E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
This roll-over study is designed to accept patients with varied disease origins. Please refer to the parent protocol for the disease background information and rationale for use of ruxolitinib in their individual indications. |
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E.1.1.1 | Medical condition in easily understood language |
This study is designed to accept patients with varied disease origins. Please refer to the parent protocol. |
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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 | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To allow continued treatment to patients receiving ruxolitinib in existing global Novartis or Incytesponsored studies who are benefiting from treatment after conclusion of the parent study. |
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E.2.2 | Secondary objectives of the trial |
To collect long term data on adverse events (AEs) and serious adverse events (SAEs). |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patient is currently enrolled in a Novartis OGD or GMA-sponsored or Incyte-sponsored clinical study (where Incyte can delegate the sponsorship to a preferred CRO, if applicable) that is approved to enroll into this rollover study, is receiving ruxolitinib and has fulfilled all of the requirements of the parent protocol.
- Patient is currently benefiting from the treatment with ruxolitinib, as determined by the investigator
- Patient has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements
- Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures
- Patient currently has no evidence of progressive disease, as determined by the investigator, following previous treatment with ruxolitinib
- Written informed consent obtained prior to enrolling in roll-over study and receiving study medication. If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness. |
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E.4 | Principal exclusion criteria |
- Patient has been permanently discontinued from study treatment in the parent study due to any reason.
- Patient’s indication is currently approved and reimbursed in the local country
- Patient has participated in a combination trial where ruxolitinib was dispensed in combination with another study medication and the patient is still receiving combination therapy.
- Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
- Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception throughout the study duration inclusive of the 30-day safety follow up. Highly effective contraception methods include: see protocol.
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E.5 End points |
E.5.1 | Primary end point(s) |
- Number of patients receiving ruxolitinib.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
No statistical analysis will be performed. |
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E.5.2 | Secondary end point(s) |
- Frequency and nature of adverse events and serious adverse events. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
The assessment of safety will be based on the frequency of adverse events (AEs) and serious adverse events (SAEs). |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
To allow continued treatment to patients receiving ruxolitinib in existing global Novartis or Incytesponsored studies who are benefiting from treatment after conclusion of the parent study |
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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) | 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 | 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
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 |
Australia |
Belgium |
Germany |
Greece |
Italy |
Lebanon |
Spain |
Thailand |
Turkey |
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 the study will occur after all patients in the study have completed their last
assessment per protocol. Patients may continue on study
treatment until one of the following criteria is met, whichever comes first:
• End of Treatment
• At least one of the premature patient withdrawal criteria are met
• 5 years after the first patient’s first visit into this clinical study
• Current treatment becomes commercially available and reimbursed in that indication |
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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 | 5 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 5 |