recruiting
- Klinik und Poliklinik für Neurologie-LeipzigVerified postcodeLeipzig, Germany· Recruiting
- Sir Ganga Ram HospitalVerified postcodeNew Delhi, India· Recruiting
- Hospital 12 de OctubreVerified postcodeMadrid, 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?
Discussion
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