E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Acute Myelogenous Leukemia, including myelodysplastic syndrome and acute promyelocytic leukemia |
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E.1.1.1 | Medical condition in easily understood language |
A serious malignant blood disorder which is fatal if not treated |
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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 | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10000880 |
E.1.2 | Term | Acute myeloid leukaemia |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
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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 |
This expanded access protocol is designed in accordance with FDA 21 CFR 312.315 to allow compassionate access to Mylotarg for treatment of patients with AML who are thought to have the potential to derive clinical benefit and who have exhausted other appropriate and reasonable treatment options including investigational studies. |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Confirmed diagnosis of relapsed or refractory AML, including MDS, with persisting or rising blasts, and no other comparable or satisfactory alternative therapy available (including patients not eligible for or with access to investigational therapies through participation in a clinical trial). Relapsed or refractory AML is defined as having >=5% bone marrow blasts by morphology or if the bone marrow has <5% blasts by morphology but the patient has evidence of relapse with extra-medullary disease progression (leukemia cutis, chloroma or other features of extra-medullary relapse). In select cases presenting with conversion of minimal residual disease (MRD) from negative to positive, treatment with GO may be allowed where no alternative treatments are feasible.
2. Documentation that malignant cells express CD33 (according to the investigator’s own institutional standards that define positive expression).
3. Age ≥3 months.
4. Adequate non-hematologic organ function as defined by:
• Total serum bilirubin ≤2 x ULN; unless the patient has documented Gilbert’s Syndrome.
• Serum transaminases <5 x ULN (≤NCICTC v4.03 Grade 2)
• Serum creatinine ≤2 x ULN.
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E.4 | Principal exclusion criteria |
1. Untreated AML and MDS or AML and MDS in complete remission.
2. Patients with a known hypersensitivity to Mylotarg or its parts: hP67.6 antibody, calicheamicin derivatives or other ingredients.
3. Patients with a known history of VOD/SOS.
4. Participation in other studies involving investigational drug(s) (Phases 1-4) during study participation. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. The objective of the protocol is to provide patients compassionate access to Mylotarg. Therefore, there is no formal primary endpoint. Instead, safety information from patients receiving Mylotarg in this setting will be carefully monitored, collected and reported. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. 60 days after the last dose of Mylotarg |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | 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 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 0 |
E.8.9.2 | In all countries concerned by the trial months | 36 |
E.8.9.2 | In all countries concerned by the trial days | 0 |