Trial · NCT03748823

Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab

NCT03748823, Phase 3, Completed, Alexion Pharmaceuticals, Inc.; results first posted 2022-10-20

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab. Trials, record NCT03748823. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03748823

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
NCT number: NCT03748823
Status: Completed
Phases: Phase 3
Enrollment: 139 participants, actual
Lead sponsor: Alexion Pharmaceuticals, Inc.
Conditions: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Ravulizumab OBDS, Ravulizumab
First posted: 2018-11-21
Last updated: 2024-09-19
Other registries' IDs: eudract:2017-002370-39
Results first posted: 2022-10-20
Limitations and caveats: To ensure the quality of the study results, all 7 participants from a noncompliant investigative site were excluded from all prespecified analysis sets due to important source documentation deviations.

Source ClinicalTrials.gov

Registered outcomes16

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Ctrough Serum Concentration of Ravulizumab	Predose at Day 71
Secondary	Ctrough Serum Concentration of Ravulizumab at Day 351	Predose at Day 351
Secondary	Free Serum Complement Component 5 (C5) Concentrations at Day 71	Predose at Day 71
Secondary	Free Serum Complement Component 5 (C5) Concentrations at Day 351	Predose at Day 351
Secondary	Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71	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.
Secondary	Percent Change From Baseline in LDH Levels at Day 351	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.
Secondary	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at Day 71	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.
Secondary	Change From Baseline in FACIT-Fatigue Scale Version 4 Score at Day 351	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.
Secondary	Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 71	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.
Secondary	Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 351	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.

1 to 10 of 16Source ClinicalTrials.gov

See all 16 registered outcomes

Outcomes16

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Ctrough Serum Concentration of Ravulizumab	Predose at Day 71	Pharmacokinetic (PK) analysis set included all participants who had evaluable PK data.	micrograms/milliliter (µg/mL)	MEAN
SECONDARY	Ctrough Serum Concentration of Ravulizumab at Day 351	Predose at Day 351	SC treated full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab SC, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	µg/mL	MEAN
SECONDARY	Free Serum Complement Component 5 (C5) Concentrations at Day 71	Predose at Day 71	Pharmacodynamic (PD) analysis set included all participants who received at least 1 dose of ravulizumab and who had evaluable PD data. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	µg/mL	MEAN
SECONDARY	Free Serum Complement Component 5 (C5) Concentrations at Day 351	Predose at Day 351	SC treated full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab SC, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	µg/mL	MEAN
SECONDARY	Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71	Baseline, Day 71	Full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	percent change	MEAN
SECONDARY	Percent Change From Baseline in LDH Levels at Day 351	Baseline, Day 351	SC treated full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab SC, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	percent change	MEAN
SECONDARY	Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at Day 71	Baseline, Day 71	Full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	units on a scale	MEAN
SECONDARY	Change From Baseline in FACIT-Fatigue Scale Version 4 Score at Day 351	Baseline, Day 351	SC treated full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab SC, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure. This outcome measure was planned to be reported for ravulizumab SC/SC treatment group only.	units on a scale	MEAN
SECONDARY	Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 71	Baseline, Day 71	Full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab, and were not excluded from analysis. Here, Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.	units on a scale	MEAN
SECONDARY	Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 351	Baseline, Day 351	SC treated full analysis set included all participants who had signed informed consent, were randomized, received at least 1 dose of ravulizumab SC, and were not excluded from analysis. Here, Number of Participants analyzed signifies those participants who were evaluable for this outcome measure. This outcome measure was planned to be reported for ravulizumab SC/SC treatment group only.	units on a scale	MEAN

1 to 10 of 16Source ClinicalTrials.gov

See all 16 outcomes

Results by group30

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Ctrough Serum Concentration of Ravulizumab	Ravulizumab IV/SC Treatment Group	457.58	Standard Deviation 108.491	micrograms/milliliter (µg/mL)
Ctrough Serum Concentration of Ravulizumab	Ravulizumab SC/SC Treatment Group	578.70	Standard Deviation 140.819	micrograms/milliliter (µg/mL)
Ctrough Serum Concentration of Ravulizumab at Day 351	Ravulizumab IV/SC Treatment Group	712.79	Standard Deviation 203.180	µg/mL
Ctrough Serum Concentration of Ravulizumab at Day 351	Ravulizumab SC/SC Treatment Group	737.65	Standard Deviation 208.894	µg/mL
Free Serum Complement Component 5 (C5) Concentrations at Day 71	Ravulizumab IV/SC Treatment Group	0.072193	Standard Deviation 0.0245225	µg/mL
Free Serum Complement Component 5 (C5) Concentrations at Day 71	Ravulizumab SC/SC Treatment Group	0.059458	Standard Deviation 0.0182180	µg/mL
Free Serum Complement Component 5 (C5) Concentrations at Day 351	Ravulizumab IV/SC Treatment Group	0.071627	Standard Deviation 0.0227980	µg/mL
Free Serum Complement Component 5 (C5) Concentrations at Day 351	Ravulizumab SC/SC Treatment Group	0.069711	Standard Deviation 0.0208784	µg/mL
Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71	Ravulizumab IV/SC Treatment Group	5.73	Standard Deviation 29.716	percent change
Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71	Ravulizumab SC/SC Treatment Group	2.57	Standard Deviation 33.883	percent change

1 to 10 of 30Source ClinicalTrials.gov

See all 30 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Randomized Treatment Period	STARTED	Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group	46
Randomized Treatment Period	STARTED	Ravulizumab SC/SC Treatment Group	90
Randomized Treatment Period	Received at Least 1 Dose of Study Drug	Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group	46
Randomized Treatment Period	Received at Least 1 Dose of Study Drug	Ravulizumab SC/SC Treatment Group	90
Randomized Treatment Period	Treated and Not Included in Analysis	Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group	1
Randomized Treatment Period	Treated and Not Included in Analysis	Ravulizumab SC/SC Treatment Group	6
Randomized Treatment Period	Full Analysis Set	Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group	45
Randomized Treatment Period	Full Analysis Set	Ravulizumab SC/SC Treatment Group	84
Randomized Treatment Period	Safety Analysis Set	Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group	45
Randomized Treatment Period	Safety Analysis Set	Ravulizumab SC/SC Treatment Group	84

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events202

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Anaemia	Blood and lymphatic system disorders	Ravulizumab IV/SC Treatment Group	4	45
other	Anaemia	Blood and lymphatic system disorders	Ravulizumab SC/SC Treatment Group	12	84
other	Haemolysis	Blood and lymphatic system disorders	Ravulizumab IV/SC Treatment Group	4	45
other	Haemolysis	Blood and lymphatic system disorders	Ravulizumab SC/SC Treatment Group	10	84
other	Diarrhoea	Gastrointestinal disorders	Ravulizumab IV/SC Treatment Group	3	45
other	Diarrhoea	Gastrointestinal disorders	Ravulizumab SC/SC Treatment Group	17	84
other	Abdominal pain	Gastrointestinal disorders	Ravulizumab IV/SC Treatment Group	6	45
other	Abdominal pain	Gastrointestinal disorders	Ravulizumab SC/SC Treatment Group	10	84
other	Nausea	Gastrointestinal disorders	Ravulizumab IV/SC Treatment Group	5	45
other	Nausea	Gastrointestinal disorders	Ravulizumab SC/SC Treatment Group	9	84

1 to 10 of 202Source ClinicalTrials.gov

See all 202 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Ravulizumab IV/SC Treatment Group	2	45
Total, all-cause mortality	Ravulizumab SC/SC Treatment Group	2	84
Total, other adverse events	Ravulizumab IV/SC Treatment Group	44	45
Total, other adverse events	Ravulizumab SC/SC Treatment Group	83	84
Total, serious adverse events	Ravulizumab IV/SC Treatment Group	16	45
Total, serious adverse events	Ravulizumab SC/SC Treatment Group	30	84

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
36272026	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Ravulizumab IV/SC Treatment Group vs Ravulizumab SC/SC Treatment Group: deaths from any cause	Trial comparisons
Ravulizumab IV/SC Treatment Group vs Ravulizumab SC/SC Treatment Group: serious adverse events	Trial comparisons

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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