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 |
|
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 |
To determine if treatment with rhEPO reduces the need for top-up transfusions in neonates with HDN due to red cell alloimmunization treated with IUT. |
|
E.2.2 | Secondary objectives of the trial |
To determine if treatment with rhEPO reduces the number of days of admission for top-up transfusions and reduces serum ferritin levels. To determine if the group treated with rhEPO has a better longterm neurodevelopmental outcome at 2 years of age (using the BSID-III test). |
|
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 IUT and admitted to the Leiden University Medical Center (LUMC) between January 2011 and January 2014 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. |
|
E.4 | Principal exclusion criteria |
Gestational age less than 35 weeks; neonates not formerly treated with intrauterine transfusion(s). |
|
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 and the percentage of infants requiring a top-up transfusion up to 3 months of life are the primary outcome measures.) |
|
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 | Yes |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
|
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 |
|
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 | Information not present in EudraCT |
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
|
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. |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 5 |
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 | 0 |
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 |