Clinical Trials Resource Center

New Medical Therapies™

Idiopathic Inflammatory Myopathies

Patient Medical Areas

June 15, 2009

Novartis released positive results from a phase III trial of Ilaris (canakinumab) for the treatment of cryopyrin-associated periodic syndrome (CAPS). This 48-week, multinational, randomized, double-blind and placebo-controlled study enrolled 35 subjects, aged 9 to74 years old. The trial was divided into three parts: in the first part, lasting 8 weeks, 35 subjects received a single dose of Ilaris 150 mg by subcutaneous injection. All but one showed a rapid and complete clinical response. Thirty-one subjects proceeded to part two, a randomized 24-week, double-blind placebo-controlled phase. Subjects were treated every 8 weeks with either Ilaris or placebo. Part two of the study included the primary endpoint: a comparison Ilaris and placebo in the proportion of subjects who experienced disease relapse or flares at the end of 8 weeks. The primary endpoint was reached; none of the subjects in the Ilaris group experienced a disease flare compared to 13 out of 16 subjects in the placebo group (0% versus. 81%, p<0.001). The subjects who relapsed during part two of the study discontinued and entered part three. Thirty-one subjects entered the last phase of the study, 29 of whom were evaluable for response. This open label period evaluated two further doses of Ilaris for a minimum of 16 weeks. Clinical and biochemical remission of CAPS was sustained in 97 % of this population.

August 28, 2006

Kemia reported positive results from two phase I trials of KC706 for the treatment of chronic inflammation. A total of 79 healthy volunteers received oral administration of KC706 in three studies designed to evaluate the safety and pharmacokinetics of the drug. The initial escalating, single-dose study revealed that the compound was well tolerated. The second escalating, multiple-dose study demonstrated the safety of various dosages and determined the range of doses to be used in upcoming trials. The third phase I trial established the bioavailability of a capsule formulation of KC706 for use in future trials. Based on these results, Kemia planned to initiate a phase IIa study in Europe by the end of September 2006.

June 20, 2005

Regeneron Pharmaceuticals has issued preliminary results of a pilot study of IL-1 Trap, for the treatment of the autoimmune disorder CIAS1-associated periodic syndrome (CAPS). Results from the ongoing study indicated that the drug was efficacious in reducing levels of several inflammatory biomarkers, including C-reactive protein, erythrocyte sedimentation rate, and serum amyloid A protein. Furthermore, the drug produced significant improvements in daily patient diary symptom severity scores. Weekly dosing was seen to maintain favorable responses. This open-label pilots study has enrolled 4 subjects to date, who received a loading dose regimen of IL-1 Trap (100 mg per day for three days), followed by a drug free washout to investigate symptom recurrence, then followed resumption of a once weekly regimen of the drug. The company hopes that full results from this study will identify optimal dosing regimens for future investigations.

This information does not represent a Lupus Research Institute endorsement of any listed study. It is merely a notice that the study is available. If you are presently under the care of a physician for lupus or other conditions, you should not disrupt your current program without discussing it with your doctor(s). Do not contact the Lupus Research Institute for information on these studies. Only contact the listed numbers. The Lupus Research Institute does not have any jurisdiction over or further involvement with these studies, other than to make people aware that they are being conducted.