Pembrolizumab in the Treatment of Advanced, Progressive Adrenocortical Carcinoma. (PEMBR-01)
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|ClinicalTrials.gov Identifier: NCT05563467|
Recruitment Status : Not yet recruiting
First Posted : October 3, 2022
Last Update Posted : October 3, 2022
This study is a national, multicenter, interventional, phase II clinical trial on the use of pembrolizumab in advanced adrenocortical carcinoma, with confirmed progression within 6 months, following EDP or EDP-M ( etoposide, doxorubicin, cisplatin- mitotan) chemotherapy.
Adrenocortical carcinoma is a very rare entity with poor prognosis and limited therapeutic options. Only radical surgical treatment of the early stages gives a chance for complete cure, however the risk of recurrence still remains high.
The results of clinical trials conducted outside Poland indicate a possible potential role of immunotherapy as a rescue treatment for adrenocortical carcinoma after standard therapeutic methods have been exhausted.
This study will evaluate the efficacy and tolerability of treatment with the immune checkpoint inhibitor pembrolizumab in locally advanced, non-operable or metastatic adrenocortical carcinoma after first line chemotherapy failure.
The study population will include adult patients (>18 years of age) with histopathologically confirmed adrenocortical carcinoma and confirmed progression according to RECIST 1.1 within 6 months, after first line chemotherapy with the EDP and EDP-M scheme. Patients must meet the inclusion criteria and must not meet the exclusion criteria described in the PEMBR-01 study protocol. The planned number of patients in the study is 24.
The treatment regimen will be based on Pembrolizumab administered intravenously in 3 weeks cycles at a dose of 200mg. For hormonally active tumors producing cortisol, it is hypothesized that the use of pembrolizumab in combination with effective steroidogenesis inhibition may enhance the effect of immunotherapy. In the study, metyrapone or ketoconazole will be used for this purpose.
The primary endpoint of the study will be the objective response rate to the treatment. The secondary endpoints will be progression-free survival, duration of response, overall survival, and treatment safety as well as the effect of therapy on patients' quality of life.
Concurrently, the analysis of biomarkers in tumor tissue will be carried out, including tumour infiltrating lymphocytes, expression of programmed death ligand, microsatellite instability and tumour mutation burden.
|Condition or disease||Intervention/treatment||Phase|
|Adrenocortical Carcinoma||Drug: Pembrolizumab 25 MG/ML [Keytruda]||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Multicentre, Open-label Phase 2 Study to Evaluate the Efficacy and Safety of Pembrolizumab in the Treatment of Advanced, Progressive Adrenocortical Carcinoma.|
|Estimated Study Start Date :||December 1, 2022|
|Estimated Primary Completion Date :||May 31, 2027|
|Estimated Study Completion Date :||May 31, 2027|
|Experimental: Pembrolizumab Treatment||
Drug: Pembrolizumab 25 MG/ML [Keytruda]
The treatment scheme will be based on pembrolizumab administered intravenously in cycles of every 3 weeks 200 mg, with supportive treatment with steroidogenesis inhibitor (metyrapone or ketoconazole) for adrenocortical carcinoma producing cortisol.
- Objective Response Rate [ Time Frame: through study completion, an average of 4 years ]Percentage of patients who achieved a partial (PR) or complete (CR) response to treatment.
- Progression-Free Survival (PFS) [ Time Frame: through study completion, an average of 4 years ]Time from first dose of treatment until disease progression or death from any cause.
- Duration of Response (DoR) [ Time Frame: through study completion, an average of 4 years ]Duration of response to treatment (from the start of CR or PR) until disease progression or death from any cause.
- Overall Survival (OS) [ Time Frame: through study completion, an average of 4 years ]Time from initiating treatment to death from any cause.
- Number of Adverse Events and Serious Adverse Events (AE and SAE) [ Time Frame: through study completion, an average of 4 years ]Number of Adverse Events and Serious Adverse Events (AE and SAE) related and not related to treatment according to CTCAE.
- Quality of life assessed based on the QLQ-C30 questionnaire. [ Time Frame: through study completion, an average of 4 years ]Quality of life assessed based on the QLQ-C30 questionnaire in which patients will respond during the clinical trial.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05563467
|Contact: Barbara Ziółkowska, MD,PhD||32-278-88-22 ext firstname.lastname@example.org|
|Contact: Agnieszka Kotecka-Blicharz, MD|