A clinical study to test the efficacy and safety of LDE225 compared to temozolomide in patients with a specific type of brain tumour

Phase 1

• Conditions: Relapsed medulloblastoma characterised by Hedgehog (Hh)-pathway activation; MedDRA version: 14.1 Level: PT Classification code 10066594 Term: Medulloblastoma recurrent System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps); Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2012-003066-40-GB
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-04-30
• Study Completion: 2016-10-05
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 22

Inclusion Criteria

- Patients aged >= 4 months
- Patients with histologically confirmed diagnosis of MB, who have experienced relapse or progression
- Only patients with a test result, using the 5-gene Hh signature assay, indicating Hh-pathway activated MB are eligible for this study.
- At least one measurable lesion
- Adequate renal function
- Adequate liver function
- Adequate bone marrow function
- Serum CK <= 1.5 ULN

Other protocol defined inclusion criteria may apply.
Are the trial subjects under 18? yes
Number of subjects for this age range: 48
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 59
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

- Prior treatment with a Smoothened inhibitor
- Systemic anticancer treatment within 2 weeks before first dose of study treatment
- Focal radiation therapy within 4 weeks before first dose of study treatment, or full spinal radiotherapy within 3 months before first dose of study treatment.
- Patients who have neuromuscular disorders that are associated with elevated CK
- Any concurrent severe and/or uncontrolled medical conditions that in the investigator’s opinion could put the patient at greater risk for treatment-related toxicities or confound the interpretation of clinical outcomes.
- Impaired cardiac function
- Clinically significant heart disease
- Known diagnosis of human immunodeficiency virus (HIV), Hepatitis B or C
- Impairment of GI function or GI disease
- Major surgery, serious illness, or traumatic injury within 2 weeks of first dose of study treatment.
- Unresolved toxicity greater than CTCAE grade 1 from previous anticancer therapy
- Patients anticipated to require major surgery within the first 8 weeks of treatment
- Patients who require a nasogastric tube for drug administration
- Patients on concomitant treatment with drugs that are recognized to cause rhabdomyolysis
- Patients receiving treatment with medications that are known to be strong inhibitors or inducers of CYP3A4/5 or are metabolized by CYP2B6 and CYP2C9.
- Patients receiving unstable or increasing doses of corticosteroids
- Patients receiving treatment with any enzyme-inducing anticonvulsant that cannot be discontinued at least 2 weeks before first dose of study treatment, and for the duration of the study
- Investigational agents within 4 weeks or = 5 x t1/2 (whichever is longer) before first dose of study treatment.
- Pregnant females.
- Patients who are not willing to apply highly effective contraception.
- Patients who are planning on embarking on a new strenuous exercise regimen after first dose of study treatment.

Other protocol defined exclusion criteria may apply.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified