E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) |
|
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 6.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10056892 |
|
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 objective of this study is to evaluate the long-term efficacy and safety of rhASB treatment in patients with MPS VI. |
|
E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
· The patient must sign an informed consent form before any protocol-related procedures can be performed. If the patient is under 18 years of age, a legally authorized guardian must sign the informed consent form. In addition, patients who are under 18 years of age and can understand the consent form may be required to indicate their willingness to participate by signing the minor assent portion of the informed consent, if stipulated by the site’s IRB. · The patient must have successfully completed Study ASB-03-05, including having received at least 20 of the 24 scheduled weekly infusions and having missed no more than two consecutive infusions. · A female patient of childbearing potential must have a negative pregnancy test (urine b–human chorionic gonadotropin) at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature male patients must be advised to use a medically accepted method of contraception throughout the study. Female patients of childbearing potential must be willing to undergo periodic pregnancy tests during the course of the study.
|
|
E.4 | Principal exclusion criteria |
· The patient is pregnant or lactating. · The patient has received an investigational drug (other than rhASB in Study ASB-03-05) within 30 days prior to study enrollment. · The patient is unwilling or unable to travel to the primary site for periodic assessments.
|
|
E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint, change in the number of metres walked in the 12-minute walk test, will be compared between two treatment groups. Differences between the rhASB/rhASB and placebo/rhASB groups with respect to the change from baseline (beginning of Study ASB-03-05) to Week 48 of the extension study and the change from Week 24 (end of Study ASB-03 05) to Week 48 of the extension study will be tested using analysis of variance models with the number of metres walked at baseline as the only covariate. Similar analyses will be performed for secondary and tertiary efficacy endpoints. |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | Information not present in EudraCT |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Information not present in EudraCT |
E.6.7 | Pharmacodynamic | Information not present in EudraCT |
E.6.8 | Bioequivalence | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
E.6.10 | Pharmacogenetic | Information not present in EudraCT |
E.6.11 | Pharmacogenomic | Information not present in EudraCT |
E.6.12 | Pharmacoeconomic | Information not present in EudraCT |
E.6.13 | Others | Information not present in EudraCT |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Information not present in EudraCT |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Information not present in EudraCT |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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.8.5 | The trial involves multiple Member States | Yes |
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 |
|
E.8.7 | Trial has a data monitoring committee | Information not present in EudraCT |
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 | 4 |
E.8.9.1 | In the Member State concerned months | 2 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 2 |