Safety and efficacy of miltefosine alone and in combination with sodium stibogluconate and liposomal amphotericin B for the treatment of primary visceral leishmaniasis in East Africa: study protocol for a randomized controlled trial.
Background: Treatment options for visceral leishmaniasis (VL) in East Africa are far from satisfactory due to cost, toxicity, prolonged treatment duration or emergence of parasite resistance. Hence there is a need to explore alternative treatment protocols such as miltefosine alone or in combination...
| Publicado en: | Trials Vol. 12; no. 1; pp. 166 - 176 |
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| Autores principales: | , , , , , , , , , , , , , |
| Formato: | clinical trial research randomized controlled trial Journal Article |
| Publicado: |
BioMed Central
2011
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| Acceso en línea: | Ver este registro en EBSCOhost |
| fields | @attributes: recordID: 1 pdfLink: plink: https://search.ebscohost.com/login.aspx?direct=true&db=ccm&AN=65279579&site=ehost-live header: @attributes: shortDbName: ccm uiTerm: 65279579 longDbName: CINAHL Complete uiTag: AN controlInfo: bkinfo: dissinfo: jinfo: jid: 17456215 38OC jtl: Trials issn: 17456215 maglogo: N pubinfo: dt: 2011 vid: 12 iid: 1 pid: 24147 pub: BioMed Central artinfo: ui: 65279579 65279579 NLM21718522 65279579 10.1186/1745-6215-12-166 NLM21718522 PMC3155829 65279579 ppf: 166 ppct: 10 formats: tig: atl: Safety and efficacy of miltefosine alone and in combination with sodium stibogluconate and liposomal amphotericin B for the treatment of primary visceral leishmaniasis in East Africa: study protocol for a randomized controlled trial. aug: au: Omollo, Raymond Alexander, Neal Edwards, Tansy Khalil, Eltahir A G Younis, Brima M Abuzaid, Abuzaid A Wasunna, Monique Njoroge, Njenga Kinoti, Dedan Kirigi, George Dorlo, Thomas P C Ellis, Sally Balasegaram, Manica Musa, Ahmed M affil: Drugs for Neglected Diseases initiative (DNDi) Africa, Centre for Clinical Research, Kenya Medical Research Institute, Nairobi, Kenya sug: subj: Amphotericin B Therapeutic Use Study Design Leishmaniasis Drug Therapy Choline Antiprotozoal Agents Therapeutic Use Middle Age Leishmaniasis Diagnosis Antiprotozoal Agents Pharmacokinetics Antiprotozoal Agents Adverse Effects Time Factors Choline Pharmacokinetics Amphotericin B Adverse Effects Sudan Choline Therapeutic Use Treatment Outcomes Child Human Young Adult Kenya Adolescence Drug Therapy, Combination Adult Choline Adverse Effects Clinical Trials Validation Studies Comparative Studies Evaluation Research Multicenter Studies Randomized Controlled Trials Scales Middle Aged: 45-64 years Child: 6-12 years Adolescent: 13-18 years Adult: 19-44 years ab: Background: Treatment options for visceral leishmaniasis (VL) in East Africa are far from satisfactory due to cost, toxicity, prolonged treatment duration or emergence of parasite resistance. Hence there is a need to explore alternative treatment protocols such as miltefosine alone or in combinations including miltefosine, sodium stibogluconate (SSG) or liposomal amphotericin B. The aim of this trial is to identify regimen(s) which are sufficiently promising for future trials in East Africa.Methods/design: A phase II randomized, parallel arm, open-labelled trial is being conducted to assess the efficacy of each of the three regimens: liposomal amphotericin B with SSG, Liposomal amphotericin B with miltefosine and miltefosine alone. The primary endpoint is cure at day 28 with secondary endpoint at day 210 (6 months). Initial cure is a single composite measure based on parasitologic evaluation (bone marrow, spleen or lymph node aspirate) and clinical assessment. Repeated interim analyses have been planned after recruitment of 15 patients in each arm with a maximum sample size of 63 for each. These will follow group-sequential methods (the triangular test) to identify when a regimen is inadequate (<75% efficacy) or adequate (>90% efficacy). We describe a method to ensure consistency of the sequential analysis of day 28 cure with the non-sequential analysis of day 210 cure.Discussion: A regimen with adequate efficacy would be a candidate for treatment of VL with reasonable costs. The design allows repeated testing throughout the trial recruitment period while maintaining good statistical properties (Type I & II error rates) and reducing the expected sample sizes.Trial Registration: ClinicalTrials.gov: NCT01067443. pubtype: Academic Journal doctype: clinical trial research randomized controlled trial Journal Article ougenre: Article language: English refInfo: holdings: @attributes: islocal: N |
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