[Editor’s Note updating article: According to ALD Life, the PEDALS trial investigating NV1205 as a potential treatment for adrenoleukodystrophy has been placed on a temporary hold by NeuroVia. A reason for this action is not given in the group’s notice, but it is reported to not be related to either the treatment or to regulatory actions. NeuroVia expects to resume it study by year’s end.]
NeuroVia started a Phase 1/2 clinical study to evaluate the potential of its lead candidate, NV1205, for the treatment of children and adolescents with cerebral adrenoleukodystrophy (ALD).
The study, named PEDALS (NCT03196765), was designed to evaluate the safety, tolerability, and overall stability in the body of increasing doses of NV1205. It is planned to enroll 25 patients, ages 4 to 18, who have been diagnosed with childhood cerebral adrenoleukodystrophy (CCALD).
The study will have two parts. In part 1 researchers will evaluate the safety of increasing doses of NV1205 for 12 weeks. This will be followed by a long-term treatment for up to a total of 96 weeks.
Patients will be recruited into one of three groups, and receive daily oral NV1205 at the assigned dose. Upon completion of the initial four weeks in the first group, the collected safety data will be evaluated by an independent Data Safety Monitoring Board (DSMB). If no safety issues are found, NV1205 dosing will start in the second group with the second predefined dose, and a similar protocol will be conducted for the third group.
Additional groups may be enrolled at the recommendation of the DSMB with an incremental dose increase.
The study is recruiting across 13 clinical centers in eight countries — Argentina, Australia, Chile, Colombia, France, the Russian Federation, Ukraine, and the United Kingdom — and is expected to be completed by the end of 2020.
“Advancing our lead candidate NV1205 into clinical trials represents a key milestone in the company’s development,” John Henderson, chief development officer of NeuroVia, said in a press release. “We believe NV1205 has real potential to better serve the X-linked ALD community as a novel pharmaceutical treatment for all X-ALD patients.”
NV1205 is a synthetic small molecule being developed to restore the metabolism of some fatty molecules, called very long chain fatty acids (VLCFA), and prevent their damaging effects to the brain and adrenal glands associated with X-ALD.
NeuroVia’s candidate is expected to stimulate the production of a gene called ABCD2, which encodes an ALD-related protein. This new protein will overcome the faulty metabolism of VLCFA in X-ALD by facilitating the transport and degradation of these fatty molecules, and preventing their accumulation in cells and tissues.
Previous tests in mice with a genetic defect similar to that found in X-ALD patients demonstrated that NV1205 can effectively reduce VLCFA levels in the blood, adrenal glands, and brain.