Neuroprotection Exploratory Trials in Parkinson's Disease

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NET-PD investigators studied 5 potential therapies: CoQ10, GPI 1485, minocycline, creatine, and pioglitazone. Of the 5, creatine was shown to have the most promising characteristics as a potential neuroprotective agent, and was evaluated further in a large, long-term trial, LS-1.

CoQ10 and GPI 1485

In this study, scientists tested CoQ10 (an antioxidant also known as ubiquinone) and GPI 1485 (a novel immunophilin compound) to determine whether either or both were safe, well-tolerated, and had possible neuroprotective potential in the treatment of PD. In the study, 213 men and women with early, untreated PD were randomized to receive CoQ10, GPI 1485, or a placebo (an inactive substance). The study found that CoQ10 and GPI-1485 may warrant further study in PD, however the data was inconsistent.

Primary Paper:

Neurology, Jan 2007; 68: 20-28 (A randomized clinical trial of coenzyme Q10 and GPI-1485 in early Parkinson disease [pdf, 259KB]) (Permission granted by Lippincott Williams & Wilkins)

Minocycline and Creatine

The primary objective of this trial was to assess the impact of minocycline and creatine on the progression of PD and determine if it was futile or non-futile to proceed with further study of these agents. In the study, 200 people with early, untreated PD were equally randomized to receive minocycline, creatine, and a placebo. The study found that both creatine and minocycline should be considered for Phase III trials to determine if they alter the long-term progression of PD.

Primary Paper:

Neurology, Mar 2006; 66: 664-671 (A randomized, double-blind, futility clinical trial of creatine and minocycline in early Parkinson disease [pdf, 82KB]) (Permission granted by Lippincott Williams & Wilkins)

LS-1

The goal of this long-term study was to determine if creatine—an investigational compound—is able to slow the progression of PD. Creatine, a widely used dietary supplement is thought to improve exercise performance. In animal models and human studies, creatine has been shown to be well tolerated and may have some ability to protect brain cells. In the LS-1 study, 1,741 participants were randomly assigned to receive either creatine or a placebo (inactive substance). The study found that creatine did not slow the progression of PD.

Primary Paper and Results:

Writing Group for the NINDS Exploratory Trials in Parkinson Disease (NET-PD) Investigators, Kieburtz K, Tilley BC, Elm JJ, Babcock D, et al. Effect of creatine monohydrate on clinical progression in patients with Parkinson disease: a randomized clinical trial. JAMA. 2015 Feb 10;313(6):584-93. doi: 10.1001/jama.2015.120.

NET-PD LS-1 Creatine in Parkinson's Disease (Clinicaltrials.gov – Results)
https://clinicaltrials.gov/ct2/show/results/NCT00449865

FS-Zone

In the FS-Zone study, 216 men and women with early PD received pioglitazone or a matching placebo (an inactive substance). Researchers compared two doses of pioglitazone, 15 and 45 milligrams, with a placebo to find out how it effects the progression of PD over 44 weeks. This study also explored the effect of pioglitazone on motor skills, cognitive function, mood, and biomarkers. Biomarkers are biological characteristics or features that can provide information on how the disease changes over time and may be useful in developing new treatments and improving clinical care in the future. The study found that pioglitazone, in the doses studied, was unlikely to modify the progression of PD.

Primary Paper:

NINDS Exploratory Trials in Parkinson Disease (NET-PD) FS-ZONE Investigators. Pioglitazone in early Parkinson’s disease: a phase 2, multicentre, double-blind, randomized trial. Lancet Neurol. 2015 Aug; 14(8):795-803. doi: 10.1016/S1474-4422(15)00144-1. Epub 2015 Jun 23.

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