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Secondary Efficacy Endpoints (secondary + efficacy_endpoint)
Selected AbstractsEfficacy and safety of pimecrolimus cream 1% in adult patients with vitiligo: Results of a randomized, double-blind, vehicle-controlled studyJOURNAL DER DEUTSCHEN DERMATOLOGISCHEN GESELLSCHAFT, Issue 11 2006Markus Dawid Pimecrolimus; Elidel®; Vitiligo Summary Background: Vitiligo is an acquired, pigmentary skin disorder which is disfiguring and difficult to treat. In an earlier open label study in adult patients with vitiligo, pimecrolimus cream 1% was reported to have similar efficacy as clobetasol propionate 0.05%. We performed a double-blind, intrapatient comparison of pimecrolimus cream 1% with placebo cream. Patients and methods: Twenty adult Caucasians with symmetrical vitiligo (predominantly on extremities, none in the face) were treated b.i.d. for 6 months left/right with pimecrolimus/vehicle (N = 10) or vehicle/pimecrolimus (N = 10), respectively. Primary efficacy endpoint was the size of the target lesion at month 6 and secondary efficacy endpoint was re-pigmentation. Results: Treatment with pimecrolimus cream 1% or vehicle resulted in no significant change in mean target lesion size. Modest repigmentation (1,25%) was noted with pimecrolimus at month 2 in 12 of 17 patients (vehicle: 9 of 17 patients). Afterwards, the number of patients who experienced an improvement of pigmentation steadily decreased (3 of 14 patients with pimecrolimus and 2 of 14 with placebo at month 6).Treatment was well tolerated. There were no treatment-related adverse events, no induction of skin atrophy nor any other application site side effects. Conclusion: In this group of adult patients with symmetrical vitiligo, treatment of body lesions (except face) with pimecrolimus cream 1% could not be shown to be effective. Zusammenfassung Hintergrund: Vitiligo ist eine erworbene Pigmentstörung der Haut, die entstellend und schwer zu behandeln ist. In einer früheren offenen Studie an erwachsenen Patienten mit Vitiligo wurde mit Pimecrolimus-Creme 1% eine ähnliche Wirksamkeit beobachtet wie mit Clobetasol propionat 0.05%. Bei der vorliegenden Studie handelte es sich um einen doppelblinden, intraindividuellen Vergleich von Pimecrolimus-Creme 1% und Plazebo-Creme. Patienten und Methodik: Zwanzig erwachsene Patienten weißer Hautfarbe mit symmetrischer Vitiligo (vorwiegend an den Extremitäten) wurden zweimal täglich über 6 Monate im Halbseitenvergleich mit Pimecrolimus/Cremegrundlage (N = 10) oder Cremegrundlage/Pimecrolimus (N = 10) behandelt. Primärer Endpunkt war die Größe der behandelten Läsion, sekundärer Endpunkt der Prozentsatz an Repigmentierung. Ergebnisse: Die Behandlung mit Pimecrolimus-Creme 1% oder Vehikel ergab keine signifikante ,nderung der durchschnittlichen Größe der Läsion. Eine mäßige (1,25%) Repigmentierung wurde mit Pimecrolimus bei 12 von 17 (Grundlage:9 von 17) Patienten in Monat 2 beobachtet. Anschließend nahm die Anzahl der Patienten, die eine Repigmentierung aufwiesen, kontinuierlich ab (3 von 14 unter Pimecrolimus, 2 von 14 unter Grundlage im Monat 6). Die Behand-lung wurde gut vertragen. Es gab keine behandlungsbedingten Zwischenfälle und keine Induktion von Hautatrophie oder irgendwelche anderen Nebenwirkungen an der Applikationsstelle. Schlussfolgerung: In dieser Gruppe erwachsener Patienten mit symmetrischer Vitiligo konnte eine Wirksamkeit der Behandlung von Läsionen am Körper (nicht Gesicht) mit Pimecrolimus-Creme nicht gezeigt werden. [source] A real-life multicentre clinical practice study to evaluate the efficacy and safety of intravesical chondroitin sulphate for the treatment of interstitial cystitisBJU INTERNATIONAL, Issue 1 2009J. Curtis Nickel OBJECTIVE To report a multicentre, community based open-label study designed to assess the efficacy and safety of intravesical sodium chondroitin sulphate in the treatment of patients with the clinical diagnosis of interstitial cystitis (IC). Chondroitin sulphate is a naturally occurring glycosaminoglycan (GAG) in the bladder mucus layer and changes in this GAG have been implicated in the pathogenesis of IC, and small single-centre studies have suggested that intravesical chondroitin sulphate may have efficacy in IC. PATIENTS AND METHODS Patients with IC were treated with sodium chondroitin sulphate (Uracyst®, Stellar Pharmaceuticals Inc., London ON, Canada) solution 2.0% via urinary catheter weekly for 6 weeks and then monthly for 16 weeks for a total of 10 treatments. The primary efficacy endpoint was the percentage of responders to treatment as indicated by a marked or moderate improvement on a seven-point patient Global Response Assessment (GRA) scale at week 10 (4 weeks after the initial six treatments) compared with baseline. A major secondary efficacy endpoint (durability) was the percentage of responders on the GRA scale after 10 treatments. Additional secondary efficacy objectives were differences from baseline in Patient Symptom/Problem Index scores over the course of the treatment compared with baseline. RESULTS In all, 47% of the 53 enrolled patients with long standing moderately severe IC (mean [sd, range] diagnosis of IC 3.0 [3.4, 0.1,16] years; duration of symptoms 9.2 [9.2, 1,39] years; baseline symptom score 14.2 [3.2]) were responders at week 10. At 24 weeks, 60% were responders. There was a statistically and clinically significant decrease in the mean (sd) symptom and bother scores from baseline at 10 weeks and 24 weeks, at 9.0 (4.3) and 8.1 (5.0), respectively (P < 0.001). There were no significant safety issues during the study. CONCLUSIONS This multicentre community based real-life clinical practice study suggests that intravesical chondroitin sulphate may have an important role in the treatment of IC and validates the rationale for a randomized placebo-controlled trial. [source] Sumatriptan Nasal Spray in Adolescent Migraineurs: A Randomized, Double-Blind, Placebo-Controlled, Acute StudyHEADACHE, Issue 2 2006Paul Winner DO Objective.,To compare the efficacy and tolerability of sumatriptan nasal spray (NS) (5, 20 mg) versus placebo in the acute treatment of migraine in adolescent subjects. Background.,Currently, no triptan is approved in the United States for the treatment of migraine in adolescent subjects (12 to 17 years). In a previous randomized, placebo-controlled study of 510 adolescent subjects, sumatriptan NS at 5, 10, and 20 mg doses was well tolerated. However, the primary efficacy analysis for headache relief with 20 mg at 2 hours did not demonstrate statistical significance (P= .059). A second study was initiated to evaluate the efficacy of sumatriptan NS in this population. Methods.,This was a randomized (1:1:1), placebo-controlled, double-blind, parallel-group study. Overall, 738 adolescent subjects (mean age: 14 years) with ,6-month history of migraine (with or without aura) self-treated a single attack of moderate or severe migraine. The primary endpoints were headache relief at 1 hour and sustained relief from 1 to 24 hours. Pain-free rates, presence/absence of associated symptoms, headache recurrence, and use of rescue medications were also assessed. Tolerability was based on adverse events (AEs) and vital signs. Results.,Sumatriptan NS 20 mg provided greater headache relief than placebo at 30 minutes (42% vs. 33%, respectively; P= .046) and 2 hours (68% vs. 58%; P= .025) postdose, but did not reach statistical significance at 1 hour (61% vs. 52%; P= .087) or for sustained headache relief from 1 to 24 hours (P= .061). Significant differences (P < .05) in favor of sumatriptan NS 20 mg over placebo were observed for several secondary efficacy endpoints including sustained relief from 2 to 24 hours. In general, sumatriptan NS 5 mg percentages were slightly higher than placebo but the differences did not reach statistical significance. Both doses of sumatriptan NS were well tolerated. No AEs were serious or led to study withdrawal. The most common event was taste disturbance (2%, placebo; 19%, sumatriptan NS 5 mg; 25%, sumatriptan NS 20 mg). Conclusions.,This study suggests that sumatriptan may be beneficial to some adolescents and is generally well tolerated in the acute treatment of migraine in this population. [source] Lamotrigine in the acute treatment of bipolar depression: results of five double-blind, placebo-controlled clinical trialsBIPOLAR DISORDERS, Issue 2 2008Joseph R Calabrese Objectives:, The efficacy of lamotrigine as maintenance treatment for bipolar disorder (BD), particularly for delaying depressive episodes, is well established, but its efficacy in the acute treatment of bipolar depression is less clear. This paper reports the results of five randomized, double-blind, placebo-controlled trials of lamotrigine monotherapy for the acute treatment of bipolar depression. Methods:, Adult subjects with bipolar I or II disorder experiencing a depressive episode were randomized to placebo or lamotrigine monotherapy (after titration, at a fixed dose of 50 mg or 200 mg daily in Study 1; a flexible dose of 100,400 mg daily in Study 2; or a fixed dose of 200 mg daily in Studies 3, 4 and 5) for 7,10 weeks. Results:, Lamotrigine did not differ significantly from placebo on primary efficacy endpoints [17-item Hamilton Depression Rating Scale in Studies 1 and 2; Montgomery,Asberg Depression Rating Scale (MADRS) in Studies 3, 4 and 5]. In Study 1, lamotrigine significantly separated from placebo on some secondary measures of efficacy, including the MADRS, the Clinical Global Impressions-Severity (CGI-S) and the CGI-Improvement (CGI-I), but seldom differed on secondary efficacy endpoints for the other studies. Conclusions:, Lamotrigine monotherapy did not demonstrate efficacy in the acute treatment of bipolar depression in four out of five placebo-controlled clinical studies. Lamotrigine was well tolerated in the acute treatment of bipolar depression. [source] Topiramate in painful diabetic polyneuropathy: findings from three double-blind placebo-controlled trialsACTA NEUROLOGICA SCANDINAVICA, Issue 4 2004The Topiramate Diabetic Neuropathic Pain Study Group Objectives , To evaluate the efficacy and tolerability of topiramate in patients with painful diabetic polyneuropathy. Materials and methods , Patients with moderate to extreme pain (0,4 Categorical Pain Scale score , 2) were randomized to placebo or topiramate (100, 200, or 400 mg/day) in three similar double-blind trials. The primary efficacy analysis was pain reduction from final visit to baseline in the 100-mm Visual Analog Scale (VAS) for the intent-to-treat populations. Results , After 18,22 weeks of double-blind treatment, pain reductions were numerically greater with topiramate in two studies but differences between topiramate and placebo in VAS scores or in the secondary efficacy endpoints did not reach statistical significance in any of the three studies. Across all studies, 24% of topiramate-treated patients and 8% of placebo-treated patients discontinued due to adverse events; groups did not differ in the occurrence of serious adverse events. Conclusion , These studies did not find topiramate to be significantly more effective than placebo in reducing pain scores in patients with painful diabetic polyneuropathy. Several design features may have precluded the studies from having sufficient sensitivity to differentiate effective and ineffective treatments. The study design and results are instructive for other investigators designing future clinical studies in neuropathic pain. [source] | ||||||||||