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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
Be younger than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 2 (day 15), week 10 (day 71), week 18 (day 127), week 26 (day 183)
Awards & highlights

Study Summary

This trial looked at how well a new drug, ravulizumab, works in children with a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH).

Eligible Conditions
  • Paroxysmal Nocturnal Hemoglobinuria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 2 (day 15), week 10 (day 71), week 18 (day 127), week 26 (day 183)
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 2 (day 15), week 10 (day 71), week 18 (day 127), week 26 (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
Change In Chicken Red Blood Cell (cRBC) Hemolytic Activity Over Time
Change In Free Complement Component C5 (C5) Concentrations Over Time
Maximum Observed Serum Concentration (Cmax) Of Ravulizumab
+3 more
Secondary outcome measures
Change In Quality Of Life (QoL) From Baseline To Week 26
Percentage Change From Baseline At Week 26 In Lactate Dehydrogenase (LDH) Levels
Percentage Change In Free Hemoglobin From Baseline To Week 26
+3 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

1Treatment groups
Experimental Treatment
Group I: RavulizumabExperimental Treatment1 Intervention
Complement inhibitor treatment-naïve and eculizumab-experienced participants received ravulizumab.
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
41,189 Total Patients Enrolled
AlexionLead Sponsor
246 Previous Clinical Trials
39,264 Total Patients Enrolled
Alexion PharmaceuticalsLead Sponsor
230 Previous Clinical Trials
37,410 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria
2018. "A Study of Ravulizumab (ALXN1210) in Children and Adolescents With Paroxysmal Nocturnal Hemoglobinuria". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03406507.
~2 spots leftby Apr 2025
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