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Be Part of Research - Trial Details - Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Not Recruiting

Open to: ALL

Age: N/A - 17.0

Medical Conditions

Vascular Diseases
Thrombotic Microangiopathies


This information is provided directly by researchers, and we recognise that it isn't always easy to understand. We are working with researchers to improve the accessibility of this information. In some summaries, you may come across links to external websites. These websites will have more information to help you better understand the study.


This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to \< 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

Start dates may differ between countries and research sites. The research team are responsible for keeping the information up-to-date.  

The recruitment start and end dates are as follows:

Dec 2020 Nov 2024

INTERVENTIONAL

Intervention Type : DRUG
Intervention Description : Weight-based doses of ravulizumab will be administered intravenously as a loading dose regimen followed by maintenance dosing every 4 or 8 weeks, depending upon weight.

Intervention Arm Group : Ravulizumab plus Best Supportive Care;

Intervention Type : OTHER
Intervention Description : Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).

Intervention Arm Group : Ravulizumab plus Best Supportive Care;



You can take part if:



You may not be able to take part if:


This is in the inclusion criteria above


Below are the locations for where you can take part in the trial. Please note that not all sites may be open.

  • Research Site
    Birmingham
    B4 6NH
  • Research Site
    Bristol
    BS2 8BJ
  • Research Site
    Newcastle Upon Tyne
    NE1 4LP


The study is sponsored by Alexion Pharmaceuticals, Inc.




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Read full details for Trial ID: NCT04557735
Last updated 24 March 2025

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