E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary immunodeficiencies |
Déficit immunitaire |
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E.1.1.1 | Medical condition in easily understood language |
Primary immunodeficiencies |
Déficit immunitaire |
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061598 |
E.1.2 | Term | Immunodeficiency |
E.1.2 | System Organ Class | 10021428 - Immune system disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To determine the change in IgG trough level at steady state after standard SCIG dosing and after HyQvia administered every other week at equivalent doses. |
Evaluer le taux résiduel d’IgG après IGSC standard et HyQvia à dose équivalente administré toutes les deux semaines. |
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E.2.2 | Secondary objectives of the trial |
• To determine the IgG trough level at steady state after HyQvia administered every 3-4 weeks at equivalent dose.
• To describe: o The safety of HyQvia, o The tolerability of HyQvia, o The effectiveness of HyQvia, o The quality of life with standard SCIG, HyQvia every other week and HyQvia every 3-4 weeks. o The treatment satisfaction o The direct costs of treatment with HyQvia.
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• Evaluer le taux résiduel d’IgG après administration d’HyQvia administré toutes les 3 ou 4 semaines à dose équivalente. • Décrire : o La tolérance de HyQvia, o L’acceptabilité de HyQvia, o L’efficacité en conditions de vie réelles de HyQvia, o La qualité de vie, o Les coûts directs liés au traitement par HyQvia.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
To be included, subjects must satisfy all the following criteria: • Male or female subject at least 18 years old at the time inclusion. • Suffering from PI requiring immunoglobulin replacement therapy. • Treated with SCIG at stable dose for at least 3 months at the time of inclusion. • Well balanced SCIG treatment according to the investigator at the time of inclusion. • If female of childbearing potential, the subject must have a negative blood or urine pregnancy test at the time of inclusion and must agree to employ adequate birth control measures during the whole study. • Willing and able to comply with the requirements of the protocol. • Having signed the informed consent form. For patients who don’t want to participate in the study, the reason of their refusal will be collected if they agree to.
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• Patient de l’un ou l’autre sexe, âgés d’au moins 18 ans à l’inclusion • Présentant une IP nécessitant un traitement substitutif par immunoglobulines. • Traité depuis au moins 3 mois par IGSC à dose stable • Considéré comme bien équilibré par leur traitement IGSC, selon le jugement de l’investigateur. • Pour les patientes en âge de procréer : o Test de grossesse sanguin ou urinaire négatif à l’inclusion. o Acceptant d’utiliser une méthode de contraception efficace pendant la durée de l’étude • Souhaitant et capable de se conformer aux exigences du protocole. • Ayant signé un consentement écrit. Les motifs de refus des patients ne souhaitant pas participer à l’étude seront recueillis.
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E.4 | Principal exclusion criteria |
Subjects satisfying one or more of the following criteria should not take part in the study: • Known history of chronic kidney disease, or glomerular filtration rate (GFR) of <60 mL/min/1.73m2 estimated based on the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation at the time of screening. • Having received a chemotherapy or immunomodulating therapy for either malignant or chronic inflammatory disease for over 6 months. • Receiving anticoagulant therapy. • Having abnormal protein loss (protein losing enteropathy, nephrotic syndrome). • Know allergy to hyaluronidase. • Family member or employee of the investigator. • Having participated in another interventional clinical study involving an investigational product (IP) or investigational device within 30 days prior to inclusion or scheduled to participate in another clinical study involving an another investigational product or investigational device during the course of this study. • If female, pregnant or breastfeeding at the time of enrolment. • If female, planning to become pregnant during the time period of the study.
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• Examen biologique anormal à l’inclusion : o antécédents connus de maladie rénale chronique, ou taux de filtration glomérulaire (DFG) de <60 mL / min / 1,73m2 estimée d'après l’équation du Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) au moment du dépistage. • Ayant reçu une chimiothérapie ou des immunosuppresseurs ou un biomédicament dans les 12 mois précédents l’inclusion. • Traités par anticoagulants. • Présentant une perte de protéines anormale (perte protéique par entéropathie ou syndrome néphrotique). • Présentant une allergie connue à la hyaluronidase. • Employé ou membre de la famille de l’investigateur. • Ayant participé à une autre étude clinique interventionnelle impliquant un produit ou un matériel expérimental dans les 30 jours précédant l’inclusion ou prévu pour participer à un essai de ce type pendant le déroulement de l’étude. • Patiente enceinte ou allaitante au moment de l’inclusion. • Patiente planifiant une grossesse pendant la durée de l’étude.
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary endpoint is the change in IgG trough level at V3 as compared to V1. |
Evolution du taux résiduel d’IgG entre l’inclusion et après 3 mois de traitement par HyQvia toutes les 2 semaines. |
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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) |
• IgG trough level at V4 The change in IgG trough level at V4 as compared to V1 and V3 will be determined.
• The safety of HyQvia will be described at the end of the study by means of the following criteria: o Number and incidence rate (overall and by injection) of systemic emergent related AEs (excluding infections). o Number and incidence rate (overall and by injection) of local emergent related AEs (excluding infections). o Number and incidence rate (overall and by injection) of all emergent related AEs (excluding infections). • Tolerability objectives and endpoints Tolerability of HyQvia will be described at visits 3 and 4 by means of the treatment satisfaction as measured by the TSMQ-9 score. The TSQM-9 score at visit 1 assessing the tolerability of standard SCIG will be used as baseline value. • Effectiveness objective and endpoints Efficacy of HyQvia will be described at the end of the study by means of the following criteria: o Number and annualized rate of acute serious bacterial infections. o Number and rate of all infections. • Quality of life Quality of life will be assessed by the SF-36 score at visits 1, 3 and 4. • Pharmaco-economic objectives and endpoint Direct cost of HyQvia treatment will be described at the end of the study by taking into account the cost of medications, rental of the pump(s), nurse time, SC needle sets, transportations, hospital costs.
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Secondaires (pharmacocinétique) : • Taux résiduel d’IgG après 3 mois de traitement par HyQvia toutes les 3 ou 4 semaines Secondaires (tolérance) : • Nombre et taux d’incidence des effets indésirables systémiques émergents et liés au traitement (hors infections). • Nombre et taux d’incidence des effets indésirables locaux émergents et liés au traitement (hors infections) • Nombre et taux d’incidence de tous les effets indésirables émergents et liés au traitement (hors infections). Secondaire (acceptabilité du traitement) Questionnaire de satisfaction (TSQM-9). Secondaires (efficacité en condition de vie réelle) : • Nombre et taux annualisé d’infections bactériennes sévères. • Nombre et taux annualisé de toutes les infections Secondaire (qualité de vie) Questionnaire SF-36. Secondaire (pharmaco-économiques) : Coût direct du traitement.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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 | Yes |
E.6.13 | Others | No |
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 | 7 |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
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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LVLS |
Dernière visite du dernier patient |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |