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Phase I-II Study With Tumor Molecular Pharmacodynamic (MPD) Evaluation and Pharmacokinetics of PD-0332991 in Patients Suffering Metastatic Melanoma

Phase 1
Conditions
Melanoma BRAF V600E/K Mutated
CDNKN2A Loss Defined
Interventions
Drug: PD- 0332991
Registration Number
NCT02202200
Lead Sponsor
Assistance Publique - Hôpitaux de Paris
Brief Summary

An open label multicentre, phase I-II study with tumour molecular pharmacodynamics (MPD) evaluation and pharmacokinetics of PD-0332991 added to vemurafenib in patients suffering metastatic melanoma with BR.

The main objective is to establish the Maximum Tolerated Dose (MTD) of PD-0332991 when added to standard vemurafenib therapy (960 mg BID). The estimated MTD is defined as the dose of PD-0332991 combined with vemurafenib that will be associated with a prespecified proportion of patients experiencing a Dose-Limiting Toxicity (DLT), ie, 1/3.

Detailed Description

Not available

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
40
Inclusion Criteria
  • Age > 18 years
  • Stage IV or un-resectable stage III melanoma
  • Presence of BRAF V600E/K mutation and CDNKN2A loss and expression of Rb using immunohistochemistry in a recent metastatic sample (< 6 months)
  • A previous exposure to BRAF inhibitor or combination of BRAF and MEK inhibitors therapy is allowed unless it has been stopped more than 3 months before study enrolment(This will defined the two strata of the trial)
  • No previous therapy by MEK inhibitor unless associated with BRAF inhibitors
  • No previous therapy with the AKT/PI3K pathway inhibitor
  • Patients should have a tumour available for repeated biopsies for pharmacodynamics evaluation
  • Life expectancy of > 3 months
  • ECOG performance status <2
  • Signed informed consent
  • Patient with health insurance coverage
  • No patient under guardianship or curators
Exclusion Criteria
  • Inadequate hepatic function defined as serum bilirubin>25 μmol/l, transaminases > 3.0 times the upper limit of normal (ULN) or 5ULN in cases of liver metastases;
  • Inadequate bone marrow function defined as absolute neutrophil count<1500/mcl, platelets<150000/mcl and haemoglobin<8g/dL
  • Inadequate renal function with serum creatinine>2.0mg/dl) and /or creatinine clearance< 60 ml/min
  • Untreated brain metastases : Patients with brain metastases will be eligible if they have completed treatment 1 months prior to the start of study medication, have discontinued corticosteroid treatment for these metastases for at least 5 days, and are neurologically asymptomatic
  • Myocardial infarct or unstable angina within the past 6 months
  • Concomitant take of drugs known to be strong inhibitor or inducers of CYP314
  • HIV positive.
  • Chemotherapy, immunotherapy within 4 weeks
  • Drugs interfering with PD-0332991 and vemurafenib metabolism
  • Malabsorption syndrome or other condition that would interfere with enteral absorption
  • Congenital long QT syndrome or screening QTc > 470 msec
  • Need for chronic corticosteroid therapy of ≥10 mg of prednisone per day

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
PD-0332991PD- 0332991-
Primary Outcome Measures
NameTimeMethod
Occurrence within the first 2 cycles of treatment of a DLT42 Days

DLTs, serious adverse events and adverse events leading to treatment discontinuations will be determined as follows:

* Any grade 3 or more non-haematological toxicity excluding:

* Grade 3 asymptomatic increase in liver function tests (AST, ALT, ALP) reversible within 7 days for subjects without liver involvement, or grade 4 for subjects with liver involvement.

* Grade 3 vomiting if it is encountered despite adequate and optimal therapy (e.g. 5HT3 antagonists and corticosteroids).

* Grade 3 diarrhoeas if encountered despite adequate and optimal anti diarrhoea therapy.

* Confirmed grade 3 QTc prolongation (QTc \>500 msec) that persists after correction of other possible causes such as electrolyte imbalance

* Grade 4 hyperlipidemia if it is encountered despite adequate and optimal therapy.

* Any grade 4 neutropenia of \> 5 days duration, or febrile neutropenia lasting for more than 1 day.

* Grade 4 thrombocytopenia \> 1 day, or grade 3 with bleeding.

Secondary Outcome Measures
NameTimeMethod
Tolerance6 months

Occurrence of clinically significant changes in a laboratory parameter and/or vital signs judged to be related to the trial medication within the first 6 months.

Efficacy42 Days

Occurrence of clinical benefit (defined as Complete Response (CR), Partial Response (PR) or stable disease (SD)) and progressive disease based on the best overall response which depends on the tumour evaluations assessed using RECIST at the end of each 2 cycles.

1 year survival rate1 year

survival

Trial Locations

Locations (1)

Saint-Louis Hospital

🇫🇷

Paris, France

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