E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Arginase I deficiency Hyperargininemia |
Deficit di arginasi 1 Iperargininemia |
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E.1.1.1 | Medical condition in easily understood language |
Arginase I deficiency Hyperargininemia |
Deficit di arginasi 1 Iperargininemia |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10062695 |
E.1.2 | Term | Arginase deficiency |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
|
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 the safety of long-term subcutaneous (SC) pegzilarginase in subjects with arginase 1 deficiency (ARG1-D) |
Valutare la sicurezza a lungo termine di pegzilarginase sottocutanea (SC) in soggetti con deficit di arginasi 1 (ARG1-D) |
|
E.2.2 | Secondary objectives of the trial |
Not applicable |
Non applicabile |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Prior enrollment in Studies 102A, 300A, or 301A, with completion of treatment or continued dosing as of the study termination date 2. Written informed consent by subject/parent/legal guardian, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol 3. Negative pregnancy test within 7 days prior to dosing in childbearing subjects if they are sexually active 4. If the subject (male or female) is engaging in sexual activity that could lead to pregnancy, must be surgically sterile, post-menopausal (female), or must agree to use a highly effective method of birth control during the study and for a minimum of 30 days after the last study drug administration. Highly effective methods of contraception include combined (estrogen and progestogen-containing) hormonal contraception associated with inhibition of ovulation, progesterone-only hormonal contraception associated with inhibition of ovulation,intrauterine device, or intrauterine hormone-releasing system. |
1. Arruolamento precedente negli Studi 102A, 300A o 301A, con completamento del trattamento o trattamento ancora in corso alla data della conclusione dello studio 2. Consenso informato scritto da parte del soggetto/genitore/tutore, che includa la conformità ai requisiti e alle restrizioni elencati nel modulo di consenso informato (MCI) e nel presente protocollo 3. Test di gravidanza negativo nei 7 giorni precedenti alla somministrazione a soggetti fertili, se sessualmente attivi 4. Se il soggetto (maschio o femmina) ha rapporti sessuali che potrebbero portare alla gravidanza, dovrà essere chirurgicamente sterile, in post-menopausa (femmina) o dovrà accettare di utilizzare un metodo altamente efficace di controllo delle nascite durante lo studio e per almeno 30 giorni dopo l'ultima somministrazione del farmaco dello studio. I metodi contraccettivi altamente efficaci includono: contraccezione ormonale combinata (contenente estrogeni e progestinici) associata all'inibizione dell'ovulazione, contraccezione ormonale a base di solo progesterone associata all'inibizione dell'ovulazione, dispositivo intrauterino o sistema di rilascio ormonale intrauterino. |
|
E.4 | Principal exclusion criteria |
1. Subjects with acute or ongoing HA within 6 weeks of the first dose 2. Subjects with medical conditions or underlying issues that, in the opinion of the Investigator, would preclude participation in the study |
1. Soggetti con HA acuta o in corso durante le 6 settimane precedenti alla prima dose 2. Soggetti con condizioni mediche o problemi pre-esistenti che, secondo il parere dello Sperimentatore, ne precluderebbero la partecipazione allo studio |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Safety assessments that will be conducted as part of this study include the following: o Adverse events (AEs), including hypersensitivity reactions (HSRs), injection site reactions (ISRs), and hyperammonemic episodes (HAs) o Vital signs o Weight |
Le valutazioni della sicurezza che saranno condotte nell'ambito di questo studio includono: o Eventi avversi (AE), incluse reazioni di ipersensibilità (HSR), reazioni in sede di iniezione (ISR) ed episodi iperammoniemici (HA) o Segni vitali o Peso |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Refer to protocol endpoint timepoints |
Si faccia riferimento timepoint dell'endpoint nel protocollo |
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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 |
Tolerability |
Tollerabilità |
|
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 | No |
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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 5 |
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 | Information not present in EudraCT |
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 |
Canada |
United States |
France |
Germany |
Italy |
Portugal |
United Kingdom |
|
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
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |