A Phase 4 interventional study of Eculizumab and Ravulizumab in Paroxysmal Nocturnal Hemoglobinuria, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 4 sites in United Kingdom. Per ClinicalTrials.gov, last updated 2024-09-05.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 4, Interventional, and Treatment
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Among 18 studies testing eculizumab in Hemoglobinuria, Paroxysmal — none open to participants now, none under way, none of them phase 3. eculizumab · Hemoglobinuria, Paroxysmal.
Among the 18 studies testing eculizumab in Hemoglobinuria, Paroxysmal, this one is the 16th largest by enrollment, is the only one that started in 2021, is one of 2 in phase 4, and was the 12th registered.
191 studies on the registry are indexed under Hemoglobinuria, Paroxysmal; 49 are open to participants now.
Browse Hemoglobinuria, Paroxysmal studies →None of the 18 studies testing eculizumab in Hemoglobinuria, Paroxysmal is open now. The open studies under Hemoglobinuria, Paroxysmal, anywhere →
eculizumab: first FDA approval 2007 (SOLIRIS), first EMA approval 2007. What it is approved for, and how studies give it, is on its page →
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: 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.
18 studies; the count open now beside each. This study's row is marked.
First in 2004; this study’s year is marked. 1 started in 2021.
This study's enrollment of 18 is below the median of 33 across 150 interventional studies indexed under Hemoglobinuria, Paroxysmal.
Median enrollment 37 across the 18 interventional studies. Of the 18 that completed or were terminated, 9 (50%) have results posted.
This tab as a report to print or save as PDF → With an account.
16 of 18 have an industry lead sponsor. The 8 most frequent:
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.
35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca: 3 open now, 7 under way, 2 of them phase 3. Of 26 finished, 18 (69%) have results posted.
Among the 35 studies in Hemoglobinuria, Paroxysmal by AstraZeneca, this one is the 14th largest by enrollment of the 23 interventional ones that give a count, is the only one that started in 2021, is one of 2 in phase 4, and was the 22nd registered.
AstraZeneca’s Hemoglobinuria, Paroxysmal studies by the year they started, since 2004.
None of the studies under way reaches primary completion within a year.
Among the 18 studies testing eculizumab in Hemoglobinuria, Paroxysmal, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among AstraZeneca’s 35 in Hemoglobinuria, Paroxysmal:
Nothing recorded among them since 2026-09-25.
This tab as a report to print or save as PDF → With an account.
The primary purpose of this study is to assess the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab in participants who are prescribed and are receiving a higher than approved dose of eculizumab to treat paroxysmal nocturnal hemoglobinuria (PNH).
Key Inclusion Criteria:
Key Exclusion Criteria:
Concomitant use of any of the following medications and not on a stable regimen (as judged by the Investigator) for the time period indicated prior to Screening:
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.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
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.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Live vaccines
Vaccines made with a weakened form of the germ, such as measles–mumps–rubella (MMR), chickenpox and yellow fever. They can be risky when the immune system is weakened. Flu injections are not live; the nasal flu spray is.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants will receive eculizumab during the 3-month Screening Period. Participants will then switch over to and receive weight-based doses of ravulizumab for the duration of the study Treatment Period (351 days).
Biological: Eculizumab · Biological: Ravulizumab
Participants must have been prescribed and be receiving a stable dose of eculizumab 1200 milligrams (mg) every 2 weeks (q2w) for at least 3 months prior to the Screening Period. During the Screening Period, participants will continue to receive eculizumab 1200 mg q2w.
Also known as: Soliris
During the Treatment Period, participants will receive a loading dose of ravulizumab on Day 1, followed by maintenance doses on Day 15 and every 8 weeks, administered by intravenous infusion. Ravulizumab loading and maintenance doses will be based on participants' body weight per approved dose regimen.
Also known as: ALXN1210, Ultomiris
Percentage of Participants Who Experienced Free C5-associated BTH
Free C5-associated BTH was defined as BTH concurrent with free C5 concentrations ≥0.5 micrograms (μg)/milliliter (mL). BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 grams {g}/deciliter {dL}\], major adverse vascular event \[MAVE\], including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) ≥2 \* upper limit of normal (ULN).
Time frame: Baseline through Day 351
Percentage of Participants Who Experienced BTH
BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 g/dL\], MAVE, including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 \* ULN.
Time frame: Baseline through Day 351
Percent Change From Baseline in LDH at Day 351
Time frame: Baseline, Day 351
Percentage of Participants Who Received a Red Blood Cell (RBC) Transfusion
Time frame: Baseline through Day 351
Percentage of Participants With Stabilized Hemoglobin
Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion from Baseline to Day 351.
Time frame: Baseline through Day 351
| Milestone | Ravulizumab |
|---|---|
| Started | 18 |
| Received at least 1 dose of study drug | 18 |
| Completed | 18 |
| Not completed | 0 |
Free C5-associated BTH was defined as BTH concurrent with free C5 concentrations ≥0.5 micrograms (μg)/milliliter (mL). BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 grams {g}/deciliter {dL}\], major adverse vascular event \[MAVE\], including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) ≥2 \* upper limit of normal (ULN).
| percentage of participants | Ravulizumab |
|---|---|
| Percentage of Participants Who Experienced Free C5-associated BTH | 0 ± 0 |
BTH was defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath \[dyspnea\], anemia \[hemoglobin \<10 g/dL\], MAVE, including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2 \* ULN.
| percentage of participants | Ravulizumab |
|---|---|
| Percentage of Participants Who Experienced BTH | 5.6 ± 0.1 |
| percent change | Ravulizumab |
|---|---|
| Percent Change From Baseline in LDH at Day 351 | -0.81 ± 6.132 |
| percentage of participants | Ravulizumab |
|---|---|
| Percentage of Participants Who Received a Red Blood Cell (RBC) Transfusion | 33.3 ± 13.3 |
Stabilized hemoglobin was defined as avoidance of a ≥2 g/dL decrease in hemoglobin level from baseline in the absence of transfusion from Baseline to Day 351.
| percentage of participants | Ravulizumab |
|---|---|
| Percentage of Participants With Stabilized Hemoglobin | 61.1 ± 35.7 |
Collected over Baseline through Day 351. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ravulizumab | 0/18 (0%) | 3/18 (16.7%) | 15/18 (83.3%) |
| Event | Ravulizumab |
|---|---|
| PneumoniaInfections and infestations | 1/18 |
| Urinary tract infectionInfections and infestations | 1/18 |
| Retinal haemorrhageEye disorders | 1/18 |
| PyrexiaGeneral disorders | 1/18 |
| HeadacheNervous system disorders | 1/18 |
| Event | Ravulizumab |
|---|---|
| FatigueGeneral disorders | 6/18 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/18 |
| COVID-19Infections and infestations | 3/18 |
| SARS-CoV-2 test positiveInvestigations | 3/18 |
| Abdominal painGastrointestinal disorders | 3/18 |
| Non-cardiac chest painGeneral disorders | 2/18 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 2/18 |
| NasopharyngitisInfections and infestations | 2/18 |
| Back painMusculoskeletal and connective tissue disorders | 2/18 |
| AstheniaGeneral disorders | 1/18 |
The full analysis set (FAS) included all participants who received at least 1 dose of ravulizumab.
| Age, Continuous(years) | Ravulizumab |
|---|---|
| Mean | 55.7 ± 11.34 |
| Age, Customized(Participants) | Ravulizumab |
|---|---|
| In utero | 0 |
| Preterm newborn infants (gestational age < 37 wks) | 0 |
| Newborns (0-27 days) | 0 |
| Infants and toddlers (28 days-23 months) | 0 |
| Children (2-11 years) | 0 |
| Adolescents (12-17 years) | 0 |
| Adults (18-64 years) | 15 |
| From 65-84 years | 3 |
| 85 years and over | 0 |
| Sex: Female, Male(Participants) | Ravulizumab |
|---|---|
| Female | 6 |
| Male | 12 |
| Ethnicity (NIH/OMB)(Participants) | Ravulizumab |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 18 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Ravulizumab |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 2 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 13 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Apr 2021 to primary completion in Dec 2022, 20 months.
Results were first posted in Aug 2024, 1 year 7 months after primary completion.
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Studies of the drugs tested here: Eculizumab · Ravulizumab
Alexion Pharmaceuticals, Inc. →