| E.1 Medical condition or disease under investigation |
| E.1.1 | Medical condition(s) being investigated |
|
| E.1.1.1 | Medical condition in easily understood language |
|
| 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 evaluate long term safety data (SAEs and AEs) |
|
| E.2.2 | Secondary objectives of the trial |
| To evaluate clinical benefit as assessed by the investigator |
|
| E.2.3 | Trial contains a sub-study | No |
| E.3 | Principal inclusion criteria |
1. Patient is currently enrolled in a Novartis-sponsored, Oncology OGD&GMA study receiving s.a. oral panobinostat and has fulfilled all their requirements in the parent study.
2. Patient is currently benefiting from the treatment with s.a. oral panobinostat as determined by the guidelines of the parent protocol and according to the Investigator’s clinical judgment.
3. Patient has demonstrated compliance, as assessed by the investigator, with the parent study protocol requirements.
4. Willingness and ability to comply with scheduled visits, treatment plans and any other study procedures.
5. Written informed consent obtained prior to enrolling into the roll-over study.
• If consent cannot be expressed in writing, it must be formally documented and witnessed, ideally via an independent trusted witness. |
|
| E.4 | Principal exclusion criteria |
1. Patient has been permanently discontinued from s.a. oral panobinostat study treatment in the parent study due to unacceptable toxicity, withdrawal of consent, non-compliance to study procedures or any other reason (including progression of disease).
2. Patient has participated in a Novartis sponsored combination trial where panobinostat was dispensed in combination with another study medication and is still receiving combination therapy.
3. Patient is pregnant or nursing (lactating) at time of entry into the roll-over protocol.
Pregnancy is defined as the state of a female after conception and until the termination of
gestation, confirmed by a positive hCG laboratory test.
4. Women of child-bearing potential and male patients with sexual partner(s) of childbearing potential unwilling to use highly effective methods of contraception during dosing and for a specified duration (6 months for male participants and 3 months for females) after stopping study treatment.
Highly effective contraception methods include:
• Total abstinence (when this is in line with the preferred and usual lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
• Female sterilization (surgical bilateral oophorectomy with or without hysterectomy)
or tubal ligation at least six weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
• Male sterilization (at least 6 months prior to study entry). For female subjects on the study, the vasectomized male partner should be the sole partner for that subject.
• Use of oral, injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception. Women using hormonal contraceptives should additionally use a barrier method of contraception.
Note: In case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before entering the roll-over protocol.
• Women are considered post-menopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (i.e. age appropriate,history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or tubal ligation at least six weeks ago. In the case of oophorectomy alone,only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of child bearing potential.
• If a study patient becomes pregnant or suspects being pregnant during the study or within 30 days after the final dose of panobinostat, the Investigator/Study Doctor needs to be informed immediately and ongoing study treatment with panobinostat has to be stopped
immediately.
5. Sexually active males unless they use a condom during intercourse while taking the drug during treatment and for 6 months after stopping treatment and should not father a child in this period. A condom is required to be used also by vasectomized men as well as during
intercourse with a male partner in order to prevent delivery of the drug via semen. |
|
| E.5 End points |
| E.5.1 | Primary end point(s) |
| Frequency and severity of AEs/SAEs |
|
| E.5.1.1 | Timepoint(s) of evaluation of this end point |
| The primary objective will be evaluated at the end of the study once all patients have discontinued. |
|
| E.5.2 | Secondary end point(s) |
| Proportion of patients with clinical benefit as assessed by the investigator at scheduled visits |
|
| E.5.2.1 | Timepoint(s) of evaluation of this end point |
| The secondary objective will be evaluated at the end of the study once all patients have discontinued. |
|
| 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 | Yes |
| E.6.13.1 | Other scope of the trial description |
| To allow continued use of single agent panobinostat in patients who are on single agent panobinostat treatment in a Novartis-sponsored, Oncology Clinical Development & Medical Affairs (CD&MA) study and are benefiting from the treatment as judged by the investigator |
|
| 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 | 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
| 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 | 4 |
| 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 |
| France |
| Israel |
| Netherlands |
| Spain |
| United Kingdom |
| United States |
|
| 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
|
End of study is defined as either a 5 year duration or when all patients in this study have permanently discontinued panobinostat treatment and the end of treatment visit plus the 30-
day safety follow up have been performed for each patient (and no further follow-up is needed), whichever comes earlier. |
|
| 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 | 0 |
| E.8.9.1 | In the Member State concerned days | 0 |
| E.8.9.2 | In all countries concerned by the trial years | 5 |
| 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 |
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