Preliminary Results from Synergy Trial Released

The long-awaited preliminary results from the K-PAX Synergy Trial are out. While both the patient group and control groups showed improvements, the difference between the two was not statistically significant. The Synergy Trial was designed to produce an FDA-approvable treatment for Myalgic Encephalmyelitis/Chronic Fatigue Syndrome. The double-blinded, placebo-controlled trial evaluated the safety and efficacy of methylphenidate (generic Ritalin), combined with a mitochondrial support nutrient formula over 12 weeks. The outcome measurement tool used in the study was the Checklist Individual Strength (CIS), a patient-reported symptoms questionnaire specifically developed to evaluate CFS symptomatology.prescription-300x199

Per-Protocol Analysis: The Per-Protocol analysis included all study subjects who completed the 12-week study and were at least 75 percent compliant with taking the study treatment:

  • The patients in the KPAX 002 Treatment Group experienced an average 22-point decline in their CIS total symptoms score.
  • The patients in the Placebo Group experienced an average 13-point decline in their CIS total symptoms score.

The company says that, although the KPAX 002 group experienced almost twice the improvement as the patients taking placebo, the difference between the two groups did not achieve statistical significance, primarily due to the relatively small sample size—87—of the Per-Protocol population.

Intent-to-Treat Analysis (ITT): The ITT analysis included all study subjects who were randomized, took at least one dose of the treatment, and completed at least one post-baseline evaluation. This group included both study dropouts and non-compliant patients and is a much more rigorous analysis:

  • The patients in the KPAX 002 Treatment Group experienced an average 17-point decline in their CIS total symptoms score.
  • The patients in the Placebo Group experienced an average 13-point decline in their CIS total symptoms score.

The company says that due to the large improvement seen in the placebo group, the difference between the two groups did not achieve statistical significance.

Previous Methylphenidate Alone Study

Only one prior study has been performed for the treatment of CFS using the same dosage of methylphenidate (without mitochondrial support nutrients) and using the same outcome variable (CIS total score). This study was published in 2006 by Blockmans and colleagues in the American Journal of Medicine.

  • The patients in the Treatment Group of this study demonstrated an average 11-point decline in their CIS total symptoms score.
  • The patients in the Placebo Group of this study demonstrated an average 3-point decline in their CIS total symptoms score

Next Steps

Overall, K-PAX says it is encouraged by the substantial decline in patient symptoms and the treatment’s significant safety profile. The company says it will continue to review the more than 2,000 pages of additional data and secondary analyses and share additional information from the clinical trial as it becomes available.The company also will be collaborating with the study’s investigators to publish the full study results in a peer-reviewed medical journal.

To have your name added to the Synergy Trial update list, send a request to ask@KPAXpharm.com

To read a description of the Synergy Trial for CFS, go to www.thesynergytrial.org

May 14, 2015
  • Camilla Cracchiolo, RN

    That’s a hell of a statement “did not achieve statistical significance primarily due to small sample size.” Or maybe it just doesn’t work. I’m not against a bigger study but a negative result should be reported as such and not modified in a way that denotes wishful thinking.

  • MR AIDAN G WALSH

    ADHD does not even exist it was a scam from day one to sell above mentioned Drug now they want to give it to CFS patients what a total Farce…