Clinical Trial: A Phase II Study of Oral LDE225 in Patients With Hedge-Hog (Hh)-Pathway Activated Relapsed Medulloblastoma (MB)

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase II, Multi-center, Open-label, Single-arm Study of the Efficacy and Safety of Oral LDE225 in Patients With Hh-pathway Activated Relapsed Medulloblastoma

Brief Summary: This Phase II study evaluates the safety and efficacy of LDE225 in adult and pediatric patients with Hh-pathway activated, relapsed MB.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Overall response rate (ORR) [ Time Frame: 8 weeks ]

ORR is defined as the proportion of patients with best overall response of complete response (CR) or partial response (PR). (As per Tumor response guidelines and criteria for Medulloblastoma.

ORR will be done by independent central review.



Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Progression free survival (PFS) [ Time Frame: 8 weeks ]
    PFS is defined as the time from date of randomization to the date of event defined as the first documented progression or death due to any cause
  • Overall response rate (ORR) [ Time Frame: 8 weeks ]
    As per Tumor response guidelines and criteria for Medulloblastoma. ORR will be done by local investigator assessment.
  • Duration of response (DoR) [ Time Frame: 8 weeks ]
    DoR is defined as the time from the first documented onset of confirmed PR or CR to the date of PD/relapse or death due to medulloblastoma.
  • Overall survival (OS) [ Time Frame: 4 weeks ]
    OS is defined as the time from date of randomization to date of death due to any cause.
  • Safety and tolerability of LDE225 treatment [ Time Frame: 4 weeks ]
    Adverse and serious adverse events, clinically significant changes in hematology and chemistry values, assessment of physical and/or neurological examinations, vital signs, electrocardiograms, bone x-rays, dental x-rays (e.g., panorex or age appropriate dental x-ray), and dental exams (as appropriate for age)
  • Pharmacokinetics (Pk) - Cmin [ Time Frame: 2 weeks ]
    PK of LDE225 and any relevant metabolites


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: October 11, 2012
Date Started: May 2013
Date Completion: October 2016
Last Updated: October 7, 2016
Last Verified: October 2016