Study for patients with relapsed, refractory, incurable teratoma with recent progression. The study is randomized (the patient may receive the investigational drug or placebo) and it is blinded (the patient and the study doctor will not know what drug the patient receives).

• Register: EUCTR
• Last refreshed on: 18 January 2016
• Main ID: EUCTR2014-000428-12-DK
• Date of registration: 19/03/2015
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharma Services AG
• Public title: Study for patients with relapsed, refractory, incurable teratoma with recent progression. The study is randomized (the patient may receive the investigational drug or placebo) and it is blinded (the patient and the study doctor will not know what drug the patient receives).
• Scientific title: A randomized, blinded, placebo-controlled, phase II trial of LEE011 in patients with relapsed, refractory, incurable teratoma with recent progression
• Date of first enrolment: 20/05/2015
• Target sample size: 42
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000428-12
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Denmark, France, Italy, Netherlands, Spain, United Kingdom, United States

Contacts

• Name: Medicinsk information
• Affiliation: Novartis Healthcare A/S

• Name: Medicinsk information
• Affiliation: Novartis Healthcare A/S

Key inclusion & exclusion criteria

Inclusion criteria:
1. Diagnosis of teratoma for which no additional standard surgical or medical therapy
exists
2. Patients must have completed at least 1 prior line of chemotherapy for germ cell tumor
3. Radiographic progression, defined by RECIST v.1.1, after the last cancer treatment and within 12 weeks
prior to enrollment, compared with scans within 1 year of enrollment.
4. Availability of an archival or newly obtained tumor sample (collected at diagnosis or progression) with accompanying pathology report
5. Measurable or evaluable extra-cranial disease as defined by RECIST v 1.1

Other protocol-defined inclusion criteria may apply
Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
1. Malignant germ cell tumors with mixed histology such as embryonal carcinoma, choriocarcinoma, yolk sac tumor or
seminoma
2. Pathologic evidence of malignant transformation
3. CNS disease unless radiation therapy and/or surgery has been completed and serial evaluation demonstrates stable disease
4. Prior treatment with any CDK4/6 inhibitor therapy
5. Systemic antineoplastic therapy or any experimental therapy within 3 weeks before the first dose of study drug (6 weeks for prior nitrosoureas, bevacizumab, or mitomycin C)
6. Major surgery = 2 weeks or radiotherapy = 4 weeks prior to planned start of study drug or patient has not
recovered from major side effects.
7. Requirement for treatment with any of the prohibited medications including strong CYP3A inhibitors, strong CYP3A inducers, CYP3A substrates with a narrow therapeutic index, and medications with strong risk of QT prolongation.

Other protocol-defined Exclusion criteria may apply


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Cancer [C04]

relapsed, refractory, incurable teratoma
MedDRA version: 18.0 Level: PT Classification code 10043276 Term: Teratoma System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)

Intervention(s)

Product Name: LEE011
Product Code: LEE011
Pharmaceutical Form: Capsule
INN or Proposed INN: ribociclib
Current Sponsor code: LEE011
Other descriptive name: LEE011
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: To assess the efficacy of LEE011 compared to placebo in patients with relapsed/refractory teratoma with recent progression

Timepoint(s) of evaluation of this end point: At 4 months

Primary end point(s): Progression Free Survival (PFS) as per RECIST v1.1 (by local investigator assessment)

Secondary Objective: 1.To assess other measures of efficacy of LEE011 compared with placebo
2.To assess safety and tolerability of LEE011 compared with placebo

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 1. At 4 months (BOR, ORR, DOR, DCR) or 12 months (OS, OS rate)
2. Study duration

Secondary end point(s): 1. Best Overall Response (BOR),Overall response rate (ORR), Duration of Response (DOR), Disease Control Rate (DCR) as per RECIST v1.1, Overall Survival (OS) and OS rate

2. Incidence and severity of adverse events and serious adverse events, changes in laboratory values, and electrocardiograms will be used to assess the safety as per CTCAE v.4.03. Dose interruptions and changes will be used to assess the tolerability.

Secondary ID(s)

2014-000428-12-ES

NCT02300987

CLEE011X2201

Source(s) of Monetary Support

Novartis Pharma Services AG