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Monoclonal Antibodies

Ravulizumab for Thrombotic Microangiopathy

Phase 3
Recruiting
Research Sponsored by Alexion
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Body weight ≥ 30 kilograms at Screening
Received HSCT within the past 12 months
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 26 weeks (treatment period)
Awards & highlights

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.

Who is the study for?
This trial is for adults and teens (12+) who've had a stem cell transplant in the last year and are now facing a complication called TMA that hasn't improved after initial treatment. They must weigh at least 30 kg, be vaccinated against meningococcal infections or on antibiotics if they can't get the vaccine, and use effective birth control if necessary. People with certain heart conditions, severe infections, HIV, or previous complement inhibitor treatments can't join.Check my eligibility
What is being tested?
The study tests Ravulizumab's effectiveness for HSCT-TMA patients. Initially, everyone gets Ravulizumab to confirm dosing (Stage 1). Then participants are randomly assigned to either continue with Ravulizumab plus supportive care or switch to a placebo plus supportive care for six months (Stage 2), followed by another six months of monitoring.See study design
What are the potential side effects?
Ravulizumab may cause headaches, nausea, high blood pressure, fever; it also increases infection risk due to its effect on the immune system. The exact side effects will be closely monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I weigh at least 30 kilograms.
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I had a stem cell transplant within the last year.
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I am 12 years old or older.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~26 weeks (treatment period)
This trial's timeline: 3 weeks for screening, Varies for treatment, and 26 weeks (treatment period) for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
TMA Response
Secondary outcome measures
Change from Baseline in eGFR
Hematologic Response
Non-relapse Mortality
+2 more

Side effects data

From 2022 Phase 3 trial • 195 Patients • NCT03056040
31%
Headache
30%
Nasopharyngitis
28%
Upper respiratory tract infection
21%
Fatigue
19%
Diarrhoea
19%
Pyrexia
18%
Nausea
17%
Cough
15%
Abdominal pain
14%
Back pain
14%
Dizziness
13%
Pain in extremity
11%
Arthralgia
11%
Influenza like illness
10%
Oropharyngeal pain
10%
Rhinitis
8%
Vomiting
8%
Abdominal pain upper
8%
Dyspnoea
8%
Urinary tract infection
8%
Anaemia
7%
Constipation
6%
Chest pain
6%
Dysphagia
5%
Gastroenteritis
5%
Pruritus
5%
Myalgia
5%
Palpitations
5%
Influenza
3%
Haemolysis
2%
Lower respiratory tract infection
2%
Haemolytic anaemia
1%
Basal cell carcinoma
1%
Hyperthermia
1%
Cholelithiasis
1%
Foot deformity
1%
Colitis
1%
Bone marrow failure
1%
Infection
1%
Pneumonia
1%
Post procedural infection
1%
Liver disorder
1%
Depression
1%
Epilepsy
1%
Respiratory failure
1%
Enteritis
1%
Pneumoperitoneum
1%
Toothache
1%
Bile duct stone
1%
Biliary colic
1%
Cholecystitis
1%
COVID-19
1%
Bacteraemia
1%
Escherichia sepsis
1%
Escherichia urinary tract infection
1%
Pneumonia bacterial
1%
Postoperative wound infection
1%
Rhinovirus infection
1%
Septic shock
1%
Ankle fracture
1%
Ligament injury
1%
Transfusion reaction
1%
Cerebrospinal fluid retention
1%
Loss of consciousness
1%
Dupuytren's contracture
1%
Intervertebral disc degeneration
1%
Osteonecrosis
1%
Ureterolithiasis
1%
Urinary retention
1%
Major depression
1%
Suicide attempt
1%
Dermal cyst
1%
Invasive papillary breast carcinoma
1%
Aplastic anaemia
1%
Breakthrough haemolysis
1%
Tibia fracture
1%
Lower limb fracture
1%
Deep vein thrombosis
1%
Endometrial cancer
1%
Lung cancer metastatic
1%
Renal cancer metastatic
1%
Seborrhoeic keratosis
1%
Pharyngitis
1%
Pneumococcal infection
1%
Liver function test increased
1%
Road traffic accident
1%
Suspected COVID-19
100%
80%
60%
40%
20%
0%
Study treatment Arm
Ravulizumab
Eculizumab

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: RavulizumabExperimental Treatment2 Interventions
In Stage 1, all participants will receive open-label ravulizumab plus Best Supportive Care (BSC). In Stage 2, participants will receive blinded ravulizumab plus Best Supportive Care (BSC).
Group II: PlaceboPlacebo Group2 Interventions
In Stage 2, participants randomized to the placebo arm will receive matching placebo plus BSC.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ravulizumab
2021
Completed Phase 4
~1080

Find a Location

Who is running the clinical trial?

AlexionLead Sponsor
246 Previous Clinical Trials
39,171 Total Patients Enrolled
Alexion Pharmaceuticals, Inc.Lead Sponsor
253 Previous Clinical Trials
41,096 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,317 Total Patients Enrolled

Media Library

Ravulizumab (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT04543591 — Phase 3
Thrombotic Microangiopathy Research Study Groups: Placebo, Ravulizumab
Thrombotic Microangiopathy Clinical Trial 2023: Ravulizumab Highlights & Side Effects. Trial Name: NCT04543591 — Phase 3
Ravulizumab (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04543591 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many test sites are there for this experiment?

"At the moment, there are 9 hospitals running this trial. They are situated in Toronto, Charlotte, Valhalla and 6 other cities. If you want to reduce travel as much as possible, try to choose a location close to where you live."

Answered by AI

Is Ravulizumab a new medication?

"The first clinical trials for ravulizumab were in 2016. To date, there have been 18301 completed studies and 16 that are actively recruiting patients. Many of the ongoing recruitment drives for this medication are based out of Toronto or North carolina."

Answered by AI

What medical condition is Ravulizumab most often used to treat?

"While most commonly used to manage disease activity, ravulizumab can also help patients with hemolysis, thrombotic microangiopathies, and atypical hemolytic uremic syndrome."

Answered by AI

How many people are included in this research project?

"The correct. According to the listing on clinicaltrials.gov, this trial is currently looking for patients. This particular study was posted on September 16th 2020 and updated most recently on August 1st 2022. There are a total of 9 different recruitment sites hoping to enroll 184 individuals in total."

Answered by AI

Has this clinical trial been done before?

"Ravulizumab has been under investigation since 2016 when the first clinical trial, which was sponsored by Alexion Pharmaceuticals Inc., took place. In total there are now 16 active trials in 31 different countries."

Answered by AI

Has Ravulizumab received FDA clearance?

"There is some evidence to support the efficacy of Ravulizumab, as this medication has progressed to a Phase 3 clinical trial. Furthermore, multiple rounds of testing have demonstrated that Ravulizumab is safe for human use, and it thus receives a score of 3."

Answered by AI

Are people still able to sign up for this experiment?

"The data on clinicaltrials.gov does indeed suggest that this trial is actively recruiting patients. The study was originally posted on 9/16/2020 and last edited on 8/1/2022. A total of 184 patients are needed for the study, which will be conducted at 9 different locations."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant
2020. "Ravulizumab in Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplant". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04543591.
~18 spots leftby Dec 2024
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