E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
The medical condition investigated are patients with acute myeloid leukemia in complete remission with persistent minimal residual disease and as such are at high risk of relapse. |
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E.1.1.1 | Medical condition in easily understood language |
Adult acute myeloid leukemia (AML) is a type of cancer in which the bone marrow makes abnormal myeloblasts (a type of white blood cell), red blood cells, or platelets. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000886 |
E.1.2 | Term | Acute myeloid leukemia |
E.1.2 | System Organ Class | 100000004864 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
• To assess the effect of DCP-001 on MRD. MRD will be measured by flow cytometry pre and post vaccination as a surrogate marker for established clinical endpoints in AML. • To assess the effect of DCP-001 on immune responses in AML patients in first complete remission (CR1) and persistent MRD. • To document safety and tolerability.
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E.2.2 | Secondary objectives of the trial |
• To identify surrogate (immunological) markers that might correlate with clinical outcome. • To quantify any lag time between initiation of treatment & onset of effect. • To determine the effect of DCP-001 on Time to Relapse (TTR). • To determine the effect of DCP-001 on Overall Survival (OS) during the study. • To document the difference between the two vaccination regimens based on MRD |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Confirmed diagnosis of AML according to WHO2016 criteria, including cytological, molecular and cytogenetic criteria (except acute pro-myelocytic leukemia/APL). 2. In CR1 (first complete remission) or CRi (incomplete blood count recovery) documented by bone marrow examination up to one month before vaccination; CR defined as less than 5% blasts in normo-cellular bone marrow, ANC >1*E9/L, platelet count 100*E9/L, no evidence of extra-medullary disease. Patients in CRi (patients in CR1 but with incomplete blood count recovery) should have platelets >50 E9/L. 3. MRD as defined by multicolor flow cytometry (MFC) at a value of > 0.1% 4. Patients that are in CR1 or Cri. Patients not having undergone consolidation therapy must have been in CR1 or CRi for at least 1 month prior to enrolment. Patients treated with hypomethylating agents must have been given at least two cycles and up to a maximum of nine cycles of hypomethylating agents. 5. Expected and willing to undergo all study procedures, including outpatient evaluations for clinical and immunological monitoring. 6. Male or female of > 18 years of age. 7. Women of childbearing potential must be on anti-conceptive therapy or use two (2) barrier contraceptive methods (one by each partner and at least one of the barrier methods must include spermicide (unless spermicide is not approved in the country or region) , or underwent tubal ligation, or the partner was vasectomized, or is sexually abstinent. 8. ECOG (WHO) performance status 0-2. 9. Willing and able to provide written informed consent for participation in the study and for tissue sample biobanking.
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E.4 | Principal exclusion criteria |
1. Acute Promyelocytic (APL; M3) type of AML. 2. Patients who have undergone or are scheduled/eligible for allogeneic stem cell transplantation. 3. History of previous allogeneic bone marrow or solid organ transplantation. 4. Uncontrolled or serious infections 5. Ongoing immunosuppressive therapy, other than short use of low dose steroids, i.e. equivalent to an average dose of ≤10mg of prednisone. 6. Chemotherapy and antineoplastic therapy within 28 days prior to the screening visits, with the exception of hypomethylating agents such as azacitidine and decitabine, or midostaurin for FLT3 mutations, or patients treated with IDH1/2 inhibitors in mIDH1/2. 7. Current or past medical history of autoimmune disease. 8. Inadequate liver function (AST and ALT > 3 x ULN, serum bilirubin >3 x ULN). 9. Other active Malignancies within the last 5 years, except for adequately treated carcinoma in situ of the cervix or squamous carcinoma of the skin or adequately controlled limited basal cell skin cancer. 10. Pregnant or lactating females. 11. Major surgical procedure (including open biopsy) within 28 days prior to the first study treatment, or anticipation of the need for major surgery during the course of the study treatment. 12. Uncontrolled hypertension (systolic > 150 mm Hg and/or diastolic > 100 mm Hg) or clinically significant (i.e. active) cardiovascular disease. 13. Evidence of any other medical conditions (such as psychiatric illness, physical examination or laboratory findings) that may interfere with the planned treatment, affect patient compliance or place the patient at high risk from treatment-related complications. 14. Known HIV, Hepatitis B or C infections. 15. History of hypersensitivity to the investigational medicinal product or to any excipient present in the pharmaceutical form of the investigational medicinal product |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary Efficacy Endpoint - The effect of DCP-001 on MRD levels as a surrogate marker for established clinical endpoints in AML. - To assess the effect of DCP-001 on immune responses in AML patients in first complete remission (CR1) with MRD. - To document safety and tolerability.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
These endpoints will be evaluated ~17 months after start last patient |
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E.5.2 | Secondary end point(s) |
Secondary Efficacy Endpoints - To quantify any lag time between initiation of treatment & onset of effect. - To determine the effect of DCP-001 on Time to Relapse (TTR). - To determine the effect of DCP-001 on Overall Survival (OS). - To document the difference between the two vaccination regimens based on MRD outcome and immunological parameters.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
These endpoints will be evaluated ~5 years after start last patient |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 7 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 7 |
E.8.9.2 | In all countries concerned by the trial months | 7 |