Ctrough Serum Concentration of Ravulizumab
Primary
· Predose at Day 71
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 457.58 | ± 108.491 |
| Ravulizumab SC/SC Treatment Group | 578.70 | ± 140.819 |
Last reviewed · How we verify
NCT03748823
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
Completed
Phase 3
Results posted
Last updated 19 September 2024
What this trial tests
Phase 3 trial testing Ravulizumab OBDS in Paroxysmal Nocturnal Hemoglobinuria in 139 participants. Completed in 31 August 2023.
Timeline
19 February 2019
Primary endpoint
2 February 2021
2 February 2021
31 August 2023
Quick facts
| Lead sponsor | Alexion Pharmaceuticals, Inc. |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 139 |
| Start date | 19 February 2019 |
| Primary completion | 2 February 2021 |
| Estimated completion | 31 August 2023 |
| Sites | 51 locations across France, Italy, Finland, Netherlands, Russia, Belgium, Austria, Sweden |
Drugs / interventions tested
- Ravulizumab OBDS
- Ravulizumab (RAVULIZUMAB) — full drug profile →
Conditions studied
- Paroxysmal Nocturnal Hemoglobinuria — all drugs for Paroxysmal Nocturnal Hemoglobinuria →
Sponsor
Alexion Pharmaceuticals, Inc. — full company profile →
Who can join
18 and older, any sex, with Paroxysmal Nocturnal Hemoglobinuria. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Ctrough Serum Concentration of Ravulizumab at Day 351
Secondary
· Predose at Day 351
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 712.79 | ± 203.180 |
| Ravulizumab SC/SC Treatment Group | 737.65 | ± 208.894 |
Free Serum Complement Component 5 (C5) Concentrations at Day 71
Secondary
· Predose at Day 71
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 0.072193 | ± 0.0245225 |
| Ravulizumab SC/SC Treatment Group | 0.059458 | ± 0.0182180 |
Free Serum Complement Component 5 (C5) Concentrations at Day 351
Secondary
· Predose at Day 351
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 0.071627 | ± 0.0227980 |
| Ravulizumab SC/SC Treatment Group | 0.069711 | ± 0.0208784 |
Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71
Secondary
· Baseline, Day 71
Baseline was defined as the last assessment prior to first study drug dose. LDH samples impacted by tabletop hemolysis were excluded from the analysis.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 5.73 | ± 29.716 |
| Ravulizumab SC/SC Treatment Group | 2.57 | ± 33.883 |
Percent Change From Baseline in LDH Levels at Day 351
Secondary
· Baseline, Day 351
SC baseline was defined as the last assessment prior to first dose of SC treatment. LDH samples impacted by tabletop hemolysis were excluded from the analysis.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | -0.83 | ± 17.225 |
| Ravulizumab SC/SC Treatment Group | 1.74 | ± 21.905 |
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at Day 71
Secondary
· Baseline, Day 71
FACIT-fatigue subscale is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Items are scored on a 5 point Likert-type scale. Item scores ranged from 0 ("not at all") to 4 ("very much"). The total, summed score ranged from 0 to 52; lower scores indicating greater fatigue and higher score indicating better health-related quality of life. Baseline was defined as the last non-missing value prior to the first dose of study drug.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | -0.83 | ± 7.378 |
| Ravulizumab SC/SC Treatment Group | 1.21 | ± 7.882 |
Change From Baseline in FACIT-Fatigue Scale Version 4 Score at Day 351
Secondary
· Baseline, Day 351
FACIT-fatigue subscale is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Items are scored on a 5 point Likert-type scale. Item scores ranged from 0 ("not at all") to 4 ("very much"). The total, summed score ranged from 0 to 52; lower scores indicating greater fatigue and higher score indicating better health-related quality of life. Baseline was defined as the last non-missing value prior to the first dose of subcutaneous treatment.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab SC/SC Treatment Group | 2.57 | ± 7.178 |
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 71
Secondary
· Baseline, Day 71
The Treatment Administration Satisfaction Questionnaire (TASQ) is a 19-item questionnaire that assesses treatment administration satisfaction across 5 domains: physical impact, psychological impact, impact on activities of daily living, convenience, and satisfaction. Each domain offers up to 5 response options; lower scores indicate a more positive response. Scoring is completed by summing each of the 5 domains. Total TASQ scores during the study ranged from 0 to 367, with a lower score indicating greater satisfaction with treatment administration.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | -7.00 | ± 34.581 |
| Ravulizumab SC/SC Treatment Group | -70.54 | ± 70.522 |
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 351
Secondary
· Baseline, Day 351
The Treatment Administration Satisfaction Questionnaire (TASQ) is a 19-item questionnaire that assesses treatment administration satisfaction across 5 domains: physical impact, psychological impact, impact on activities of daily living, convenience, and satisfaction. Each domain offers up to 5 response options; lower scores indicate a more positive response. Scoring is completed by summing each of the 5 domains. Total TASQ scores during the study ranged from 0 to 367, with a lower score indicating greater satisfaction with treatment administration.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab SC/SC Treatment Group | -69.29 | ± 80.068 |
Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 71
Secondary
· Baseline up to Day 71
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 grams/deciliter (g/dL)\], major adverse vascular event \[MAVE, including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2\*upper limit of normal (ULN). Denominator for a percentage was participants with at least one post-baseline data for the period. For Through Day 71, only visits with data were used to assess breakthrough hemolysis.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 2.2 | 0.06 – 11.77 |
| Ravulizumab SC/SC Treatment Group | 1.2 | 0.03 – 6.46 |
Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 351
Secondary
· Baseline up to Day 351
Breakthrough hemolysis was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 g/dL\], major adverse vascular event \[MAVE, including thrombosis\], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2\*ULN. Denominator for a percentage was participants with at least one post-baseline data for the period.
| Group | Value | 95% CI |
|---|---|---|
| Ravulizumab IV/SC Treatment Group | 4.5 | 0.56 – 15.47 |
| Ravulizumab SC/SC Treatment Group | 3.6 | 0.74 – 10.08 |
Adverse events — posted to ClinicalTrials.gov
Time frame: Baseline up to approximately 3.5 years. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Ravulizumab IV/SC Treatment Group
Serious: 16/45 (36%)
Deaths: 2/45
Ravulizumab SC/SC Treatment Group
Serious: 30/84 (36%)
Deaths: 2/84
Serious adverse events (59 terms)
| Reaction | System | Ravulizumab IV/SC Treatmen… | Ravulizumab SC/SC Treatmen… |
|---|---|---|---|
| COVID-19 | Infections and infestations | — | — |
| Haemolysis | Blood and lymphatic system disorders | — | — |
| Anaemia | Blood and lymphatic system disorders | — | — |
| Aplastic anaemia | Blood and lymphatic system disorders | — | — |
| Cholecystitis | Hepatobiliary disorders | — | — |
| COVID-19 pneumonia | Infections and infestations | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Haemolytic anaemia | Blood and lymphatic system disorders | — | — |
| Neutropenia | Blood and lymphatic system disorders | — | — |
| Lens dislocation | Eye disorders | — | — |
| Gastritis | Gastrointestinal disorders | — | — |
| Pyrexia | General disorders | — | — |
| Application site induration | General disorders | — | — |
| Fatigue | General disorders | — | — |
| Cholangitis | Hepatobiliary disorders | — | — |
| Cholecystitis acute | Hepatobiliary disorders | — | — |
| Bacterial infection | Infections and infestations | — | — |
| Bacterial sepsis | Infections and infestations | — | — |
| Gastroenteritis | Infections and infestations | — | — |
| Hepatitis viral | Infections and infestations | — | — |
| Salmonellosis | Infections and infestations | — | — |
| Sinusitis | Infections and infestations | — | — |
| Suspected COVID-19 | Infections and infestations | — | — |
| Tubo-ovarian abscess | Infections and infestations | — | — |
Other adverse events (42 terms — click to expand)
| Reaction | System | Ravulizumab IV/SC Treatmen… | Ravulizumab SC/SC Treatmen… |
|---|---|---|---|
| Device delivery system issue | Product Issues | — | — |
| COVID-19 | Infections and infestations | — | — |
| Pyrexia | General disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Headache | Nervous system disorders | — | — |
| Asthenia | General disorders | — | — |
| Anaemia | Blood and lymphatic system disorders | — | — |
| Nasopharyngitis | Infections and infestations | — | — |
| Urinary tract infection | Infections and infestations | — | — |
| Back pain | Musculoskeletal and connective tissue disorders | — | — |
| Haemolysis | Blood and lymphatic system disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Influenza | Infections and infestations | — | — |
| Toothache | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Pain in extremity | Musculoskeletal and connective tissue disorders | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Influenza like illness | General disorders | — | — |
| Injection site erythema | General disorders | — | — |
| Fatigue | General disorders | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — |
| Neutropenia | Blood and lymphatic system disorders | — | — |
| Post vaccination fever | Injury, poisoning and procedural complications | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — |
| Dizziness | Nervous system disorders | — | — |
| Gastroenteritis | Infections and infestations | — | — |
| Insomnia | Psychiatric disorders | — | — |
| Anxiety | Psychiatric disorders | — | — |
| Cholelithiasis | Hepatobiliary disorders | — | — |
| Infusion site erythema | General disorders | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Dyspepsia | Gastrointestinal disorders | — | — |
| Paronychia | Infections and infestations | — | — |
| Depression | Psychiatric disorders | — | — |
| Haemoglobinuria | Renal and urinary disorders | — | — |
| Intermenstrual bleeding | Reproductive system and breast disorders | — | — |
Most-reported serious reactions: COVID-19, Haemolysis, Anaemia, Aplastic anaemia, Cholecystitis, COVID-19 pneumonia, Febrile neutropenia, Thrombocytopenia.
Data from ClinicalTrials.gov NCT03748823 adverse events section.
Sponsor's own description
The primary objective of this study is to evaluate pharmacokinetics (PK) of ravulizumab administered subcutaneously via an on-body delivery system (OBDS) compared with intravenously administered ravulizumab in adult participants with PNH who are clinically stable on eculizumab for at least 3 months prior to study entry.
Publications & conference data
6 peer-reviewed publications reference this trial (live from Europe PMC):
-
The importance of terminal complement inhibition in paroxysmal nocturnal hemoglobinuria.
Kulasekararaj AG, Brodsky RA, Nishimura JI, Patriquin CJ, et al · · 2022 · cited 24× · PMID 35663504 · DOI 10.1177/20406207221091046 -
Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up.
Yenerel MN, Sicre de Fontbrune F, Piatek C, Sahin F, et al · · 2023 · cited 18× · PMID 36272026 · DOI 10.1007/s12325-022-02339-3 -
The dysfunction of complement and coagulation in diseases: the implications for the therapeutic interventions.
Jiang H, Guo Y, Wang Q, Wang Y, et al · · 2024 · cited 5× · PMID 39445002 · DOI 10.1002/mco2.785 -
Injection Site Reactions with Long-Term Pegcetacoplan Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: A Brief Report.
Sharma V, Koprivnikar J, Drago K, Savage J, et al · · 2023 · cited 1× · PMID 37707673 · DOI 10.1007/s12325-023-02653-4 -
Abstract Book for the 27th Congress of the European Hematology Association
· 2022 -
P813: EFFICACY, TREATMENT ADMINISTRATION SATISFACTION AND SAFETY OF SUBCUTANEOUS RAVULIZUMAB THROUGH 1 YEAR IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO RECEIVED PRIOR INTRAVENOUS ECULIZUMAB
Yenerel M, Sicre de Fontbrune F, Piatek C, Sahin F, et al · · 2022
Verify or expand the search:
- PubMed search for NCT03748823
- Europe PMC full search
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT03748823 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Alexion Pharmaceuticals, Inc.
- Last refreshed: 19 September 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03748823.
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