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Chronic Fatigue Syndrome

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October 18, 2004

Hemispherx Biopharma presented expanded data from their phase III study of Ampligen, for the treatment of chronic fatigue syndrome (CFS), at the 7th International Conference for CFS in Madison, Wisconsin. The data, which covered subjects in the intent-to-treat population, demonstrated that subjects receiving the drug experienced symptom relief, with a significant improvement in treadmill exercise tolerance (p=0.037) compared with placebo; this improvement was more than twice the level required to achieve medical significance. Secondary safety endpoints were also satisfied, with no significant difference in serious adverse events, missed dosages or dropouts between Ampligen and placebo groups. The double-blind, placebo-controlled study randomized 234 patients with severe/debilitating CFS across 12 US sites to receive twice weekly doses of either 400 mg. Ampligen or placebo for 40 weeks. Following these results, Hemispherx announced that they were preparing for the filing of a NDA for Ampligen; if approved, the drug would be the first drug indicated for the treatment of CFS.

NPS Pharmaceuticals reported negative results of a proof-of-concept clinical trial of NPS 1776 (isovaleramide), for the treatment of acute migraine headaches. The study failed to meet its primary endpoint, a significant reduction in acute headache pain two hours after administration compared with placebo. NPS attributed this failure to a higher-than expected placebo effect: 56% of subjects receiving placebo achieved primary response, compared with 60% of subjects receiving the higher dose of the drug and 64% of subjects receiving the lower dose. Secondary safety endpoints were met, with no serious adverse events and no minor events occurring more frequently with the drug than placebo. The double-blind, placebo-controlled study randomized 189 patients with moderate-to-severe migraine pain to receive one of two doses of the drug or placebo. NPS announced that they planned to continue investigation of the drug in other indications, but did not report the intention to continue pursuing the drug for migraine.

Prana Biotechnology announced the results of an open-label extension of a phase II study of PBT-1 (clioquinol), a copper-zinc attenuator/amyloid-beta aggregation inhibitor for the treatment of Alzheimer’s disease (AD). Data from the study confirmed the positive results achieved in the original trial: subjects experienced a mean decline of 8 points on the ADAS-cog scale, a standardized diagnostic scale for cognitive performance in AD, compared with a literature-established expected decline of 18 points. The drug was also found to be safe and well tolerated, with no significant increase in adverse events over untreated subjects. This 48-week open-label extension to the 36-week double-blind, placebo-controlled phase II CQAD study enrolled 18 of the original participants, 9 of whom completed treatment. Prana announced that they were continuing to evaluate the study data, in preparation for advancing the drug into further clinical studies.

May 17, 2004

Genzyme reported positive results from a phase II trial investigating tolevamer sodium, an investigational polymer therapy for the treatment of Clostridium difficile associated diarrhea. The primary endpoint was non-inferiority to vancomycin with respect to time to resolution of diarrhea. Data demonstrated that tolevamer met the non- inferiority endpoint at the six gram dose level. In addition, tolevamer was found to be similar to vancomycin in median days to resolution of diarrhea. The randomized, double-blind, active-controlled study enrolled 300 subjects at 58 sites in the U.S., Canada and the UK. It was designed to determine the safety and effectiveness of tolevamer at two dose levels (6-3 g per day), versus an oral dose of vancomycin. Results were reported at the European Congress of Clinical Microbiology and Infectious Diseases meeting in Prague. Based on these results, Genzyme is now planning phase III trials to begin in early 2005.

Hemispherx Biopharma reported positive results from a phase III trial investigating Ampligen, an immunomodulator and antiviral drug for the treatment of Chronic Fatigue Syndrome (CFS). Results demonstrated a statistically significant increase in physical performance as measured by Treadmill Exercise Tolerance Testing compared with placebo, the study’s primary endpoint. Data showed that subjects on Ampligen had an improved exercise treadmill performance of 19.4% compared with 5.1% for subjects given placebo. The multi-center, double-blind, randomized, placebo-controlled pivotal study enrolled 234 subjects with CFS at 12 sites in the U.S. The study was designed to test the efficacy and safety of Ampligen at 400 mg twice weekly for 40 weeks. Results were reported at the 17th International Conference on Antiviral Research in Tucson, Arizona.

Vicuron Pharmaceuticals reported positive results from a phase II trial investigating dalbavancin, an injectable glycopeptide antibiotic for the treatment of catheter-related bloodstream infections (CR-BSI). The primary endpoint showed an overall response rate of 87% in dalbavancin treated subjects compared with 50% in those treated twice daily with vancomycin. The randomized, comparative, open-label study enrolled 67 subjects with CR-BSI in North America. Subjects received two doses of dalbavancin one week apart or vancomycin twice daily for 14 days. The primary endpoint was based on a composite of clinical and microbiological responses measured at 21 days after treatment. Results were reported at the 14th annual European Congress of Clinical Microbiology and Infectious Diseases meeting in Prague.