A Phase 2 interventional study of Ravulizumab in Paroxysmal Nocturnal Hemoglobinuria and PNH, sponsored by Alexion Pharmaceuticals, Inc.. Completed at 16 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2023-01-04.
Sponsored by Alexion Pharmaceuticals, Inc. (part of AstraZeneca) · Phase 2, 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 12th largest by enrollment of the 23 interventional ones that give a count, is one of 2 that started in 2016, is one of 7 in phase 2, and was the 13th 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 2016.
This study's enrollment of 26 is below 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.
This tab as a report to print or save as PDF → With an account.
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
The study consisted of a screening period of up to 30 days and a Treatment Period of up to 253 days for Cohorts 1-3 and 281 days for Cohort 4. After completion of the Treatment Period, all participants had the opportunity to enter the Extension Period, wherein participants continue to receive ravulizumab for up to 5 years. The first dose in the Extension Period occurred on Day 253 for Cohorts 1-3 and on Day 281 for Cohort 4.
The data presented includes the Primary Completion date of the study for the Treatment Period. The results for the Extension Period will be reported after study completion.
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.
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.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
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.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
During the Treatment Period, participants were administered ravulizumab 1400 milligram (mg) on Day 1, ravulizumab 1000 mg on Day 15 and Day 29, and then ravulizumab 1000 mg every 4 weeks for 7 doses. In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kilograms (kg), 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.
Biological: Ravulizumab
During the Treatment Period, participants were administered ravulizumab 2000 mg on Day 1, ravulizumab 1600 mg on Day 22 and Day 43, and then ravulizumab 1600 mg every 6 weeks for 4 doses. In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.
Biological: Ravulizumab
During the Treatment Period, participants were administered ravulizumab 1600 mg on Day 1 and Day 15, ravulizumab 2400 mg on Day 29, and then ravulizumab 2400 mg every 8 weeks for 3 doses. In the Extension Period, participants initially continued to receive their dose. During the second year of the study, participants were administered weight-based doses of ravulizumab every 8 weeks for up to 5 years: 3000 mg for participants weighing 40 to less than 60 kg, 3300 mg for participants weighing 60 to less than 100 kg, and 3600 mg for participants weighing 100 kg or more.
Biological: Ravulizumab
During the Treatment Period, participants were administered ravulizumab 3000 mg on Day 1, ravulizumab 5400 mg on Day 29, and then ravulizumab 5400 mg every 12 weeks for 2 doses. During the Extension Period, participants were administered ravulizumab 5400 mg every 12 weeks for up to 5 years.
Biological: Ravulizumab
All treatments were given as IV infusions.
Also known as: ALXN1210, Ultomiris
Percent Change In LDH Levels From Baseline To Day 253 And Day 281
The percent change in LDH levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Free Hemoglobin Levels From Baseline To Day 253 And Day 281
The percent change in free hemoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Haptoglobin Levels From Baseline To Day 253 And Day 281
The percent change in haptoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In Reticulocyte/Erythrocyte Count From Baseline To Day 253 And Day 281
The percent change in reticulocyte/erythrocyte count levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline, Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253
The percent change in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell (RBC), summed types II and III, clone size levels were assessed from Baseline to Day 253 for Cohorts 1 to 4.
Time frame: Baseline, Day 253 (Cohorts 1 to 4)
Percent Change In D-dimer From Baseline To Day 253 And Day 281
The percent change in D-dimer levels were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
Time frame: Baseline to Day 253 (Cohorts 1 to 4) and Day 281 (Cohort 4)
Change In Clinical Manifestations Of PNH From Baseline To Day 253 And Day 281
Clinical manifestations were assessed from Baseline to Day 253 for Cohorts 1 to 3 and from Baseline to Day 281 for Cohort 4 only. Clinical manifestations were defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (male participants only). Improvement was defined as present at Baseline and absent at Day endpoint. Worsening was defined as absent at Baseline and present at Day endpoint. No Change was defined as no change from Baseline and time point of endpoint.
Time frame: Baseline, Day 253 (Cohorts 1 to 3) and Day 281 (Cohort 4)
| Milestone | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Started | 6 | 6 | 7 | 7 |
| Received at least 1 dose of study drug | 6 | 6 | 7 | 7 |
| Completed | 6 | 6 | 7 | 7 |
| Not completed | 0 | 0 | 0 | 0 |
| Milestone | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Started | 6 | 6 | 7 | 7 |
| Received at least 1 dose of study drug | 6 | 6 | 7 | 7 |
| Completed | 6 | 6 | 6 | 7 |
| Not completed | 0 | 0 | 1 | 0 |
| Withdrew: Participant underwent bone marrow transplantation due to chronic myelomonocytic leukemia | 0 | 0 | 1 | 0 |
The percent change in LDH levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Day 253 | -72.85 ± 12.082 | -78.12 ± 6.635 | -84.96 ± 4.423 | -87.63 ± 6.923 |
| Day 281 | — | — | — | -89.89 ± 2.885 |
The percent change in free hemoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Day 253 | 14.07 ± 124.755 | -12.32 ± 57.213 | -22.14 ± 94.388 | -40.80 ± 27.474 |
| Day 281 | — | — | — | -45.64 ± 28.938 |
The percent change in haptoglobin levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Day 253 | 21.67 ± 53.072 | 81.67 ± 200.042 | 4.29 ± 11.339 | 34.29 ± 74.578 |
| Day 281 | — | — | — | 27.14 ± 56.188 |
The percent change in reticulocyte/erythrocyte count levels was assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Day 253 | -1.27 ± 43.019 | -5.05 ± 35.691 | 10.46 ± 59.510 | 14.66 ± 81.390 |
| Day 281 | — | — | — | -4.66 ± 68.502 |
The percent change in paroxysmal nocturnal hemoglobinuria (PNH) red blood cell (RBC), summed types II and III, clone size levels were assessed from Baseline to Day 253 for Cohorts 1 to 4.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Percent Change In PNH RBC Types II And III Clone Size From Baseline To Day 253 | 6.65 ± 24.101 | 5.47 ± 21.940 | 54.14 ± 98.713 | 88.21 ± 138.290 |
The percent change in D-dimer levels were assessed from Baseline to Day 253 for Cohorts 1 to 4 and from Baseline to Day 281 for Cohort 4 only.
| Percent Change | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Day 253 | -24.80 ± 32.436 | -29.47 ± 26.547 | -10.90 ± 41.032 | -16.08 ± 34.783 |
| Day 281 | — | — | — | -27.05 ± 27.663 |
Clinical manifestations were assessed from Baseline to Day 253 for Cohorts 1 to 3 and from Baseline to Day 281 for Cohort 4 only. Clinical manifestations were defined as fatigue, abdominal pain, dyspnea, dysphagia, chest pain, and erectile dysfunction (male participants only). Improvement was defined as present at Baseline and absent at Day endpoint. Worsening was defined as absent at Baseline and present at Day endpoint. No Change was defined as no change from Baseline and time point of endpoint.
| Participants | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Fatigue at Day 253 — Improved from Baseline | 4 | 2 | 3 | 3 |
| Fatigue at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Fatigue at Day 253 — No Change | 2 | 4 | 4 | 4 |
| Fatigue at Day 253 — Not Applicable | 0 | 0 | 0 | 0 |
| Fatigue at Day 281 — Improved from Baseline | — | — | — | 4 |
| Fatigue at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Fatigue at Day 281 — No Change | — | — | — | 3 |
| Fatigue at Day 281 — Not Applicable | — | — | — | 0 |
| Abdominal Pain at Day 253 — Improved from Baseline | 1 | 1 | 1 | 0 |
| Abdominal Pain at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Abdominal Pain at Day 253 — No Change | 5 | 5 | 6 | 7 |
| Abdominal Pain at Day 253 — Not Applicable | 0 | 0 | 0 | 0 |
| Abdominal Pain at Day 281 — Improved from Baseline | — | — | — | 0 |
| Abdominal Pain at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Abdominal Pain at Day 281 — No Change | — | — | — | 7 |
| Abdominal Pain at Day 281 — Not Applicable | — | — | — | 0 |
| Dyspnea at Day 253 — Improved from Baseline | 1 | 1 | 4 | 2 |
| Dyspnea at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Dyspnea at Day 253 — No Change | 5 | 5 | 3 | 5 |
| Dyspnea at Day 253 — Not Applicable | 0 | 0 | 0 | 0 |
| Dyspnea at Day 281 — Improved from Baseline | — | — | — | 2 |
| Dyspnea at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Dyspnea at Day 281 — No Change | — | — | — | 5 |
| Dyspnea at Day 281 — Not Applicable | — | — | — | 0 |
| Dysphagia at Day 253 — Improved from Baseline | 0 | 1 | 1 | 1 |
| Dysphagia at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Dysphagia at Day 253 — No Change | 6 | 5 | 6 | 6 |
| Dysphagia at Day 253 — Not Applicable | 0 | 0 | 0 | 0 |
| Dysphagia at Day 281 — Improved from Baseline | — | — | — | 1 |
| Dysphagia at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Dysphagia at Day 281 — No Change | — | — | — | 6 |
| Dysphagia at Day 281 — Not Applicable | — | — | — | 0 |
| Chest Pain at Day 253 — Improved from Baseline | 1 | 0 | 2 | 0 |
| Chest Pain at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Chest Pain at Day 253 — No Change | 5 | 6 | 5 | 7 |
| Chest Pain at Day 253 — Not Applicable | 0 | 0 | 0 | 0 |
| Chest Pain at Day 281 — Improved from Baseline | — | — | — | 0 |
| Chest Pain at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Chest Pain at Day 281 — No Change | — | — | — | 7 |
| Chest Pain at Day 281 — Not Applicable | — | — | — | 0 |
| Erectile Dysfunction at Day 253 — Improved from Baseline | 2 | 0 | 1 | 1 |
| Erectile Dysfunction at Day 253 — Worsened from Baseline | 0 | 0 | 0 | 0 |
| Erectile Dysfunction at Day 253 — No Change | 2 | 5 | 5 | 4 |
| Erectile Dysfunction at Day 253 — Not Applicable | 2 | 1 | 1 | 2 |
| Erectile Dysfunction at Day 281 — Improved from Baseline | — | — | — | 1 |
| Erectile Dysfunction at Day 281 — Worsened from Baseline | — | — | — | 0 |
| Erectile Dysfunction at Day 281 — No Change | — | — | — | 4 |
| Erectile Dysfunction at Day 281 — Not Applicable | — | — | — | 2 |
Collected over Adverse events were monitored continuously from Screening to Day 253 (for Cohorts 1, 2, and 3) and Day 281 (for Cohort 4) (Treatment Period) and up to Day 2157 (Extension Period).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Cohort 1 | 0/6 (0%) | 3/6 (50%) | 6/6 (100%) |
| Cohort 2 | 0/6 (0%) | 3/6 (50%) | 6/6 (100%) |
| Cohort 3 | 0/7 (0%) | 4/7 (57.1%) | 7/7 (100%) |
| Cohort 4 | 0/7 (0%) | 1/7 (14.3%) | 7/7 (100%) |
| Event | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Febrile neutropeniaBlood and lymphatic system disorders | 1/6 | 1/6 | 0/7 | 0/7 |
| PyrexiaGeneral disorders | 1/6 | 0/6 | 0/7 | 0/7 |
| Meningococcal infectionInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Urinary tract infectionInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Femoral neck fractureInjury, poisoning and procedural complications | 0/6 | 1/6 | 0/7 | 0/7 |
| InfectionInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Klebsiella bacteraemiaInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Enterobacter bacteraemiaInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Enterobacter sepsisInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Escherichia urinary tract infectionInfections and infestations | 0/6 | 1/6 | 0/7 | 0/7 |
| Event | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| HeadacheNervous system disorders | 3/6 | 5/6 | 4/7 | 3/7 |
| Upper respiratory tract infectionInfections and infestations | 2/6 | 1/6 | 5/7 | 4/7 |
| NasopharyngitisInfections and infestations | 4/6 | 2/6 | 3/7 | 2/7 |
| Back painMusculoskeletal and connective tissue disorders | 4/6 | 0/6 | 2/7 | 1/7 |
| FatigueGeneral disorders | 3/6 | 2/6 | 0/7 | 0/7 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 3/6 | 0/6 | 0/7 | 2/7 |
| DyspnoeaRespiratory, thoracic and mediastinal disorders | 3/6 | 0/6 | 0/7 | 1/7 |
| Abdominal painGastrointestinal disorders | 3/6 | 0/6 | 1/7 | 2/7 |
| DizzinessNervous system disorders | 1/6 | 0/6 | 3/7 | 0/7 |
| DiarrhoeaGastrointestinal disorders | 0/6 | 1/6 | 3/7 | 0/7 |
Safety set: All participants who received at least 1 dose of ravulizumab.
| Age, Continuous(years) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| Mean | 43.1 ± 14.57 | 48.6 ± 23.48 | 37.3 ± 14.03 | 48.5 ± 13.43 | 44.3 ± 16.33 |
| Sex: Female, Male(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| Female | 2 | 1 | 1 | 2 | 6 |
| Male | 4 | 5 | 6 | 5 | 20 |
| Ethnicity (NIH/OMB)(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 1 | 0 | 0 | 0 | 1 |
| Not Hispanic or Latino | 4 | 4 | 7 | 7 | 22 |
| Unknown or Not Reported | 1 | 2 | 0 | 0 | 3 |
| Race/Ethnicity, Customized(Participants) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| White | 5 | 4 | 3 | 3 | 15 |
| Asian | 0 | 0 | 4 | 3 | 7 |
| Not Reported | 1 | 2 | 0 | 0 | 3 |
| Other | 0 | 0 | 0 | 1 | 1 |
| Lactate Dehydrogenase (LDH) Levels(units/liter (U/L)) | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|
| Mean | 1026.88 ± 547.843 | 1223.55 ± 149.693 | 2127.57 ± 815.875 | 2142.24 ± 366.511 | 1668.90 ± 724.341 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jan 2016 to primary completion in Feb 2017, 13 months.
Results were first posted in Feb 2019, 1 year 11 months after primary completion. A results delay was requested in Feb 2018.
Of 26 industry-sponsored phase 2 interventional studies under Hemoglobinuria, Paroxysmal, half reached primary completion within 16 months of starting (the median), counting those still running; a quarter within 11 and three quarters within 25. This study's 13 months is below that.
Counting every sponsor, half of 34 studies reached it within 24 months; a quarter within 12 and three quarters within 80.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2022. 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. →