E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Metachromatic Leukodystrophy (MLD) in late infantile patients |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10024381 |
E.1.2 | Term | Leukodystrophy |
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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 |
The primary objective of this study is to provide ongoing treatment with HGT-1111 to patients who have completed study HGT-MLD-048 (previously study rhASA-03) until HGT-1111 is commercially available or the study is terminated by the Sponsor, provided no safety concerns have emerged. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to monitor disease progression, as assessed by functional capacity, neurological examinations, cerebrospinal fluid (CSF) sulfatide concentrations and white matter metabolites by Magnetic Resonance Spectroscopic Imaging (MRSI) and the safety profile of HGT-1111 administered to patients who have completed study HGT-MLD-048 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The patient's legally authorized guardian(s) must provide signed informed consent prior to the performance of any study-related activities. (Study-related activities are any procedures that would not have been performed during normal management of the subject). 2. Completion of study HGT-MLD-048 3. The patient and his/her guardian(s) must have the ability to comply with the clinical protocol.
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E.4 | Principal exclusion criteria |
1. Spasticity so severe that it inhibiits the patient from being transported to the treatment site. 2. Presence of known clinically significant cardiovascular, hepatic, pulmonary or renal disease or any other medical condition that, in the opinion of the investigator, would preclude participatioin in the trial. 3. Any other medical condition or serious intercurrent illness or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety endpoints: Adverse events Vital signs Urine analysis Laboratory data Antibody and inhibitory antibody profile
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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 | No |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
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 | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
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 | 2 |
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 | 2 |
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 |