E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of Alpha-Mannosidosis |
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E.1.1.1 | Medical condition in easily understood language |
Treatment of Alpha-Mannosidosis |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nutritional and Metabolic Diseases [C18] |
MedDRA Classification |
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 overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamazym i.v. treatment of subjects with alpha-Mannosidosis who previously participated in Lamazym-trials. |
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E.2.2 | Secondary objectives of the trial |
Efficacy will be evaluated once yearly to follow the subject’s progress in clinical parameters. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The subject must have participated in previous Lamazym-trials: phase 2b: 2011-004355-40 or phase 3: 2012-000979-17
2. Subject or subjects legally authorized guardian(s) must provide signed, informed consent prior to performing any trial-related activities
3. The subject and his/her guardian(s) must have the ability to comply with the protocol
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E.4 | Principal exclusion criteria |
1. Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial
2. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial
3. Pregnancy: Pregnant women are excluded. Before start of the treatment the investigators will for women of childbearing potential perform a pregnancy test and decide whether or not there is a need for contraception
4. Psychosis; any psychotic disease, also in remission, is an exclusion criteria
5. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial
6. Adult patients who, in the opinion of the investigator, would be unable to give consent, and who does not have any legal protection or guardianship
7. Total IgE >800 IU/mL
8. Known allergy to the IMP or any excipients (Sodium-Phosphate, Glycine, Mannitol)
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety Endpoints:
• Adverse events (AEs)
• Vital signs and change in physical examination
• Clinical laboratory parameters (hematology, biochemistry and urinalysis)
• Development of Lamazym antibodies and neutralizing/inhibitory antibodies
Efficacy Endpoints:
• 3-minute stair climb test (3MSCT)
• Leiter R, equivalent age
• Pulmonary Function Testing (PFT)
• 6 minute walk test (6MWT)
• Pure tone audiometry (PTA)
• Questionnaires (CHAQ and EQ-5D)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Safety endpoints will be assessed at every visits.
Efficacy endpoints are evaluated as change from baseline (pre-treatment in the subject’s original Lamazym trial), once a year (3 visits total) at the Danish site. |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | 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 | 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 | Yes |
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.8.3 |
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.8.4.1 | 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 | 3 |
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 | No |
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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Last patient last visit is the date when all subjects have completed their EOT visit and this is defined as the End of Trial date. |
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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 | 3 |
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 | 3 |
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 |