Ravulizumab for Thrombotic Microangiopathy

Recruiting at 116 trial locations

Overseen By Alexion Pharmaceuticals, Inc. (Sponsor)

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Alexion

Must not be taking: Complement inhibitors

Disqualifiers: TTP, HIV, Pregnancy, Sepsis, others

No Placebo Group All 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 Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 26 weeks

41 Participants Needed

Trial Summary

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.

Eligibility Criteria

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 am under 18 years old.

I will use effective birth control from screening until 8 months after my last dose.

My TMA condition continues despite initial treatments.

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.

Timeline

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

Treatment Details

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.

Participant Groups

Group I: Ravulizumab plus Best Supportive Care Experimental Treatment 2 Interventions

Participants will receive ravulizumab plus Best Supportive Care as background therapy.

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

Approved in United States as Ultomiris for:

• Paroxysmal nocturnal hemoglobinuria (PNH)
• Atypical hemolytic uremic syndrome (aHUS)

Approved in European Union as Ultomiris for:

• Paroxysmal nocturnal haemoglobinuria (PNH)

Who Is Running the Clinical Trial?

Alexion

Lead Sponsor

Trials

247

Recruited

38,600+

Marc Dunoyer

Alexion

Chief Executive Officer since 2021

PhD in Molecular Biology, University of Brussels

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

Alexion Pharmaceuticals, Inc.

Chief Medical Officer since 2010

MD from University of Sao Paulo

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

Alexion Pharmaceuticals

Chief Executive Officer since 2021

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

Christophe Hotermans

Alexion Pharmaceuticals

Chief Medical Officer since 2023

MD