A Phase 1/2 clinical trial of the experimental drug NV1205 in patients with childhood cerebral adrenoleukodystrophy (CCALD) has been initiated, announced NeuroVia Inc. The source article can be found here, at Business Wire.
About Childhood Cerebral Adrenoleukodystrophy (CCALD)
Adrenoleukodystrophy (ALD) is a genetic condition that damages myelin, the protective cover around neurones (nerve cells) in the brain, which are involved in thought and movement. Without the myelin sheath, the nerve cells aren’t able to function properly.
According to NORD, childhood cerebral ALD (CCALD) is a form of ALD that may lead to symptoms such as attention deficit disorder, progressive loss of intellectual function, and a decline in motor, hearing, and vision. CCALD can be fatal, often between three to five years after symptoms begin.
An estimated one out of every 17,000 people worldwide are affected by X-ALD.
NV1205 is an experimental synthetic small molecule drug, which NeuroVia says is being developed for the treatment of patients with X-ALD of all phenotypes. It is designed to work by helping cells to process very long chain fatty acids (VLCFA), which can build up and cause damage. It aims to target VLCFA in the brain and adrenal tissue in particular since these are typically main sites that the condition affects.
The Clinical Trial
The initiated clinical trial is a Phase 1/2 study that will investigate how safe and tolerable NV1205 is in patients diagnosed with CCALD, as well as the pharmacokinetics of the drug (what happens to the drug in the body). The study, which will be carried out over eight countries, will include increasing dosage levels and has a long-term treatment phase.
More information about this clinical trial can be found on the government’s website for clinical trials, here. The identifying tag for this clinical trial on the website is NCT03196765.
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