Clinical Trials Resource Center

Male Hormonal Deficiencies/Abnormalities

February 8, 2016

Zafgen issued results of a pivotal, double-blind, placebo-controlled, phase III trial evaluating the safety and efficacy of beloranib for Prader-Willi syndrome (PWS). The bestPWS ZAF-311 study randomized 107 patients to receive twice-weekly subcutaneous injections of either 2.4mg or 1.8mg of beloranib or placebo. Seventy-four patients completed the full 26 weeks of treatment per the trial protocol, and 27 patients completed at least 75% of the randomized treatment period prior to the suspension of dosing in the trial in October 2015. There were six patients who discontinued early. The co-primary efficacy endpoints for this trial were improvement in hyperphagia-related behaviors and reduction in body weight. Patients in the trial were on average 20 years old, had an average BMI of 40kg/m2 and an average hyperphagia total score of 16.9, consistent with moderate to severe hyperphagia, at the beginning of randomized treatment. Treatment with the 2.4mg and the 1.8mg doses of beloranib resulted in reductions of hyperphagia-related behaviors of 7 units (p=0.0001) and 6.3 units (p=0.0003) relative to placebo, respectively, as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT). Patients randomized to receive placebo displayed substantial (4.15%) gain in body weight over the course of the six months of randomized treatment. Patients treated with beloranib, in contrast to placebo, lost weight, with the 2.4mg dose arm displaying a 5.3% reduction from baseline, with a placebo-adjusted weight loss of 9.45%. In December 2015, the FDA placed the beloranib IND application on complete clinical hold due to an imbalance in severe venous thromboembolic events, including two patient deaths. Zafgen plans to present to the FDA the efficacy and safety data from the bestPWS ZAF-311 study, data from the phase IIb trial of beloranib in severe obesity complicated by type 2 diabetes and a proposal for a risk mitigation strategy for beloranib in PWS.

September 20, 2004

Auxilium Pharmaceuticals has reported the results of a phase IV study of their topical testosterone therapy Testim, for the treatment of hypogonadism in men unable to achieve symptom amelioration with AndroGel, another topical testosterone therapeutic. The study found that Testim therapy was effective in treating symptoms of hypogonadism, significantly improving sexual function and satisfaction, compared with men remaining on a comparable dose of AndroGel. In addition, a significantly higher portion of subjects receiving Testim did not require dose escalation to achieve results at the end of the study period, compared with AndroGel. The study enrolled a total of 151 hypogonadal men who were refractory to or insufficiently served by AndroGel therapy; these subjects were randomized to receive either continued treatments with AndroGel or Testim once daily for 4 weeks.

August 9, 2004

Columbia Laboratories has reported the final results of their phase III study of their approved drug Striant, their testosterone buccal delivery system for the treatment of hypergonadism in men. Results have demonstrated that Striant is and efficacious in treating hypergonadism, with 86.6% of subjects achieving average serum testosterone levels within the literature-established standard physiological range. The drug was safe and generally well tolerated, with 16% of subjects reporting mild application site events. This open-label, multi-center study enrolled a total of 98 hypogonadal men (ages 20-75), who received twice daily intra-buccal applications of the drug for 12 weeks.

June 24, 2002

Phase III trial results suggest that long-term use of Unimed Pharmaceuticals' AndroGel (testosterone gel), a transdermal hormone replacement therapy, is safe and effective in men with low testosterone. The trial included 92 hypogonadal men with an average age of 52 years. Subjects received continuous replacement with testosterone gel for up to 42 months (average of 29 months). At six months, results showed that sexual motivation and performance improved significantly in subjects receiving testosterone gel, and this effect was maintained throughout treatment. An average decrease of 2.5 kg in fat mass was observed at 30 months, in addition to an average increase in lean body mass of 3.5 kg. Bone mineral density was increased at six months, and it remained increased by approximately 4% in the spine and 2% in the hip at 30 months.

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