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Monoclonal Antibodies

Ravulizumab for Paroxysmal Nocturnal Hemoglobinuria

Phase 3
Waitlist Available
Research Sponsored by Alexion Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Timeline
Screening 3 weeks
Treatment Varies
Follow Up day 29 through day 183
Awards & highlights

Study Summary

This trial compares the effectiveness of two drugs in treating a blood disorder called PNH.

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~day 29 through day 183
This trial's timeline: 3 weeks for screening, Varies for treatment, and day 29 through day 183 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percentage Of Participants Who Achieved Transfusion Avoidance (TA)
Proportion Of Participants With Normalization Of Lactate Dehydrogenase (LDH) Levels
Secondary outcome measures
Change From Baseline In Quality Of Life As Assessed By The Functional Assessment Of Chronic Illness Therapy (FACIT)-Fatigue
Percent Change From Baseline In Lactate Dehydrogenase (LDH) Levels
Percentage Of Participants With Breakthrough Hemolysis (BTH)
+1 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
Active Control
Group I: RavulizumabExperimental Treatment1 Intervention
Participants received weight-based doses of ravulizumab ranging from 2400 to 3000 milligram (mg) on Day 1. Thereafter, weight-based doses of ravulizumab ranging from 3000 to 3600 mg were administered on Day 15 and every 8 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 years.
Group II: EculizumabActive Control2 Interventions
Participants received 600 mg of eculizumab on Days 1, 8, 15, and 22, followed by 900 mg of eculizumab on Day 29 and every 2 weeks thereafter for 26 weeks. After completion of the Primary Evaluation Period, all participants had the opportunity to enter the Extension Period, wherein participants will receive weight-based doses of ravulizumab for up to 5 years.
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?

Alexion Pharmaceuticals, Inc.Lead Sponsor
253 Previous Clinical Trials
40,912 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,153 Total Patients Enrolled
AlexionLead Sponsor
246 Previous Clinical Trials
39,007 Total Patients Enrolled

Frequently Asked Questions

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

Has the federal government given its blessing to Ravulizumab?

"Ravulizumab has been shown to be safe in multiple clinical trials, so it received a score of 3."

Answered by AI

What are the unique aspects of this particular clinical trial?

"Currently, there are 25 ongoing Ravulizumab studies in 45 countries and 240 cities. The first clinical trial for the drug was conducted by Alexion Pharmaceuticals in 2016 with 270 participants. As of now, 47 trials have been completed and Phase 3 approval has been granted."

Answered by AI

What are the planned benefits of this clinical test?

"The primary outcome of this study, which will be measured over a Day 29 through Day 183 time frame is to Percentage Of Participants Who Achieved Transfusion Avoidance (TA). Secondary outcomes include Percentage Of Participants With Stabilized Hemoglobin Levels which is defined as Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion through Day 183., Percentage Of Participants With Breakthrough Hemolysis (BTH) which is defined as Breakthrough hemolysis was defined as at least 1 new or worsening symptom or sign of intravascular hem"

Answered by AI

In how many different geographical areas is this study being conducted?

"There are 4 primary enrolment centres for this study, situated in Los Angeles, Toronto, and Fort Worth. There are also other locations that participating patients can choose from to minimize travel requirements."

Answered by AI

How many study participants are involved in this clinical trial?

"This trial is not actively searching for patients at this time. The study was first posted on December 20th, 2016 and was most recently edited on July 14th, 2022. If you are seeking other studies, there are currently 33 clinical trials actively recruiting patients with hemoglobinuria paroxysmal and 25 trials for Ravulizumab actively enrolling patients."

Answered by AI

What are the primary indications for ravulizumab?

"Ravulizumab can be used as a treatment for patients who are complement inhibitor naive, have myasthenia gravis or generalized disease, or neuromyelitis optica."

Answered by AI

Can new participants still join this clinical trial?

"The clinical trial mentioned is not currently looking for patients, as per the information available on clinicaltrials.gov. This particular trial was originally posted on December 20th 2016 and was edited most recently on July 14th 2022. There are 58 other trials which are actively recruiting patients."

Answered by AI

Could you please share what other research studies have been done on Ravulizumab?

"Ravulizumab was first studied in 2016 and, since then, 47 trials have completed. Currently, 25 clinical trials are recruiting patients with a notable number of these taking place in Los Angeles and Texas."

Answered by AI
Recent research and studies
~32 spots leftby Apr 2025