E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Infantile hypoposphatasia |
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E.1.1.1 | Medical condition in easily understood language |
Hypophosphatasia, a bone disorder caused by gene mutations or changes. These gene mutations result in low levels of an enzyme needed to harden bones. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10049933 |
E.1.2 | Term | Hypophosphatasia |
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 |
- To determine the long-term tolerability of subcutaneous (SC) Asfotase alfa
- To assess the long-term efficacy of Asfotase alfa in treating rickets in infants and young children with HPP |
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E.2.2 | Secondary objectives of the trial |
- To assess the long-term pharmacodynamics (PD) of SC Asfotase alfa
- To assess the effect of SC Asfotase alfa on growth and development
- To assess the effect of SC Asfotase alfa on mortality and other clinical signs and symptoms of HPP in infants and young children |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
· Compliant and satisfactory completion, in the opinion of the Sponsor and Investigator, of study
ENB-002-08
· Written informed consent by parent or other legal guardian prior to any study procedures being
performed
· Parent or other legal guardian willing to comply with study requirements |
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E.4 | Principal exclusion criteria |
- The patient has a history of sensitivity to any ENB 0040 constituents
- The patient has a clinically significant disease that precludes study participation, in the Investigator’s opinion
- The patient has been enrolled in any study involving an investigational drug, device, or treatment for HPP (e.g., one marrow transplantation)
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E.5 End points |
E.5.1 | Primary end point(s) |
Skeletal radiograph using a qualitative Radiographic Global Impression of Change (RGI-C) scale.
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Long-term pharmacodynamics of SC Asfotase alfa.
Effect of SC Asfotase alfa on growth and development, mortality, and other clinical signs and symptoms of HPP in infants and young children. |
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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 | Yes |
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) | 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 | Information not present in EudraCT |
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.8.2.4 | Number of treatment arms in the trial | 1 |
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 | No |
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 |
United Arab Emirates |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
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 | 4 |
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 | 4 |
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 |