E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10040644 |
E.1.2 | Term | Sickle cell disease |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10002077 |
E.1.2 | Term | Anaemia sickle cell |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 allow access of crizanlizumab to patients with SCD who are on crizanlizumab treatment in a Novartis-sponsored study |
Permettre aux patients atteints de drépanocytose traités par crizanlizumab dans une étude promue par Novartis d’avoir accès à ce médicament |
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E.2.2 | Secondary objectives of the trial |
To assess the overall safety of crizanlizumab in SCD patients |
Evaluer la tolérance globale au crizanlizumab chez des patients atteints de drépanocytose |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Written informed consent/assent, according to local guidelines, signed by the adult patients. In the population under 18 years, it will be signed by the patient and/or by the parents or legal guardian prior to enrolling in the rollover study and receiving study medication
2. SCD patient currently enrolled in a Novartis-sponsored study receiving crizanlizumab and has fulfilled all the requirements in the parent study. Patient is currently benefiting from the treatment with crizanlizumab as determined by the investigator and has completed the treatment schedule as planned in the parent study
3. Patient has demonstrated compliance to the planned visit schedule in the parent study, and in the opinion of the investigator has shown willingness and
ability to comply with future visit schedules |
Pour l’inclusion dans l’étude, les patients doivent être affiliés à un régime de sécurité sociale (y compris la Couverture Médicale Universelle – CMU) ou être bénéficiaires d’un tel régime, et doivent remplir tous le2. Patient atteint de drépanocytose recevant le crizanlizumab dans le cadre d’une étude promue par Novartis et s’étant conformé aux exigences de l’étude parente. Le patient doit tirer bénéfice du traitement par crizanlizumab selon l’avis du médecin-investigateur et avoir terminé le traitement tel que prévu dans l’étude parente.
3. Le patient a respecté le calendrier des visites planifiées dans l’étude parente et a, selon le médecin-investigateur, la volonté et la capacité à se conformer à un autre calendrier de visites.s critères suivants :
1. Signature du consentement éclairé par les patients adultes ou par les parents/le représentant légal des patients âgés de moins de 18 ans, avant l’inclusion dans cette étude de roll-over et l’administration du médicament à l’étude. L’assentiment des patients âgés de moins de 18 ans devra également être obtenu en fonction des recommandations locales. |
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E.4 | Principal exclusion criteria |
1. Patient had permanently discontinued from crizanlizumab study treatment in the parent study before the parent study completion
2. Ongoing/unresolved treatment-related Grade 3 or higher AEs, and/or any ongoing AE requiring dose interruption. Patients meeting all other eligibility criteria
may be enrolled once toxicities have resolved unless those toxicities were grade 4
3. Concurrent participation in any other investigational clinical trial other than the parent study or plan to participate in any other investigational clinical trial
4. Pregnant or nursing women
5. Women of childbearing potential who are unwilling to be on highly effective contraceptives during dosing and until 15 weeks after stopping treatment with crizanlizumab
6. SCD patients who do not meet parent study protocol criteria to continue with crizanlizumab |
Les patients remplissant l’un des critères suivants ne sont pas éligibles à l’inclusion dans cette étude :
1. Patients ayant définitivement arrêté le crizanlizumab au cours de l’étude parente avant d’avoir terminé l’étude parente
2. Patients présentant des effets indésirables de grade 3 ou plus élevé en cours/non résolus, liés au traitement et/ou présentant tout effet indésirable en cours nécessitant une interruption du traitement. Les patients remplissant tous les autres critères d’éligibilité pourront être inclus lorsque ces toxicités seront résolues, excepté pour les toxicités de grade 4.
3. Participation simultanée à toute étude clinique autre que l’étude parente ou intention de participer à toute autre étude clinique
4. Femmes enceintes ou qui allaitent
5. Femmes en mesure d’avoir des enfants ne voulant pas utiliser de méthode de contraception très efficace pendant le traitement et jusqu’à 15 semaines après l’arrêt du traitement par crizanlizumab
6. Patients atteints de drépanocytose ne remplissant pas les critères du protocole de l’étude parente relatifs à la poursuite du traitement par crizanlizumab |
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E.5 End points |
E.5.1 | Primary end point(s) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Frequency, severity and causality of treatment emergent adverse events |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
from day of first dose of study medication to 105 days after last dose of study medication |
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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 | 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 access of crizanlizumab to patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored 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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 26 |
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 |
Bahrain |
Brazil |
Canada |
Colombia |
Egypt |
Ghana |
India |
Kenya |
Lebanon |
Oman |
Panama |
Saudi Arabia |
South Africa |
Turkey |
United States |
Belgium |
France |
Germany |
Ireland |
Italy |
Netherlands |
Spain |
Switzerland |
United Kingdom |
Greece |
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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.8.9.1 | In the Member State concerned years | 9 |
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 | 10 |