E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Hemolytic disease of the newborn due to red blood cell alloimmunization |
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E.1.1.1 | Medical condition in easily understood language |
Anemia in the newborn due to red cell antibodies received from the mother (for example rhesus D hemolytic disease) |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10019513 |
E.1.2 | Term | Hemolytic disease of fetus or newborn, due to isoimmunization |
E.1.2 | System Organ Class | 100000004851 |
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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 investigate whether darbepoetin alfa is effective in reducing the incidence of late anemia in infants with alloimmune hemolytic disease treated with intrauterine transfusion and therefore in decreasing the percentage of infants that required a top-up transfusions. |
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E.2.2 | Secondary objectives of the trial |
As secondary objectives the number of top-up transfusions per infant will be assessed, as well as the number of days of admission for top-up transfusions, reduction in ferritin levels and long-term neurodevelopmental outcome at 2 years of age using the BSID-III test. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
All (near)-term neonates (gestational age ≥ 35 weeks) with HDN (due to Rhesus-D, -C, -c, -E, Kell or other red blood cell alloimmunization) treated with intrauterine transfusion and admitted to the Leiden University Medical Center (LUMC) are eligible for the study. The LUMC is the single national referral center in the Netherlands for pregnancies complicated by maternal red blood cell alloimmunization. |
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E.4 | Principal exclusion criteria |
Gestational age less than 35 weeks |
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E.5 End points |
E.5.1 | Primary end point(s) |
An absolute reduction of 50% of top-up transfusions. The number of top-up transfusions required per infant up to 3 months of life is the primary outcome measure. |
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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) |
The number of top-up transfusions per infant will be assessed, as well as the number of days of admission for top-up transfusions, reduction in ferritin levels and long-term neurodevelopmental outcome at 2 years of age using the BSID-III test. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
3 months after birth and neurodevelopmental outcome at two years of age. |
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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 | Yes |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Long term effects (eg. on neurodevelopmental outcome) |
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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 | Yes |
E.8.1.1 | Randomised | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
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E.8.2.4 | Number of treatment arms in the trial | 2 |
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.8.5 | 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 | 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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The end of the trial will be after the last visit of the last subject at the age of two years, although the results of the primary end point will be identified 3 months after inclusion of the last subject. |
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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 | |
E.8.9.1 | In the Member State concerned days | |