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Melanoma Metastasized to the Brain and Steroids

Registration Number
NCT03563729
Lead Sponsor
Inge Marie Svane
Brief Summary

This clinical trial is to clarify whether treatment with a checkpoint inhibitor alone (pembrolizumab) or two in combination (ipilimumab and nivolumab), results in clinical benefit for MM patients with brain metastases and in need of steroid treatment. Patients will be treated in four arms depending on steroid dose level at inclusion (\> 10 \< 25 mg prednisolone or \> 25 mg prednisolone) and treatment (pembrolizumab alone or the combination of ipilimumab and nivolumab).

Detailed Description

Cancer immunotherapy with checkpoint inhibitors (CPI) has demonstrated significant response rates, with clinical responses of exceptional duration observed in pivotal clinical trials for multiple types of solid tumors. Results from clinical trials demonstrate a considerable survival benefit of CPI over standard treatments, leading to registration of CPI for lung-, head and neck-, bladder-, renal cancer, lymphomas and metastatic melanoma (MM). To date, CPI appear to hold the key for longterm survival - at least for patients treated in clinical trials.

Patients enrolled in pivotal clinical trials for immunotherapy of MM are highly selected and does not include patients with brain metastases. Small phase II studies lend support to CPI to yield responses in melanoma that has metastasized to the brain. However, a large proportion of patients that develop brain metastasis will require continued systemic treatment with steroids to alleviate symptoms from the central nervous system (CNS). This group of patients are not offered treatment with CPI, as it is generally assumed that steroid treatment hamper their clinical efficacy. Thus, this group of patients face a large unmet need.

Due to the immune inhibiting effects, steroids are used to manage immune-related adverse events (irAEs) induced by CPI treatment. However, patients receiving steroids in this context are still able to achieve and maintain clinical benefit even after stopping treatment.

It is not known whether steroid treatment at the time of initiation of CPI treatment diminishes the treatment effect, as patients in need of steroid treatment are generally excluded from clinical trials.

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
80
Inclusion Criteria
  • Histologically confirmed metastatic melanoma with radiologically verified brain metastasis
  • Need for systemic steroid treatment (prednisolone > 10 mg daily; dexamethasone > 1.6 mg daily, hydrocortisone > 40 mg daily or equivalent) due to brain metastasis
  • At least one measurable lesion according to RECIST version 1.1 guidelines
  • Evaluable intracranial disease
  • 18 years of age or older
  • Performance status 0-2
  • Able to undergo MRI with gadolinium contrast agent
  • Adequate hematological and organ function
  • No significant toxicity from previous cancer treatments (CTC<1)
  • Women of childbearing potential: Negative serum pregnancy test and must use effective contraception. This applies from screening and until 6 months after treatment. Birth control pills, spiral, depot injection with gestagen, subdermal implantation, hormonal vaginal ring and transdermal depot patch are all considered effective contraceptives
  • Men with female partner of childbearing potential must use effective contraception from screening and until 6 months after treatment. Effective contraceptives are as described above for the female partner. In addition documented vasectomy and sterility or double barrier contraception are considered effective contraceptives
  • Signed statement of consent after receiving oral and written study information.
  • Willingness to participate in the planned treatment and follow-up and capable of handling toxicities.
  • For arm E specifically: Tumor cells must harbor BRAF mutation.
Exclusion Criteria
  • Another malignancy or concurrent malignancy unless disease-free for 3 years
  • Ocular melanoma
  • Neurological symptoms from brain metastases present at baseline despite steroid treatment, unless symptoms are related to prior surgery
  • Known hypersensitivity to one of the active drugs or excipients
  • Acute or chronic infections with HIV or hepatitis
  • Any medical condition that will interfere with patient compliance or safety
  • Prior treatment with anti-PD-1/PD-L1/PD-L2/CTLA-4 antibodies in the metastatic setting
  • Prior systemic treatment with anti-PD-1/PD-L1/PD-L2/CTLA-4 antibodies in the adjuvant setting, unless completed more than 6 months before enrolment in this study
  • Simultaneous treatment with other experimental drugs or other anti-cancer drugs
  • Pregnant or breastfeeding females.
  • For arm E specifically: Prior treatment with BRAF/MEK inhibitors.

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
D: Ipilimumab/nivolumab (Prednisolone >25 mg)Ipilimumab Injection [Yervoy]Intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
C: Ipilimumab/nivolumab (Prednisolone 11-25 mg)Nivolumab Injection [Opdivo]Intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
C: Ipilimumab/nivolumab (Prednisolone 11-25 mg)Ipilimumab Injection [Yervoy]Intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
D: Ipilimumab/nivolumab (Prednisolone >25 mg)Nivolumab Injection [Opdivo]Intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
B: Pembrolizumab (Prednisolone >10 mg)Pembrolizumab Injection [Keytruda]Intravenous infusion of pembrolizumab 2 mg/kg every third week for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)Ipilimumab Injection [Yervoy]Induction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)Nivolumab Injection [Opdivo]Induction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)EncorafenibInduction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)TrametinibInduction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)BinimetinibInduction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
E: BRAF/MEK -> ipi/nivo (prednisolone >10 mg)DabrafenibInduction treatment with BRAF/MEK inhibitors (either the combination of encorafenib/binimetinib or dabrafenib/trametinib) orally for 28 days followed by intravenous infusion of ipilimumab 3 mg/kg and nivolumab 1 mg/kg four times every three weeks in the induction phase and nivolumab 480 mg every four weeks in the maintenance phase for up to two years.
Primary Outcome Measures
NameTimeMethod
6 months progression-free survival rate6 months

Proportion of patients who did not progress or die within 6 months from commencing study treatment.

6 months overall survival rate6 months

Proportion of patients who did not die within 6 months from commencing study treatment.

Secondary Outcome Measures
NameTimeMethod
Overall response rate4 years

Proportion of patients with an overall complete or partial response according to modified RECIST 1.1.

Blood and tissue biomarkers of response and progression5 years

Correlation of the baseline PD-L1 status, immune markers, genomics and other biomarkers in tumour tissue and blood with complete or partial response and at subsequent disease progressionanalyses of potential specific biomarkers predictive of response or progression.

Overall progression-free survival4 years

Time from commencing study treatment to the date of progression or death.

Intracranial response rate4 years

Proportion of patients with an overall complete or partial response in intracranial lesions according to modified RECIST 1.1.

Extracranial response rate4 years

Proportion of patients with an overall complete or partial response in extracranial lesions according to modified RECIST 1.1.

Intracranial clinical benefit rate4 years

Proportion of patients with an overall complete, partial response or stable disease \> 6 months according to modified RECIST 1.1.

Overall survival4 years

Time from commencing study treatment to the date of death from any cause.

Trial Locations

Locations (3)

Herlev Universityhospital

🇩🇰

Herlev, Hovedstaden, Denmark

Aarhus Universityhospital

🇩🇰

Aarhus, Midt, Denmark

Odense Universityhospital

🇩🇰

Odense, Syd, Denmark

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