E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hematologic malignancies |
Neoplasias hematologicas |
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E.1.1.1 | Medical condition in easily understood language |
Hematologic malignancies |
Neoplasias hematologicas |
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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 use of s.a. panobinostat to patients receiving oral panobinostat in a Novartissponsored, Oncology CD&MA study which has reached its objectives and who are benefiting from treatment with oral panobinostat |
Permitir el uso continuado de panobinostat a.u a los pacientes que reciben panobinostat oral en un estudio de CDyMA en Oncología patrocinado por Novartis que haya alcanzado sus objetivos y que se estén beneficiando del tratamiento con panobinostat oral |
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E.2.2 | Secondary objectives of the trial |
To collect long term data on SAEs |
Obtener datos a largo plazo sobre AAGs |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient is currently enrolled in a Novartis-sponsored, Oncology CD&MA 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. |
1. El paciente está actualmente incluido en un estudio de CDyMA en Oncología patrocinado por Novartis, recibiendo panobinostat oral a.u y ha cumplido todos sus requisitos en el estudio principal. 2. El paciente actualmente se está beneficiando del tratamiento con panobinostat oral a.u determinado por las guías del protocolo principal y de acuerdo con el criterio clínico del Investigador. 3. El paciente ha demostrado cumplimiento, evaluado por el investigador, con los requisitos del protocolo principal. 4. Deseo y capacidad para cumplir con las visitas programadas, planes de tratamiento y cualquier otro procedimiento del estudio. 5. Consentimiento informado por escrito obtenido antes del reclutamiento en el estudio de extensión. ? Si el consentimiento no se puede expresar por escrito, debe ser formalmente documentado y atestiguado, idealmente por un testigo de confianza independiente. |
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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 (13 weeks for male participants and 1 week 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. 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. ? Combination of any two of the following (a+b* or a+c, or b+c): a. Use of oral, injected or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception. b. Placement of an intrauterine device (IUD) or intrauterine system (IUS) c. Barrier methods of contraception: Condom or Occlusive cap (diaphragm or cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository. *This option is only applicable for female participants of this trial. 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. A condom is required to be used by all men participating in this trial, including vasectomized men, as well as during intercourse with a male partner in order to prevent delivery of the genotoxic drug via semen. |
1. El paciente ha sido retirado permanentemente del tratamiento del estudio con panobinostat oral a.u en el estudio principal debido a toxicidad inaceptable, retirada del consentimiento, no cumplimiento con los procedimientos del estudio o cualquier otra razón (incluyendo progresión de la enfermedad). 2. El paciente ha participado en un ensayo de combinación patrocinado por Novartis en que panobinostat fue dispensado en combinación con otra medicación del estudio y todavía está recibiendo terapia en combinación. 3. La paciente está embarazada o en periodo de lactancia en el momento de la entrada en el estudio de extensión. El embarazo se define como el estado de una mujer tras la concepción y hasta la finalización de la gestación, confirmada por un análisis de laboratorio de hCG positivo. 4. Mujeres en edad fértil y pacientes hombres con pareja(s) sexuales que puedan quedar embarazadas y que no quieran utilizar métodos anticonceptivos altamente eficaces durante la dosificación y por una duración específica (13 semanas para participantes hombres y 1 semana para mujeres) después de suspender el tratamiento del estudio. Métodos anticonceptivos altamente efectivos incluyen: ? Abstinencia total (cuando ello concuerda con el estilo de vida preferido y habitual del paciente. Abstinencia periódica (p.e., método del calendario, ovulación, sintotérmico, post-ovulación) y retirada no son métodos anticonceptivos aceptables. ? Esterilización femenina (ooforectomía bilateral quirúrgica con o sin histerectomía) o ligadura de trompas. En caso de ooforectomía sola, solo cuando el estado reproductor de la mujer haya sido confirmado por seguimiento del análisis del nivel hormonal. ? Esterilización masculina (al menos 6 meses antes de la entrada en el estudio). Para sujetos mujeres en el estudio, la pareja masculina vasectomizada debe ser la única pareja de dicho sujeto.? Combinación de dos cualesquiera de los siguientes (a+b* or a+c, o b+c): a. Uso de métodos anticonceptivos hormonales orales, inyectados o implantados u otra forma de anticonceptivos hormonales que tengan eficacia comparable (tasa de fallo < 1%), por ejemplo anillo vaginal hormonal u anticonceptivo hormonal transdérmico. b. Colocación de un dispositivo intrauterino (DIU) o sistema intrauterino (SIU) c. Métodos anticonceptivos de barrera: preservativo o tapón oclusivo (diafragma o tapón cervical/vaginal) con espuma/gel/crema espermicida/supositorio vaginal. *Esta opción solo es aplicable para mujeres participantes de este ensayo. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Number of patients receiving s.a. panobinostat |
Número de pacientes que reciben panobinostat a.u |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
From Cycle 1 Day 1 through end of trial |
Desde el Ciclo 1 dia 1 hasta el final del estudio |
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E.5.2 | Secondary end point(s) |
Frequency and type of SAEs |
Frecuencia y tipo de AAGs |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
From Cycle 1 Day 1 (signing of ICF) through 30 day follow-up visit |
Desde el Ciclo 1 dia 1 (una vez firmado el consentimiento informado) hasta la visita de 30 dias de seguimiento |
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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 | 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 |
Permitir el uso continuado de panobinostat como agente unico a los pacientes que reciben panobinostat oral en un estudio de CDyMA en Oncología patrocinado por Novartis que haya alcanzado sus objetivos y que se estén beneficiando del tratamiento con panobinostat oral |
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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 | 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 | 3 |
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 |
Israel |
Spain |
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. |
El fin del estudio se define como una duración de 5 años o cuando todos los pacientes en este estudio hayan retirado permanentemente el tratamiento con panobinostat y la visita de fin de tratamiento más el seguimiento de seguridad a los 30 días hayan sido realizados para cada paciente (y no sea necesario ningún otro seguimiento), lo que suceda antes. |
|
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 |