E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cerebral Adrenoleukodystrophy (CALD) |
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E.1.1.1 | Medical condition in easily understood language |
Cerebral Adrenoleukodystrophy (CALD) |
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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 |
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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 |
- 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. |
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E.2.2 | Secondary objectives of the trial |
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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 Lenti-D Drug Product in a parent clinical study. 3.Able to comply with study requirements.
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E.4 | Principal exclusion criteria |
There are no exclusion criteria for this Study. |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Major functional disability (MFD)-free survival over time through Year 15 post-drug product infusion Note: interim analyses (including evaluation of MFD-free survival [i.e., subjects who were alive without an MFD or need for hematopoietic stem cell transplant [HSCT]; also referred to as event-free survival by FDA) will occur once all subjects have completed 5 years and 10 years of post infusion follow-up (see Section 7.6.1). • The number of subjects with malignancies through 15 years post-drug product infusion • 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 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Visits are scheduled every 4 months through their post-drug product infusion Year 10 Visit, and then every 6 months from the Year 10.5 Visit through their post-drug product infusion Year 15 Visit. |
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E.5.2 | Secondary end point(s) |
• The number of subjects who undergo subsequent stem cell transplantation (i.e. second HSC infusion) through 15 years post-drug product infusion • 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 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Visits are scheduled every 4 months through their post-drug product infusion Year 10 Visit, and then every 6 months from the Year 10.5 Visit through their post-drug product infusion Year 15 Visit. |
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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 | 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 |
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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 | 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 | 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 |
Argentina |
Australia |
Brazil |
Canada |
United States |
France |
Germany |
Italy |
United Kingdom |
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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 | 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 |