E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cerebral Adrenoleukodystrophy (CALD) |
adrenoleucodistrofia cerebrale (CALD) |
|
E.1.1.1 | Medical condition in easily understood language |
Cerebral Adrenoleukodystrophy (CALD) |
adrenoleucodistrofia cerebrale (CALD) |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
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 | 10051260 |
E.1.2 | Term | Adrenoleukodystrophy |
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 |
- Monitor for long-term safety of the Lenti-D Drug Product (also known as elivaldogene autotemcel; hereafter referred to as eli-cel) administered in parent clinical studies. - Monitor for long-term efficacy of eli - cel administered in parent clinical studies. |
• Monitorare la sicurezza a lungo termine del prodotto medicinale Lenti-D (noto anche come elivaldogene autotemcel; di seguito indicato come eli-cel) somministrato negli studi clinici originari. • Monitorare l’efficacia a lungo termine di eli-cel somministrato negli studi clinici originari. |
|
E.2.2 | Secondary objectives of the trial |
Not Applicable |
Not applicable |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Provision of written informed consent for this study by the subject or subject’s parent(s)/ legal guardian(s) and written informed assent by subject, if applicable 2.Have received eli-cel Drug Product in a parent clinical study. |
1. Rilascio del consenso informato scritto per questo studio da parte del soggetto o, se pertinente, del/i genitore/i/tutore/i legale/i del soggetto e assenso informato scritto da parte del soggetto 2. Aver ricevuto eli-cel in uno studio clinico originario |
|
E.4 | Principal exclusion criteria |
There are no exclusion criteria for this Study. |
Non vi sono criteri di esclusione per questo studio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
• The number of subjects who experience graft versus host disease (GVHD) through 15 years post-drug product infusion • The number of subjects with immune-related AEs (e.g., autoimmune disorders, GVHD, opportunistic infections, HIV) through 15 years post drug product infusion • The number of subjects with new or worsening hematologic disorders through 15 years post-drug product infusion • The number of subjects with new or worsening neurologic disorders through 15 years post-drug product infusion • The number of subjects with malignancies through 15 years post-drug product infusion • Major functional disability (MFD)-free survival over time through Year 15 post-drug product infusion |
• Numero di soggetti che manifestano malattia del trapianto contro l’ospite (GVHD) per 15 anni dopo l’infusione del prodotto medicinale • Numero di soggetti con EA immuno-correlati (per es. disturbi autoimmuni, GVHD, infezioni opportunistiche, HIV) per 15 anni dopo l’infusione del prodotto medicinale • Numero di soggetti con disturbi ematologici nuovi o in peggioramento per 15 anni dopo l’infusione del prodotto medicinale • Numero di soggetti con disturbi neurologici nuovi o in peggioramento per 15 anni dopo l’infusione del prodotto medicinale • Numero di soggetti con neoplasie maligne per 15 anni dopo l’infusione del prodotto medicinale • Sopravvivenza libera da disabilità funzionale maggiore (MFD) nel tempo, fino all’Anno 15 dopo l’infusione del prodotto medicinale |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
In Study LTF-304, follow-up visits are scheduled every 6 months through Year 5 post-drug product infusion, and then annually thereafter through Year 15 post-drug product infusion. |
Le visite di follow-up nello studio LTF-304 sono programmate almeno ogni 6 mesi fino all’Anno 5 successivamente all’infusione del prodotto medicinale e in seguito ogni anno fino all’Anno 15 successivamente all’infusione del prodotto medicinale. |
|
E.5.2 | Secondary end point(s) |
• Change from Baseline (defined in parent study) through Year 15 post-drug product infusion in neurologic function score (NFS) • The number of subjects without gadolinium enhancement (GdE) on MRI over time through Year 15 post-drug product infusion • The number of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) through 15 years post-drug product infusion |
• Variazione dal basale (definita nello studio originario) fino all’Anno 15 dopo l’infusione del prodotto medicinale nel punteggio della funzione neurologica (NFS) • Numero di soggetti che non presentano potenziamento del gadolinio (GdE) alla RM nel tempo, fino all’Anno 15 dopo l’infusione del prodotto medicinale • Numero di soggetti sottoposti a successivo trapianto di cellule staminali (ovvero, seconda infusione di CSE) per 15 anni dopo l’infusione del prodotto medicinale |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
In Study LTF-304, follow-up visits are scheduled every 6 months through Year 5 post-drug product infusion, and then annually thereafter through Year 15 post-drug product infusion. |
Le visite di follow-up nello studio LTF-304 sono programmate almeno ogni 6 mesi fino all’Anno 5 successivamente all’infusione del prodotto medicinale e in seguito ogni anno fino all’Anno 15 successivamente all’infusione del prodotto medicinale. |
|
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 | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
Long-term follow up |
Long-term follow up |
|
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 | 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 | 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 |
Argentina |
Australia |
Brazil |
Canada |
United States |
France |
Netherlands |
Germany |
Italy |
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 | 13 |
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 | 13 |
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 |