41 Participants Needed

Ravulizumab for Thrombotic Microangiopathy

Recruiting at 116 trial locations
AP
AP
Overseen ByAlexion Pharmaceuticals, Inc. (Sponsor)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial will test the safety and effectiveness of ravulizumab, an IV medication, in children with HSCT-TMA. The treatment aims to stop the immune system from damaging blood vessels. Ravulizumab is a longer-acting medication developed to reduce blood cell destruction and improve quality of life in patients with a specific blood condition.

Do I need to stop my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Are You a Good Fit for This Trial?

This trial is for children and teens from 1 month to under 18 years who've had a stem cell transplant in the last year and are suffering from HSCT-TMA. They must be able to consent, use effective birth control if applicable, be vaccinated against meningococcal infections or on antibiotics, weigh at least 5 kg, and not have certain other health conditions.

Inclusion Criteria

I will use effective birth control from screening until 8 months after my last dose.
My TMA condition continues despite initial treatments.
I am vaccinated against meningococcal infections as recommended after my stem cell transplant.
See 5 more

Exclusion Criteria

I have a deficiency in ADAMTS13 (less than 5% activity).
I might have a condition that causes abnormal blood clotting throughout my body.
I have been diagnosed with veno-occlusive disease.
See 9 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive ravulizumab plus Best Supportive Care for 26 weeks

26 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

26 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Ravulizumab
Trial Overview The study tests Ravulizumab given by IV to kids with HSCT-TMA. It looks at how safe it is, how well it works, what the body does with it (pharmacokinetics), and what it does to the body (pharmacodynamics) over a treatment period of 26 weeks plus another 26 weeks of follow-up without treatment.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Ravulizumab plus Best Supportive CareExperimental Treatment2 Interventions

Ravulizumab is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Ultomiris for:
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Approved in European Union as Ultomiris for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Alexion

Lead Sponsor

Trials
247
Recruited
38,600+
Marc Dunoyer profile image

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

Christophe Hotermans profile image

Christophe Hotermans

Alexion

Chief Medical Officer since 2021

MD, University of Leuven

Alexion Pharmaceuticals, Inc.

Lead Sponsor

Trials
267
Recruited
141,000+
Dr. Alberto R. Martinez profile image

Dr. Alberto R. Martinez

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

Marc Dunoyer profile image

Marc Dunoyer

Alexion Pharmaceuticals, Inc.

Chief Executive Officer since 2021

PhD in Molecular Biology

Alexion Pharmaceuticals

Lead Sponsor

Trials
231
Recruited
36,700+
Marc Dunoyer profile image

Marc Dunoyer

Alexion Pharmaceuticals

Chief Executive Officer since 2021

B.A. in Psychology from the University of New Hampshire

Christophe Hotermans profile image

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD

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