Ravulizumab for Thrombotic Microangiopathy

Phase-Based Progress Estimates
2
Effectiveness
3
Safety
Thrombotic MicroangiopathyRavulizumab - Biological
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

This trial will study whether a new drug, ravulizumab, is effective and safe for treating a condition called HSCT-TMA. The trial will have two stages: in the first stage, participants will receive the drug to see what dose works best; in the second stage, participants will be randomly assigned to receive either the drug or a placebo, and the effects of the drug will be compared to the placebo. The trial will last for 52 weeks total, with 26 weeks of treatment and 26 weeks of follow-up.

Eligible Conditions
  • Thrombotic Microangiopathy

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 8 Secondary · Reporting Duration: During the Follow-up Period (183-365 Days after start of study medication)

26 weeks (treatment period)
Proportion of Participants with a Loss of TMA Response
TMA Response
Week 52
Change from Baseline in eGFR
Hematologic Response
Non-relapse Mortality
Overall Survival
Platelet Response
Time To TMA Response
Day 365
TMA Relapse

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

Eculizumab
20%Nasopharyngitis
17%Headache
10%Cough
10%Upper respiratory tract infection
9%Abdominal pain
9%Oropharyngeal pain
9%Nausea
9%Chest pain
8%Influenza like illness
7%Dizziness
7%Diarrhoea
6%Fatigue
6%Dyspnoea
5%Constipation
5%Musculoskeletal pain
4%Vomiting
4%Pain in extremity
4%Rhinitis
3%Pyrexia
3%Anaemia
2%Haemolysis
1%Palpitations
1%Cholelithiasis
1%Pyelonephritis acute
This histogram enumerates side effects from a completed 2022 Phase 3 trial (NCT03056040) in the Eculizumab ARM group. Side effects include: Nasopharyngitis with 20%, Headache with 17%, Cough with 10%, Upper respiratory tract infection with 10%, Abdominal pain with 9%.

Trial Design

2 Treatment Groups

Ravulizumab
1 of 2
Placebo
1 of 2

Experimental Treatment

Non-Treatment Group

184 Total Participants · 2 Treatment Groups

Primary Treatment: Ravulizumab · Has Placebo Group · Phase 3

RavulizumabExperimental Group · 2 Interventions: Best supportive care, Ravulizumab · Intervention Types: Other, Biological
PlaceboPlaceboComparator Group · 2 Interventions: Best supportive care, Placebo · Intervention Types: Other, Other
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: during the follow-up period (183-365 days after start of study medication)

Who is running the clinical trial?

Alexion PharmaceuticalsLead Sponsor
229 Previous Clinical Trials
38,296 Total Patients Enrolled

Eligibility Criteria

Age Any Age · All Participants · 8 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
You are capable of giving informed consent or assent.
Vaccination against meningococcal infections is recommended for all participants.