The IACFSME conference last year included a report from Lucinda Bateman on this trial.
I looked at the data at the time. The patients got a lot better but so did the controls! Certainly a frustrating one.
Here's the rundown from the conference:
The Synergy Trial for CFS – A Phase 2 Study of Low-Dose Methylphenidate plus Mitochondrial Support in Patients with Chronic Fatigue Syndrome
Lucinda Bateman, MD, Nancy Klimas, MD, Jose Montoya, MD, Susan Levine, MD, Jon D. Kaiser, MD
Background
CNS stimulants have been utilized to treat CFS symptoms though their benefits are often limited and their tolerability is uncertain. Evidence of mitochondrial dysfunction in CFS patients has also been identified in several publications. Micronutrient support has been shown to improve mitochondrial function. One open-label trial in CFS patients has been published showing a significant benefit to combining these two treatment modalities.
Objectives
This Phase 2 multicenter, double-blinded, placebo-controlled trial (n=128) was performed at four US research clinics to examine the clinical effects and safety profile of combining low-dose methylphenidate plus mitochondrial micronutrient support in patients with CFS.
Methods
128 CFS patients (1994 Fukuda criteria) were randomized in double-blinded fashion to two parallel arms: a double treatment group (methylphenidate + mitochondrial support) and a double placebo group to investigate the potential synergistic effect of this combination. Fatigue and concentration disturbance symptoms were measured at baseline, 4 weeks, and 12 weeks using two clinically validated tools: Checklist Individual Strength (CIS) and Visual Analog Scale (VAS). The primary objective of the study was to measure the treatment’s safety as well as its efficacy utilizing the change in CIS total score.
Results
At twelve weeks there was a change in the mean CIS total score of -16.9 in the treatment group and -13.8 for the placebo group when compared to baseline (P = 0.36). In the PP population, there was a change in the mean CIS total score of -20.9 in the treatment group and -12.6 for the placebo groups at Day 84 (P = 0.19). The change in the mean VAS score for fatigue from baseline was -18.2 in the treatment group and -11.1 in the placebo group (P = 0.19). Adverse events were not significantly different between the two groups.
Conclusion
Treatment with low-dose methylphenidate plus mitochondrial micronutrient support in CFS patients was well tolerated during this trial. Using a validated patient reported outcome measurement tool, overall CFS symptoms decreased in a majority of the treatment subjects. Though statistical significance was not achieved in this Phase 2 trial, all analyses revealed an advantage to the treatment group. Further investigation is warranted.
- Lucinda Bateman, MD, Medical Director Bateman Horne Center1002 E. South Temple, Suite 408
Salt Lake City, Utah 84102
- This trial was sponsored by K-PAX Pharmaceuticals, Mill Valley, CA - No conflicts of interest.