E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
mucocopolysaccharidoses I |
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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 | 10056886 |
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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 |
1. To determine whether Aldurazyme affects lactation in women with MPS I. 2. To determine whether α-L-iduronidase is present in the breast milk of mothers with MPS I who are being treated with Aldurazyme. 3. To determine whether Aldurazyme affects the growth, development, and immunologic response of breastfed infants born to mothers with MPS I who are being treated with Aldurazyme. |
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E.2.2 | Secondary objectives of the trial |
There are no secondary objectives |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Mothers must meet the following criteria: 1. Be pregnant, planning to breastfeed postpartum, and receiving Aldurazyme while breastfeeding. 2. Provide signed, written informed consent prior to any protocol-related procedures. Consent of a legally authorized guardian is required for mothers younger than 18 years of age. If a mother is younger than 18 years of age and can understand the consent, written informed consent is required from both the mother and the authorized guardian. 3. Provide signed, written informed consent for their infants to participate as study subjects. If a mother is younger than 18 years of age, consent for mother and infant will be obtained from the legal guardian. |
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E.4 | Principal exclusion criteria |
Mothers (and their infants) will be excluded from this study if any of the following exclusion criteria are met: 1. Are unwilling to breastfeed. 2. Have a medical condition, serious intercurrent illness, or other extenuating circumstance that may interfere with study compliance, including all prescribed evaluations and follow-up activities. 3. Have an acute illness that requires surgical intervention, and/or anticipates surgery during study participation. 4. Have received an investigational drug within 30 days prior to study enrollment. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The analyses of the data from this study are designed to address the following questions: • Does Aldurazyme affect lactation in women with MPS I? • Is α-L-iduronidase present in the breast milk of treated mothers? • Does Aldurazyme have any effects on the growth, development, and immunologic response of infants born to mothers receiving Aldurazyme? Effects of Aldurazyme on lactation in mothers will be assessed in terms of the proportion of lactating women with serum IgG antibodies to laronidase, and the levels of IgG antibody titers to laronidase, the proportion of women choosing to breastfeed and who are successful at breastfeeding, the proportion of women whose breast milk contains α-L-iduronidase, the levels of a α-L-iduronidase in the breast milk of lactating MPS I mothers, and medical history and physical examination findings. Effects of Aldurazyme on infants will be assessed in terms of the proportion of infants attempting to and successfully breastfeeding, the proportion of infants with IgM and IgG antibodies present at any time point during the 12 to 18 month study period, the levels of IgG and IgM antibody titers to laronidase, medical history and physical and developmental examination findings (including growth), and the time to development of IgM and IgG antibodies. |
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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 | No |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | No |
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.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 2 |
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 |
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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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dell`ultima visita dell`ultimo soggetto |
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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 | 10 |
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 |