Phase II open-label single-arm prospective multicentric clinical trial of PF-05212384 (PKI-587) delivered by intravenous route. A 2-stage Fleming design will be employed.
The treatment is administered in cycles of 28 days for a period of 4 cycles. Patients will be treated on a weekly basis continuously during 112 days or until progression. Blood tests (hemogram) are assessed weekly before each injection of PF-05212384 (PKI-587). Bone marrow aspiration (myelogram) is performed to evaluate the response before starting treatment and before the start of cycle 3 (after two cycles) and at the end of the study (after four cycles). Good responders who continue treatment after four cycles will be evaluated by bone marrow aspiration (myelogram) every two cycles and after the end of treatment
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
10
PF-05212384 will be delivered by intra-venous route at a fixed dose of 150 mg per week. Each treatment cycle includes four weekly injections The treatment is administered in cycles of 28 days for a period of 4 cycles. Patients will be treated on a weekly basis continuously during 112 days or until progression.
CHU de Toulouse
Toulouse, Midi-Pyrénées, France
Institut Paoli Calmette
Marseille, PACA, France
Hôpital Saint-Louis
Paris, Île-de-France Region, France
Hôpital Cochin
Paris, Île-de-France Region, France
To evaluate the efficacy of PF-05212384
The overall response rate will be assessed according to the International Working Group (IWG) AML and MDS criteria (by B.D. Cheson).
Time frame: 4 months after treatment
Tolerance and toxicity during treatment
Issued the Common Terminology Criteria for Adverse Events (CTCAE) version 4 National Cancer Institute (NCI)
Time frame: 4 months
Treatment compliance
Treatment compliance will be assessed by the ratio between the number of cycles administered on the expected number of cycles, and on time between treatment cycles
Time frame: 4 months
Progressive Free Survival (PFS)
Progressive Free Survival at one year from the date of inclusion to the date of progression of the disease or death
Time frame: one year
Overall survival
Overall Survival from the date of inclusion to the date of death
Time frame: 48 months
Evaluation of Quality of life
Quality of life (QLQ-C30) questionnaire according to European Organisation for Research and Treatment of Cancer (EORTC)
Time frame: 4 months
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Institut Curie - Hôpital René Huguenin
Saint-Cloud, Île-de-France Region, France