E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Defined by parent protocol. The study will enroll all adult and paediatric subjects who received at least one genetically modified T cells infusion in a previous Celgene sponsored study. |
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E.1.1.1 | Medical condition in easily understood language |
Defined by parent protocol |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
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 | 10025631 |
E.1.2 | Term | Malignant lymphoid neoplasm NOS |
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 risk of delayed adverse events following exposure to GM T cells - To monitor for long-term persistence of GM T cells, including analysis of vector integration sites, as appropriate. - To monitor for generation of replication-competent lentiviruses - To assess long-term efficacy following treatment with GM T cells - Describe growth and sexual maturity status for subjects who were aged < 18 years at time of GM T-cell treatment
GM = gene modified
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E.2.2 | Secondary objectives of the trial |
To monitor for B-cell levels in subjects who received CD19-directed GM T-cell therapy |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. All adult and pediatric subjects who received at least one GM T cell infusion in a previous Celgene-sponsored or Celgene alliance partner-sponsored study, and have discontinued, or completed the post-treatment follow-up period in the parent treatment protocol, as applicable. 2. Subject (and, parental/legal representative, when applicable) must understand and voluntarily sign an Informed Consent Form (ICF) / Informed Assent Form (IAF) prior to any study-related assessments/procedures being conducted. 3. Not applicable as of Protocol Amendment 01: Subject is willing and able to adhere to the study visit schedule and other protocol requirements. |
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety: - Incidence of delayed Adverse Events considered at least possibly related to prior GM T-cell therapy, including: - New neurologic disorder, or exacerbation of a pre-existing neurologic disorder - New rheumatologic or autoimmune disorder, or exacerbation of a prior rheumatologic or other autoimmune disorder - New hematologic disorder - New infection - Other new clinical conditions considered related to the prior GM T-cell therapy by the investigator, including an SPM. Hospitalizations, for events considered related to or possibly related to prior GM T-cell therapy and unexpected illness, rare disorders or events of unknown etiology, including reasons and dates
- Persistence of GM T cells - Analysis of vector integration sites - Incidence of replication-competent lentiviruses - Physical growth as assessed by physical examination (pediatric subjects only) - Sexual maturity (pediatric subjects only)
Efficacy Where applicable: - Disease Status - Date of Disease Progression - Date of Relapse - Survival Status
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Other endpoints: Up to 15 years from last GM T-cell infusion |
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E.5.2 | Secondary end point(s) |
Safety (subjects who received CD19-directed GM T-cell therapy) - Lymphocyte count (B-cell) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Up to 5 years from last GM T-cell infusion and then until B-cell recovery (B-cell ≥ 3% of lymphocytes and within testing laboratory's reference range of normal) confirmed by 2 consecutive analyses, or up to 15 years, until subject withdrawal of consent, or death, whichever occurs first |
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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 | No |
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 | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Health-related quality of life (HRQoL) |
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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 | No |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Long-Term Follow-up Protocol |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 0 |
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 | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 60 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Japan |
United States |
Austria |
Finland |
France |
Sweden |
Netherlands |
Spain |
Switzerland |
Germany |
Italy |
Belgium |
Norway |
United Kingdom |
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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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The End of Trial is defined as either the date of the last visit of the last subject or the date of receipt of the last data point from the last subject that is required for primary analysis, as pre-specified in the protocol, whichever is the later date. |
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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 | 15 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 15 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |