ADULT CEREBRAL ADRENOLEUKODYSTROPHY (cALD)
RESEARCH IS HAPPENING NOW.
Cerebral adrenoleukodystrophy (cALD) is a rare neurological disease that may develop in men with adrenomyeloneuropathy (AMN). Those with AMN are generally recommended to do yearly MRI screenings for cerebral ALD. If you have AMN or cALD, this clinical trial could be a good option. For pre-qualified participants, an MRI for cALD is provided at no cost. If a diagnosis of cALD is made, you may be able to receive an investigational medication for cALD.
FILL OUT THE FORM BELOW TO APPLY
Your name and information will not be shared to third parties without your authorization.
THE SCREENING PROCESS
Also known as X-linked ALD, is a rare inherited genetic disease with progressive neurological (both the brain and spinal cord) and adrenal gland disorder. Both males and females can have the disease. The adrenal part of the disease is typically in the male patients. The genetic mutation affects how the body breaks down a particular fat molecule (very long chain fatty acids) which leads to high levels of these causing damage to the white matter in the brain and spinal cord.
The disease is often described by two terms related with the neurological disorder which can co-exist in adults and only one form occurs in childhood:
Cerebral adrenoleukodystrophy (cALD)
A rare neurological disease that may develop in men with AMN, a form of X-linked ALD. There is currently no cure for adult cALD, and treatment options are limited.
A form of ALD that primarily affects men and can cause difficulty walking, numbness and tingling in the feet, and muscle spasms. Those with AMN are generally recommended to do yearly brain MRI screenings for cerebral ALD, which can develop at any time.
FREQUENTLY ASKED QUESTIONS
The purpose of this study is to evaluate if the study drug, leriglitazone, is able to increase survival and slow disease progression for those with cALD when compared to placebo.
Study-related care is provided at no cost to participants and no insurance is necessary to participate. Travel reimbursement is available. Ask your local study site for more details.
No. A diagnosis of X-ALD, AMN, or cALD is not required to apply for pre-screening if you have a family history of these diagnoses, or symptoms that strongly suggest you may have one of these diagnoses.
Minoryx Therapeutics is the sponsor of this study. Minoryx is a biotech company focused on developing novel therapies for severe genetic diseases of the central nervous system.
Leriglitazone is an investigational oral medication for central nervous system diseases such as cALD.
Yes. Your personal and health information will only be used for the purposes of the study and is never shared with unauthorized 3rd parties.
If you move on to the screening stage of the study, the site staff will explain the study in more detail as part of the informed consent process.