Clinical trial NCT01412515: A Phase II Study With Tumor Molecular Pharmacodynamic (MPD) Evaluation of Oral mTOR-inhibitor Everolimus (RAD001) 10 mg Daily in Patients Suffering From Classic or Endemic Kaposi's Sarcoma
Official title: A Phase II Study With Tumor Molecular Pharmacodynamic (MPD) Evaluation of Oral mTOR-inhibitor Everolimus (RAD001) 10 mg Daily in Patients Suffering From Classic or Endemic Kaposi's Sarcoma
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Kaposi Sarcoma
Keywords: mTOR inhibitors
Lead sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Sponsor class: OTHER_GOV
First posted: 2011-08-09
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: Classic Kaposi's sarcoma (CKS) is an angioproliferation associated with human herpes virus 8 (HHV8), which sometimes requires systemic treatment. Rapamycin and everolimus are mTOR inhibitors. The PI3K-AKT-mTOR pathway is activated in CKS.The aim of this study is to evaluate the rate of clinical response and tolerance to everolimus 10mg/d in CKS. Patients suffering from CKS will be enrolled in a multicenter two-stage phase II trial. At inclusion, all patients will have at least 10 lesions or more than one limb or 3% of body surface affected, in the absence of symptomatic visceral CKS. The primary endpoint is objective response to everolimus after 6 months therapy (complete or partial response per ACTG criteria). The trial is planned using Simon's minimax two-stage design to demonstrate a response rate of 50% as compared to 20% with type I error rate 2.5% and power of 90%. Accordingly, 11 patients will to be enrolled in the first stage and provided at least 3 patients responded, 15 patients will be accrued in a second stage.

Detailed description: The primary endpoint is objective response to everolimus after 6 months therapy (complete or partial response per ACTG criteria).

Secondary endpoints are response according to physician global assessment, lesions size , lesions infiltration, lymphedema, tolerance, pharmacodynamic (pathologic, angiogenic and lymphangiogenic biomarkers, HHV8 viral load sequential evaluation in the lesions, pharmacokinetic evaluation.

Eligibility criteria: Inclusion Criteria:

Histologic confirmed classic or endemic KS (non transplant non HIV patients) \> 18 Years old at least 10 lesions or more than one limb or 3% of body surface affected, in the absence of symptomatic visceral KS Presence of at least 4 targets lesions \> 5mm At least 4 weeks wash out from any KS specific therapy ECOG \< 2

Exclusion Criteria:

immunosupressive regimen patients HIV positive creatinine clearance \<40ml/mn,AST ALT\>3N, neutropénia\<1500, thrombopenia\<150000, anemia\<8g/dL

Primary outcomes:
measure: objective response to everolimus after 6 months therapy
timeFrame: duration of study 30 months
Secondary outcomes:
measure: response according to physician global assessment, lesions size , lesions infiltration
timeFrame: 30 months
Other outcomes:
Locations:
city: Paris
country: France
facility: Hospital Saint Louis
Study officials:
affiliation: Institut National de la Santé Et de la Recherche Médicale, France
name: Celeste Lebbé, MD, PhD
role: STUDY_DIRECTOR
References: