E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
hydroxyurea resistant or intolerant patients with polycythemia vera |
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E.1.1.1 | Medical condition in easily understood language |
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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 | 17.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10036061 |
E.1.2 | Term | Polycythemia vera |
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 evaluate the safety of ruxolitinib |
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E.2.2 | Secondary objectives of the trial |
Efficacy
- to evaluate change in hematrocrit levels
- to evaluate change in speen length
Patient Reported Outcomes:
- to evaluate change in MPN-SAF TSS score |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female aged 18 or over
2. Confirmed diagnosis of PV according to the revised World Health Organization (WHO) criteria
3. HU resistant or intolerant
4. Peripheral blood blast count of 0% at screening
5. Palpable spleen (unless the patient is entering the protocol after he/she has successfully completed another clinical trial with ruxolitinib in PV and the study team has agreed that the patients can be included in this protocol)
6. ECOG performance status of 0, 1, or 2 at Baseline
7. Must have recovered or stabilized sufficiently from adverse drug reactions associated with any prior treatments before beginning treatment with ruxolitinib
8. Does not have access to a comparable or satisfactory alternative treatment (i.e., comparable or satisfactory treatment is not available or does not exist)
9. Is not eligible for participation in any of the ruxolitinib ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (e.g., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient)
10. Is not being transferred from an ongoing clinical trial for which he/she is still eligible |
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E.4 | Principal exclusion criteria |
1. Inadequate liver or renal function
2. Platelet counts < 50 ×109/L or an Absolute Neutrophil Count (ANC) < 1 × 109/L at screening
3. Clinically significant bacterial, fungal, parasitic or viral infection which require therapy
4. History of progressive multifocal leukoencephalopathy (PML)
5. Patients with any concurrent condition that, in the Investigator’s opinion would jeopardize the safety of the patient or compliance with the protocol
Other exclusion criteria as per protocol section 5.3 may apply. |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. AEs, Grade 3&4 AEs & SAEs, events of special interest, AEs leading to
discontinuation, and deaths.
2. Evaluate changes in hematology |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
refer to protocol section 10.4 |
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E.5.2 | Secondary end point(s) |
Efficacy:
- Change in Hct levels from Baseline to each visit where measured
- Change in spleen length from Baseline to each visit where measured
Patient Reported Outcome:
Change in MPN-SAF TSS score from baseline to each visit where measured |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Efficacy:
- Week 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108
- Week 12, 24, 36, 48, 60, 72, 84, 96, 108
Patient Reported Outcome:
Week 12, 24, 36, 48, 60, 72, 84, 96, 108 |
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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 | 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 |
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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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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
| 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 | 146 |
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 |
Algeria |
Argentina |
Austria |
Belgium |
Brazil |
Colombia |
Croatia |
Estonia |
Germany |
Hungary |
India |
Israel |
Italy |
Korea, Republic of |
Lebanon |
Mexico |
Morocco |
Netherlands |
Norway |
Poland |
Portugal |
Saudi Arabia |
Slovakia |
Slovenia |
South Africa |
Spain |
Sweden |
Thailand |
Tunisia |
Jordan |
Venezuela, Bolivarian Republic of |
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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 study will end for each country 3 months after the drug is commercially available in the country or by 31 December 2017, whichever occurs first. The entire study will end when all countries have completed participation. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 3 |