Clinical trials - ALS

Planned Clinical Trial in ALS Patients Evaluating Adeona’s Zinc-Based Therapy

Preparations are underway to evaluate the safety and efficacy of Adeona’s proprietary drug candidate, AEN-100, a gastroretentive, sustained-release zinc-based tablet, in a multi-center, double-blind, placebo-controlled clinical trial in ALS patients intended to be conducted under an investigator-initiated Investigational New Drug (IND) application. Adeona will provide the study medications and fund the clinical trial, which will be conducted by the neurology team at the PNA Center for Neurological Research in collaboration with The Institute for EthnoMedicine.

Efforts are also underway for the manufacturing of clinical trial medications and protocol review by an Institutional Review Board. The multi-center trial is intended to take place at up to six major ALS centers in the United States. It is anticipated that the trial will enroll at least 60 ALS patients, who will continue on RILUTEK® (riluzole) as the standard of care treatment. The patients will be randomized into treatment and matching placebo groups and will receive clinical trial medications for up to 9 months with periodic monitoring. The treatment group will receive Adeona’s proprietary drug candidate, AEN-100, and a commercially available copper pill.

The planned co-primary endpoints are: 1) determining the safety of AEN-100 given to ALS patients once daily, by assessing adverse events and measuring zinc and copper levels every three months; and 2) determining the efficacy of AEN-100 by assessing the rate of disease progression as measured by the ALSFRS-R, a revised ALS functional rating scale that incorporates assessments of respiratory function.

The planned secondary endpoints are: 1) measuring levels of beta-methylamino-L-alanine (BMAA) in blood and urine to determine if there is a decline in these levels over the course of treatment; 2) following disease progression as measured by Forced Vital Capacity (FVC), a measurement of lung function that has been shown to correlate with disease survival; and 3) following progression of muscle weakness through quantitative strength measurements using hand-held dynamometry.

Ongoing Phase I/II Open Label Safety Study of Zinc Therapy in ALS Patients

Currently the clinical investigators at the PNA Center for Neurological Research are conducting a Phase I/II open label study of zinc therapy in ALS patients to determine the safety of zinc in conjunction with low doses of copper. To date, no safety issues related to zinc therapy have been observed in the ALS patients.

RILUTEK® (riluzole) is a registered trademark of sanofi-aventis U.S. LLC.

Additional links:

PNA Center for Neurological Research

Phoenix Neurological Associates, Ltd.

The Institute for EthnoMedicine