A Phase 3 interventional study of Ravulizumab OBDS and Ravulizumab in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 51 sites in 14 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-09-19.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 3, Interventional, and Treatment
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Among 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca — 3 open to participants now, 7 under way, 2 of them phase 3. Hemoglobinuria, Paroxysmal · AstraZeneca.
Among the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, this one is the 4th largest by enrollment of the 23 interventional ones that give a count, is one of 2 that started in 2019, is one of 12 in phase 3, and was the 20th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
Browse Hemoglobinuria, Paroxysmal studies →3 of the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca are open now, in 8 countries. Recruiting first, the newest first:
AstraZeneca · Sites in Korea, Republic of
Alexion Pharmaceuticals, Inc. · Sites in Canada, France, United Kingdom
Ages 12–17
Alexion Pharmaceuticals, Inc. · Sites in United States, Australia, France and 4 more countries
Women only
By country, each count listing its studies:
Of the 30 Hemoglobinuria, Paroxysmal studies recruiting now, 14 (47%) set no upper age limit and 12 (40%) stop at 75 or younger; 12 take children under 18; 4 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
35 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 2 · Phase 2 8 · Phase 3 12 · Phase 4 2
First in 2004; this study’s year is marked. 2 started in 2019.
This study's enrollment of 139 is above the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Median enrollment 26 across the 23 interventional studies, 75 across the 10 observational. Of the 26 that completed or were terminated, 18 (69%) have results posted.
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This study is registered under Alexion Pharmaceuticals, Inc., one of the names counted as AstraZeneca. Under that name alone:
Alexion Pharmaceuticals, Inc. is the lead sponsor of 255 studies on the registry; 23 are open to participants now.
Of its 101 completed or terminated interventional studies of FDA-regulated products, 75 (74%) have results posted.
1 of the studies in Hemoglobinuria, Paroxysmal by AstraZeneca that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06449001Phase 3Alexion Pharmaceuticals, Inc.Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis | 2027-05-28 |
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Among the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, from their registry updates:
Nothing recorded among them since 2026-09-25.
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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.
The study will consist of up to a 30-day Screening Period, a 10-week Randomized Treatment Period, and an Extension Period of up to 172 weeks.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by SC maintenance doses of ravulizumab administered via the ravulizumab OBDS on Day 15 and every week (qw) thereafter for a total of 10 weeks of study treatment. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
Combination Product: Ravulizumab OBDS · Biological: Ravulizumab
In the Randomized Treatment Period, participants will receive an IV loading dose of ravulizumab on Day 1 followed by IV maintenance doses of ravulizumab on Day 15. In the Extension Period, ravulizumab SC will be administered via the ravulizumab OBDS from Day 71 qw through Day 1274.
Combination Product: Ravulizumab OBDS · Biological: Ravulizumab
The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
Also known as: ALXN1210
Ctrough Serum Concentration of Ravulizumab
Time frame: Predose at Day 71
Ctrough Serum Concentration of Ravulizumab at Day 351
Time frame: Predose at Day 351
Free Serum Complement Component 5 (C5) Concentrations at Day 71
Time frame: Predose at Day 71
Free Serum Complement Component 5 (C5) Concentrations at Day 351
Time frame: Predose at Day 351
Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at 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.
Time frame: Baseline, Day 71
Percent Change From Baseline in LDH Levels at 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.
Time frame: Baseline, Day 351
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at 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.
Time frame: Baseline, Day 71
Change From Baseline in FACIT-Fatigue Scale Version 4 Score at 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.
Time frame: Baseline, Day 351
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at 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.
Time frame: Baseline, Day 71
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at 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.
Time frame: Baseline, Day 351
Percentage of Participants Who Experienced Breakthrough Hemolysis 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.
Time frame: Baseline up to Day 71
Percentage of Participants Who Experienced Breakthrough Hemolysis 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.
Time frame: Baseline up to Day 351
Percentage of Participants Who Achieved Transfusion Avoidance up to Day 71
Transfusion Avoidance was defined as participants who remained transfusion free and did not require a transfusion after the first dose of study drug through the period of interest. Percentages are based on participants with any post-baseline data for the period. For Through Day 71, only visits with data were used to assess transfusion avoidance.
Time frame: Baseline up to Day 71
Percentage of Participants Who Achieved Transfusion Avoidance up to Day 351
Transfusion Avoidance was defined as participants who remained transfusion free and did not require a transfusion after the first dose of study drug through the period of interest. Denominator for a percentage was participants with at least one post-baseline data for the period.
Time frame: Baseline up to Day 351
Percentage of Participants Who Maintained Stabilized Hemoglobin (SHg) up to Day 71
SHg was defined as the avoidance of a ≥2 g/dL decrease in hemoglobin level from Baseline (defined as the last assessment prior to the first dose of the study drug) in the absence of transfusion to the end of the period of interest. Percentages were based on participants with at least one post-baseline data for the period. For Through Day 71, only visits with data were used to assess SHg.
Time frame: Baseline up to Day 71
Percentage of Participants Who Maintained SHg up to Day 351
SHg was defined as the avoidance of a ≥2 g/dL decrease in hemoglobin level from SC Baseline (defined as the last assessment prior to the first dose of SC treatment) in the absence of transfusion to the end of the period of interest. Denominator for a percentage was participants with at least one post-baseline data for the period. Visits were based on the number of days since first dose of SC treatment.
Time frame: Baseline up to Day 351
| Milestone | Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Started | 46 | 90 |
| Received at least 1 dose of study drug | 46 | 90 |
| Treated and not included in analysis | 1 | 6 |
| Full analysis set | 45 | 84 |
| Safety analysis set | 45 | 84 |
| Completed | 44 | 84 |
| Not completed | 2 | 6 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Site source document deviations | 1 | 6 |
| Milestone | Ravulizumab Intravenous (IV)/Subcutaneous (SC) Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Started | 44 | 83 |
| Received at least 1 dose of study drug | 44 | 83 |
| Treated but not included in the analysis | 1 | 6 |
| Completed | 31 | 64 |
| Not completed | 13 | 19 |
| Withdrew: Withdrawal by subject | 9 | 12 |
| Withdrew: Death | 2 | 2 |
| Withdrew: Other than specified | 0 | 1 |
| Withdrew: Protocol violation | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Lack of efficacy | 0 | 1 |
| Withdrew: Physician decision | 1 | 2 |
| micrograms/milliliter (µg/mL) | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Ctrough Serum Concentration of Ravulizumab | 457.58 ± 108.491 | 578.70 ± 140.819 |
| µg/mL | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Ctrough Serum Concentration of Ravulizumab at Day 351 | 712.79 ± 203.180 | 737.65 ± 208.894 |
| µg/mL | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Free Serum Complement Component 5 (C5) Concentrations at Day 71 | 0.072193 ± 0.0245225 | 0.059458 ± 0.0182180 |
| µg/mL | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Free Serum Complement Component 5 (C5) Concentrations at Day 351 | 0.071627 ± 0.0227980 | 0.069711 ± 0.0208784 |
Baseline was defined as the last assessment prior to first study drug dose. LDH samples impacted by tabletop hemolysis were excluded from the analysis.
| percent change | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percent Change From Baseline in Lactate Dehydrogenase (LDH) Levels at Day 71 | 5.73 ± 29.716 | 2.57 ± 33.883 |
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.
| percent change | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percent Change From Baseline in LDH Levels at Day 351 | -0.83 ± 17.225 | 1.74 ± 21.905 |
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.
| units on a scale | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at Day 71 | -0.83 ± 7.378 | 1.21 ± 7.882 |
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.
| units on a scale | Ravulizumab SC/SC Treatment Group |
|---|---|
| Change From Baseline in FACIT-Fatigue Scale Version 4 Score at Day 351 | 2.57 ± 7.178 |
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.
| units on a scale | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 71 | -7.00 ± 34.581 | -70.54 ± 70.522 |
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.
| units on a scale | Ravulizumab SC/SC Treatment Group |
|---|---|
| Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 351 | -69.29 ± 80.068 |
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.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 71 | 2.2 (0.06 to 11.77) | 1.2 (0.03 to 6.46) |
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.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 351 | 4.5 (0.56 to 15.47) | 3.6 (0.74 to 10.08) |
Transfusion Avoidance was defined as participants who remained transfusion free and did not require a transfusion after the first dose of study drug through the period of interest. Percentages are based on participants with any post-baseline data for the period. For Through Day 71, only visits with data were used to assess transfusion avoidance.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Achieved Transfusion Avoidance up to Day 71 | 86.7 (73.21 to 94.95) | 94.0 (86.65 to 98.04) |
Transfusion Avoidance was defined as participants who remained transfusion free and did not require a transfusion after the first dose of study drug through the period of interest. Denominator for a percentage was participants with at least one post-baseline data for the period.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Achieved Transfusion Avoidance up to Day 351 | 79.5 (64.70 to 90.20) | 85.7 (76.38 to 92.39) |
SHg was defined as the avoidance of a ≥2 g/dL decrease in hemoglobin level from Baseline (defined as the last assessment prior to the first dose of the study drug) in the absence of transfusion to the end of the period of interest. Percentages were based on participants with at least one post-baseline data for the period. For Through Day 71, only visits with data were used to assess SHg.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Maintained Stabilized Hemoglobin (SHg) up to Day 71 | 81.8 (67.29 to 91.81) | 93.6 (85.67 to 97.89) |
SHg was defined as the avoidance of a ≥2 g/dL decrease in hemoglobin level from SC Baseline (defined as the last assessment prior to the first dose of SC treatment) in the absence of transfusion to the end of the period of interest. Denominator for a percentage was participants with at least one post-baseline data for the period. Visits were based on the number of days since first dose of SC treatment.
| percentage of participants | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Percentage of Participants Who Maintained SHg up to Day 351 | 72.7 (57.21 to 85.04) | 83.5 (73.51 to 90.94) |
Collected over Baseline up to approximately 3.5 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ravulizumab IV/SC Treatment Group | 2/45 (4.4%) | 16/45 (35.6%) | 44/45 (97.8%) |
| Ravulizumab SC/SC Treatment Group | 2/84 (2.4%) | 30/84 (35.7%) | 83/84 (98.8%) |
| Event | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| COVID-19Infections and infestations | 2/45 | 6/84 |
| HaemolysisBlood and lymphatic system disorders | 0/45 | 5/84 |
| CholecystitisHepatobiliary disorders | 2/45 | 1/84 |
| AnaemiaBlood and lymphatic system disorders | 1/45 | 2/84 |
| Aplastic anaemiaBlood and lymphatic system disorders | 0/45 | 2/84 |
| COVID-19 pneumoniaInfections and infestations | 0/45 | 2/84 |
| Febrile neutropeniaBlood and lymphatic system disorders | 1/45 | 2/84 |
| Tubo-ovarian abscessInfections and infestations | 0/25 | 1/44 |
| Adnexal torsionReproductive system and breast disorders | 0/25 | 1/44 |
| ThrombocytopeniaBlood and lymphatic system disorders | 1/45 | 1/84 |
| Event | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group |
|---|---|---|
| Device delivery system issueProduct Issues | 42/45 | 74/84 |
| COVID-19Infections and infestations | 16/45 | 39/84 |
| HeadacheNervous system disorders | 14/45 | 16/84 |
| PyrexiaGeneral disorders | 7/45 | 20/84 |
| DiarrhoeaGastrointestinal disorders | 3/45 | 17/84 |
| NasopharyngitisInfections and infestations | 8/45 | 11/84 |
| AstheniaGeneral disorders | 3/45 | 13/84 |
| AnaemiaBlood and lymphatic system disorders | 4/45 | 12/84 |
| Abdominal painGastrointestinal disorders | 6/45 | 10/84 |
| InfluenzaInfections and infestations | 6/45 | 10/84 |
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.
| Age, Continuous(years) | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group | Total |
|---|---|---|---|
| Mean | 46.4 ± 13.22 | 45.3 ± 14.47 | 45.7 ± 14.00 |
| Sex: Female, Male(Participants) | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group | Total |
|---|---|---|---|
| Female | 25 | 44 | 69 |
| Male | 20 | 40 | 60 |
| Ethnicity (NIH/OMB)(Participants) | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group | Total |
|---|---|---|---|
| Hispanic or Latino | 7 | 15 | 22 |
| Not Hispanic or Latino | 29 | 53 | 82 |
| Unknown or Not Reported | 9 | 16 | 25 |
| Race (NIH/OMB)(Participants) | Ravulizumab IV/SC Treatment Group | Ravulizumab SC/SC Treatment Group | Total |
|---|---|---|---|
| American Indian or Alaska Native | 1 | 0 | 1 |
| Asian | 2 | 0 | 2 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 4 | 3 | 7 |
| White | 29 | 63 | 92 |
| More than one race | 2 | 4 | 6 |
| Unknown or Not Reported | 7 | 14 | 21 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Feb 2019 to primary completion in Feb 2021, 23 months.
Results were first posted in Oct 2022, 1 year 8 months after primary completion. A results delay was requested in Jan 2021.
Of 35 industry-sponsored phase 3 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 23 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 41. This study's 23 months is close to that.
Counting every sponsor, half of 35 studies reached it within 23 months; a quarter within 15 and three quarters within 41.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ravulizumab
Alexion Pharmaceuticals, Inc. →