Largest-ever Parkinson's disease trials
Medical Studies/Trials
Published: Sunday, 8-Apr-2007



Researchers with the National Institute of Neurological Disorders and Stroke (NINDS) have initiated one of the largest-ever Parkinson's disease clinical trials.

The study, which involves more than 50 sites and will enroll over 1,700 patients in the U.S. and Canada, is intended to test a new drug therapy, developed by Palo Alto-based Avicena Group, to slow the progress of Parkinson's disease and help millions of people around the world afflicted with this debilitating disease.

"This study will harness the expertise of some of the top researchers in the country," says Dr. Karl Kieburtz, lead investigator for the study. "Projects like this are evidence of the scientific and business communities' commitment and willingness to collaborate to improve the lives of Parkinson's patients."

Despite the large patient population, and high profile sufferers including Muhammad Ali, Michael J. Fox and Janet Reno, current drugs can only treat the disease symptoms. None has been shown to slow or halt disease progression.

The NINDS-sponsored study will examine the efficacy and safety of PD-02, which has demonstrated the potential to improve neurological function and to slow disease progression in Parkinson's patients.

Parkinson's disease belongs to a group of conditions called movement disorders. Some of the common symptoms of Parkinson's are tremors of the hands, arms, legs and jaw, rigidity or stiffness of the body, slowness of movement, and impaired balance and coordination. Because Parkinson's is a chronic disease symptoms worsen over time.

According to the Parkinson's Disease Foundation, approximately 40,000 new cases of Parkinson's are diagnosed each year in the United States with as many as one and a half million people suffering from the disease in total. Individuals eligible for the NIH study are those who have been diagnosed with Parkinson's within the past five years, and have received and been responsive to therapy in the past three months to two years. This trial is the first in a series of NINDS-sponsored clinical trials called NET-PD (NIH Exploratory Studies in Parkinson's Disease).
www.avicena group.com/

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Promising Phase II Results From Parkinson's Disease Trial Of PD-02
15 Mar 2006

Avicena Group, Inc. (“Avicena”), a developer of novel pharmaceutical and therapeutic products, announced today results from a Phase II study designed to evaluate whether further study is warranted for PD-02, a proprietary Avicena drug candidate, and the antibiotic minocycline, as potential treatments for Parkinson's disease. Data demonstrated that neither PD-02 nor minocycline was found to be futile as compared to predetermined thresholds, suggesting both agents may be worthy of further consideration as potential therapeutics. Results from this study, which was sponsored by the National Institute of Neurological Disorders and Stroke (NIH/NINDS), were published today in the journal Neurology.

This randomized, Phase II, multi-center, double-blind trial enrolled 200 early, untreated Parkinson's disease patients into three different treatment arms (PD-02, 10 grams per day; minocycline, 200 mg per day; or placebo). After twelve months of daily treatment, investigators found that the rate of disease progression for both PD-02 and minocycline was lower than the threshold for futility, as measured by the Unified Parkinson's Disease Rating Scale (UPDRS). Additionally, PD-02 was found to be safe and generally well-tolerated by the subjects during this study.

“We are pleased with the results of this trial. The compounds were found to be generally well tolerated by the subjects participating in the trial,” said Karl Kieburtz, MD, M.P.H., Director, Clinical Trials Coordination Center, University of Rochester. “While these findings were encouraging for both PD-02 and minocycline, we want to emphasize that further research would be needed to determine if either agent can slow the progression of Parkinson's disease.”

PD-02 is a proprietary therapeutic that incorporates an ultra-pure, clinical form of creatine. This ultra-pure, clinical form of creatine has demonstrated the potential to improve neurological function in certain patient populations. In preclinical animal studies of Parkinson's disease, PD-02 has demonstrated significant neuroprotective properties including protection of the dopaminergic cells which are affected in Parkinson's disease.

“Based on the findings from this trial and our strong ongoing relationships with the University of Rochester and NINDS, we are now discussing the potential initiation of a Phase III efficacy trial to evaluate if PD-02 modifies the progression of Parkinson's disease,” stated Belinda Tsao-Nivaggioli, Avicena's chief executive officer. “This potential trial, combined with our ongoing Phase III program in ALS and Phase II program in Huntington's disease, places us in the strong position of having multiple late-stage clinical programs targeting patients in need of new, effective therapies.”

A separate paper outlining the design of this futility study, authored by Barbara C. Tilley, Ph.D., Professor and Chair, Department of Biostatistics, Bioinformatics & Epidemiology, Medical University of South Carolina and colleagues, was also published today in Neurology.

ABOUT FUTILITY STUDIES

Futility trials are unique Phase II studies designed to assess whether it is worthwhile to consider evaluating drug candidates in Phase III efficacy trials. In measuring outcomes of specific compounds against predetermined thresholds, investigators are able to discard those treatments deemed unlikely to be effective in Phase III trials. As a result, these studies may decrease the time to identify treatments unworthy of further pursuit and reduce subjects' exposure to futile treatments. Futility trials, which have most often been used in evaluating cancer therapeutics, are particularly attractive in those diseases, such as Parkinson's disease, for which a number of agents are being considered for investigation.

Avicena Clarifies Description of Creatine Versus PD-02, Company's Lead Drug Compound Used in NIH Phase III Clinical Trial in Parkinson's Disease

(posted on 23/03/2007)

PALO ALTO, Calif., March 23 /PRNewswire-FirstCall/ — Avicena Group, Inc. (OTC Bulletin Board: AVGO), a late stage biotechnology company focused on commercializing its proprietary cellular energy modulation technology, wishes to clarify the difference between its compound, PD-02, being used in the Phase III Parkinson’s disease trial announced on March 22, 2007, versus a common (nutritional supplement) form of creatine.

PD-02, the Parkinson’s disease drug candidate being used in this Phase III clinical trial, is a unique creatine-based formulation that has been manufactured to strict FDA drug GMP guidelines. PD-02 is produced using a patented process that ensures a highly purified form of creatine without the harmful neurotoxins, such as cyanamide, which may be dangerous to patients with neurodegenerative diseases like Parkinson’s.

In addition, only this GMP drug formulation has been shown to be safe and well tolerated in high doses in previous clinical studies conducted by Karl Kieburtz, M.D., M.P.H., of the University of Rochester in New York, the lead investigator of this NINDS sponsored Parkinson’s trial.

Dr. Kieburtz pointed out, “There is a critical distinction between the pharmaceutical grade creatine formulation that is being used in this Parkinson’s disease trial and the creatine that may be sold over-the-counter in retail stores. It is unwise for Parkinson’s patients to conclude that the common form of creatine has any clinical benefits, and I recommend against using it.”

“We go to great lengths to ensure that Avicena’s Parkinson’s disease drug candidate, PD-02, is manufactured under the tightest FDA standards,” stated Belinda Tsao-Nivaggioli, Avicena’s Chief Executive Officer. “Only as a result of these efforts, can the drug’s purity as well as safety and efficacy profile be guaranteed.”

About the Trial

The double-blind, placebo-controlled, phase III study is one of the largest PD clinical trials to date. It will enroll 1,720 people with early- stage Parkinson’s disease at fifty-one medical centers in the United States and Canada. Participants in the phase III study will be evaluated on an ongoing basis for five to seven years. The trial is the first large study in a series of NINDS-sponsored clinical trials called NET-PD (NIH Exploratory Trials in Parkinson’s Disease). NINDS has organized this large network of sites to allow researchers to work with Parkinson’s patients over a long period of time, with a goal of finding effective and lasting treatments. The effort will be led by Karl Kieburtz, M.D., M.P.H., of the University of Rochester in New York, and Barbara C. Tilley, Ph.D., of the Medical University of South Carolina in Charleston, and the patients will be seen by movement disorders specialists at the NET-PD sites across the United States and Canada.