MedPath

RAMSETE: RAD001 in Advanced and Metastatic Silent Neuro-endocrine Tumors in Europe

Phase 2
Completed
Conditions
Advanced and Metastatic Silent Neuro-Endocrine Tumors
Neuroendocrine
Non Functioning Neuroendocrine Tumors (NETs)
Non Syndromic Neuroendocrine Tumors
Carcinoma
Carcinoids
Non Functioning
Interventions
Registration Number
NCT00688623
Lead Sponsor
Novartis Pharmaceuticals
Brief Summary

To evaluate the preliminary efficacy and safety of RAD001 as monotherapy for first-line treatment of patients with metastatic papillary carcinoma of the kidney.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
73
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
EverolimusEverolimusEverolimus
Primary Outcome Measures
NameTimeMethod
Percentage of Participants' Best Overall Response Rate at 12 Months - Per Protocol Set (PP)baseline up to approximately 12 months

Overall response rate (ORR) was based on RECIST central assessment and defined as the percentage of patients with best overall response (BOR) of a confirmed complete response (CR) or partial response (PR). The BOR was calculated on basis of the tumor of overall lesion response evaluated at each visit. To be assigned a status of PR or CR, changes in tumor measurements had to be confirmed by repeat assessments obtained within 4 weeks after the criteria for response were first met. Assessments was based on RECIST criteria 1.0. Measurable disease lesions had to be accurately measured in at least one dimension with longest diameter ≥ 20 mm using conventional techniques or ≥ 10 mm with spiral CT scan (with minimum lesion size no less than double the slice thickness). PR required at least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters. CR required disappearance of all target and non-target lesions.

Percentage of Participants With Objective Response Rate at 12 Months - Per Protocol Set (PP)baseline up to approximately 12 months

Overall Response Rate (ORR) was calculated for total PP population based on central review as confirmatory, primary analysis as well as for ITT population as sensitivity analysis. It was presented with relative frequencies and the exact 2-sided 80% confidence limit (CI) computed using the Clopper-Pearson method). If the lower limit of the CI did not include p0=5%, the hypothesis that p ≤ 5% was rejected. The primary analysis was based on the PP Set

Percentage of Participants With a Overall Response Rate With a Complete Response (CR) or Partial Response (PR) at 12 Months ITT Setbaseline up to approximately 12 months

The best overall response (BOR) was calculated on basis of the tumor of overall lesion response evaluated at each visit. To be assigned a status of PR or CR, changes in tumor measurements had to be confirmed by repeat assessments that should have been performed not less than 4 weeks after the criteria for response were first met. Assessments was based on RECIST criteria 1.0. Measurable disease lesions had to be accurately measured in at least one dimension with longest diameter ≥ 20 mm using conventional techniques or ≥ 10 mm with spiral CT scan (with minimum lesion size no less than double the slice thickness). PR required at least a 30% decrease in the sum of the longest diameter of all target lesions, taking as reference the baseline sum of the longest diameters.

CR required disappearance of all target and non-target lesions.

Percentage of Participants With Objective Response Rate at 12 Months ITT Setbaseline up to approximately 12 months

Overall Response Rate (ORR) was presented for ITT population as sensitivity analysis. It was presented with relative frequencies and the exact 2-sided 80% confidence limit (CL; computed using the Clopper-Pearson method). If the lower limit of the CI did not include p0=5%, the hypothesis that p ≤ 5% was rejected.

Secondary Outcome Measures
NameTimeMethod
Percentage of Participants With Disease Control Rate (DCR) at 12 Months for Per Protocol (PP) and ITT Setsbaseline up to approximately 12 months

DCR was based on central radiologic review and is defined as the percentage of patients with a best overall response of 'Complete response' (CR), 'Partial response' (PR) or 'Stable disease' (SD). Relative frequencies together with their exact 2-sided 80% confidence intervals were presented

Percentage of Participants' Biochemical Response Rate Based on the Tumor Marker Chromogranin A (CgA)baseline up to approximately 12 months

Biochemical response was defined as level and change from baseline in CgA during the course of the trial. The resulting values showed a high variation and were not interpretable, as different methodology was used for the assessment of CgA at the individual centers.

Duration of Progression Free Survival (PFS) for Per Protocol (PP) and ITT Setsbaseline up to approximately 12 months

Duration of PFS was defined as the time from first study drug administration to objective tumor progression or death from any cause. Observations from patients not experiencing tumor progression or death at date of database closure were censored with the date of their last adequate tumor assessment. Progression was either 1) a 20% increase in the sum of the longest diameter of all target lesions, taking as reference the smallest sum of longest diameter of all target lesions recorded at or after baseline or 2) the appearance of a new lesion or 3) the unequivocal progression of non-target lesions.

Overall Survival (OS) for Per Protocol (PP) and ITT Setsbaseline up to approximately 15 months

OS was defined as the time from first study drug administration to death from any cause. If a patient was not known to have died at date of database closure, overall survival was censored at the date of last contact.

Trial Locations

Locations (1)

Novartis Investigative Site

🇬🇧

Manchester, United Kingdom

© Copyright 2025. All Rights Reserved by MedPath