E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
CD19+ B cell lymphoma or leukemia |
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E.1.1.1 | Medical condition in easily understood language |
Leukemia and lymphoma, blood cell cancer |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the feasibility of a single administration of CAR T cells to patients with disseminated B cell lymphoma or leukemia by studying the tolerance and toxicity. |
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E.2.2 | Secondary objectives of the trial |
To: - establish persistence of CAR T cells - evaluate the level of B cells as a measurement of CAR T cell efficacy - evaluate immune profile shifts post treatment as a measurement of CAR T cell function - tumor response |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients may be included in the study only if they meet all of the following criteria:
1) Relapsed or refractory CD19+ B-cell lymphoma or leukemia 2) Measurable disease. 3) Performance status ECOG 0-2. 4) >18 years old. 5) Fertile females/males must consent to use contraceptives during participation of the trial. 6) Adequate bone marrow, renal, hepatic and cardiovascular function. 7) Signed informed consent.
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E.4 | Principal exclusion criteria |
The presence of any of the following criteria will exclude the patient from participating in the study:
1. Any significant medical or psychiatric illness that would prevent the patient from giving informed consent or from following the study procedures. 2. Patients with primary CNS lymphoma. 3. Known human immunodeficiency virus (HIV) infection. 4. Active and/or severe infection (e.g. tuberculosis, sepsis and opportunistic infections, active hepatitis B virus (HBV) or active hepatitis C virus (HCV) infection). 5. Other serious underlying medical conditions, which, in the Investigator’s judgment, could impair the ability of the patient. 6. Treatment with an investigational product within 30 days prior to enrolment. 7. Patients that do not consent to that tissue and blood samples are stored in a biobank. 8. Pregnancy
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E.5 End points |
E.5.1 | Primary end point(s) |
Registration of the safety profile such as inflammation, fever, pain, changes in blood pressure, pulse and other adverse events.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Weekly the first 5 weeks, then at 3, 6, 9 and 12 months. |
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E.5.2 | Secondary end point(s) |
- Determination of the level of CAR T cells (mRNA and cells) in blood and biopsies. - Determination of the levels of circulating B cells. - Determination of the presence of immunological markers in blood and biopsies. - Determination of tumor size and the tumor marker CD19. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
At week 5 and then at 3 and 9 months |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
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) | Yes |
E.7.1.1 | First administration to humans | Yes |
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.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
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 | No |
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 | 2 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | 0 |