An unusual aHUS trial study ( No.NCT06312644) has begun which involves mothers with aHUS and their babies. The official title of the trial is Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
Unusual because it:
- is for the USA only
- is a long term (10 years) observational Registry
- crosses the four diseases which are treated with ravulizumab PNH, NMSOD, gMG and Primary aHUS.
- there is a call centre for recruitment for patients and doctors
Participants will have been “exposed to ravulizumab” at any point up to 40 weeks prior t conception through pregnancy and/or breastfeeding through the first 52 weeks of the infant’s life.
The study is looking for 75 participants from the four disease cohorts. It originally estimated 300.
Over the duration of the Registry the study will be looking to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to ravulizumab and to describe the frequency and characteristics of selected fetal/neonatal/infant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.
Unlike eculizumab for which there was no trial of its impact on the baby but its use in pregnancy was studied in case studies. Mainly for PNH cases.
There has been a concern from the trials of ravulizumab that the re-engineering of eculizumab to make it last longer in between infusions exposes the baby to potential harm. Doctors initially did not support ravulizumab use in pregnancy but did not really know if it caused harmed or not. There is no evidence that eculizumab has caused harm. So a different treatment strategy was recommended.
Participants in this study must have been or are pregnant while treated with ravulizumab. They must give informed consent to participation and be prepared for their own as well as the baby’s medical information to be given to the Registry. And of course be diagnosed with one of the four conditions. If equally spread there should be opportunities for 18 mothers with aHUS in the USA to participate in this aHUS Trial.
Participants in the trial will not be paid for doing so. Nor will their doctor. Nor will it confer any health care privileges.
Participation will help others with informed advice about pregnancy and ravulizumab in the future. Maybe for those outside of the USA too.
There is limited follow up for the mother of 4 weeks and 52 weeks at designated intervals for the baby. All data will be gathered without any effort needed other than a phone call by the participants.
The study will end in 2034.
It is sponsored by Alexion and the Principle Investigator is Sydney Williams whose credentials are unclear.
The registry has been listed on the FDA’s List of Pregnancy Exposure Registries.
Alexion has a glossy dedicated trial recruitment website. To find out more about participating use this LINK.
Article No. 717
