PEDALS (Pediatric Adrenoleukodystrophy Study) is an ongoing clinical study to investigate the potential of the investigational drug NV1205 and its effect on Childhood Cerebral Adrenoleukostrophy (CCALD). This website is intended to serve as a resource for the X-ALD community. 

What is X-linked Adrenoleukodystrophy?

X-linked adrenoleukodystrophy (X-ALD) is a genetic disorder that can damage the nervous system and adrenal glands.

X-ALD is a genetic disease caused by the mutation of a gene located on the X chromosome.  Since males carry only one copy of the X-chromosome, mutation of genes on the X-chromosome generally have a much greater impact in males compared with females who have two X-chromosomes and may therefore have a normal copy of the gene maintaining some degree of functionality.

The affected gene is called the ABCD1 gene and its function is to facilitate the breakdown (metabolism) of certain fatty acids known as very long chain fatty acids (VLCFA). When the gene is not working, abnormal accumulation of VLCFA happens in cells especially in adrenal glands and the nervous system and causes damage.

The red box in the image below indicates patients targeted for the PEDALS study (NV1205-009).

What is NV1205?

NV1205 is an investigational oral drug that is taken once a day and may have utility in treating CCALD. NV1205 has not yet been approved by any national regulatory agency, such as the United States Food and Drug Administration (FDA).

NV1205 has been given to a number of healthy adult males in Phase I studies. However, since it has not been tested in X-ALD patients, the potential safety risks are not yet known, but patients will be carefully followed by their physician.

How Does NV1205 Work?

VLCFA need to be transported into the peroxisome (structures in a cell), to be broken down. The protein responsible for this transport is the adrenoleukodystrophy protein (ALDP) and is produced by ABCD1 gene, which is missing in ALD patients. NV1205 stimulates the expression of another gene called ABCD2 that produces the ALD-related protein (ALDRP). This protein has a similar function to ALDP and facilitates VLCFA transport into and degradation by the peroxisome within cells which inhibits the damaging  accumulation of VLCFA in cells. NV1205 has been tested in animals that have a genetic defect similar to X-ALD patients. In these animal studies, NV1205 reduced VLCFA in plasma, adrenal glands and brain tissues. However, until we test NV1205 in people with the disease we cannot know for certain if a similar reduction in VLCFA will happen in patients or if such lowering is beneficial.

What is this Study About?

PEDALS, which stands for Pediatric Adrenoleukodystrophy Study, is a clinical trial that has been set up to learn more about the drug NV1205 (sobetirome) and its effect on X-ALD patients. Because patients with X-ALD are missing a gene that allows the body to handle VLCFA normally, high concentrations of VLCFA develop in a variety of cell types where they are believed to cause damage. Therefore, the PEDALS study drug is set to explore the therapeutic potential of stimulating the expression of a gene that can help cells metabolize these fatty acids. In experiments in animals with a similar genetic mutation as found in X-ALD resulting in high levels of VLCFA, NV1205 was able to reduce VLCFA in certain tissues.  However, until NV1205 is tested in people with the disease we cannot know for certain if a similar reduction in VLCFA will happen in patients, or if a similar reduction in VLCFA will result in improvement in the symptoms of the disease in people.

Clinical research studies, such as PEDALS, are a way for doctors to determine if a medication is useful in fighting a disease. NV1205 has never been tested in children with CCALD. However, NV1205 has been shown to be safe and tolerable in healthy volunteers. Talk to your doctor before joining a clinical study.

Who Can Participate in this Study?

Boys between the ages of 4-18 years

Diagnosed with cerebral form of X-ALD

Evidence of brain lesions in MRI

How Long Will the Study Last?

The study may take up to 2 years.

What is Involved in the Study?

If you agree to participate in this study, you will be asked to do the following things:

  • Have blood samples collected
  • Have brain Magnetic Resonance Images (MRI) taken
  • Have tests of your heart function and vital signs (blood pressure, heart rate, and breathing rate)
  • Have neurological and eye exams performed several times

For More Information

For more information on the study visit:
www.clinicaltrials.gov (NCT03196765)

If you or someone you know would like more information about this study please contact
patients@neurovia-inc.com

Current Planned Study Locations
Australia, Argentina, Chile, Colombia, United Kingdom

This website contains information related to general medical conditions and is provided for educational purposes only. It is not intended to replace discussions with a healthcare provider. All decisions regarding patient care must be made with a healthcare provider, considering the unique characteristics of the patient.

The study described here investigates treatments or outcomes that have not received regulatory approval from a health authority. The information presented here is not meant to convey conclusions of safety or effectiveness. There is no guarantee that the outcome of this trial will result in approval by a regulatory authority.