Paroxysmal Nocturnal HemoglobinuriaPhase 3NCT03748823
Ravulizumab Subcutaneous (SC) Versus Ravulizumab Intravenous (IV) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Eculizumab
A Phase 3 study testing Ravulizumab OBDS and Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 51 sites in 14 countries.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Paroxysmal Nocturnal Hemoglobinuria
- What is being tested?
- Ravulizumab OBDS and Ravulizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 139 participants
- Where
- 51 sitesin 14 countries
- Study dates
- 19 Feb 2019to 31 Aug 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female ≥18 years of age
- Treated with eculizumab for PNH for at least 3 months prior to Day 1
- LDH level ≤1.5 × upper limit of normal (ULN) at screening
- PNH diagnosis confirmed by documented high-sensitivity flow cytometry.
- Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
- Body weight ≥40 to <100 kilogram (kg)
- Female participants of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ravulizumab.
- Willing and able to give written informed consent and comply with study visit schedule.
Who cannot join
- More than 1 LDH value > 2 × ULN within the 3 months prior to study entry
- History of bone marrow transplantation.
- History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the Investigator or Sponsor, would preclude participation.
- Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH).
- Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1.
- Participation in another interventional clinical study or use of any experimental therapy within 30 days before initiation of study drug on Day 1 in this study or within 5 half-lives of that investigational product, whichever is greater.
Trial locations and contacts
51 sites in 14 countries.
51 sites in 14 countries
France13 sites
Research Site
Amiens, 80054
Research Site
Brest, 29609
Research Site
Lille, 59037
Research Site
Montpellier, 34090
Research Site
Nantes, 44093
Research Site
Nice, 6200
Research Site
Paris, 75010
Research Site
Pessac, F-33604
Research Site
Pierre-Bénite, 69495
Research Site
Poitiers, 86021
Research Site
Rennes, 35033
Research Site
Strasbourg, 67098
Research Site
Tours, 37044
Spain7 sites
Research Site
Badalona, 8916
Research Site
Barcelona, 08036
Research Site
Donostia / San Sebastian, 20014
Research Site
Las Palmas de Gran Canaria, 35020
Research Site
Madrid, 28040
Research Site
Majadahonda, 28222
Research Site
Seville, 41013
Brazil6 sites
Research Site
Botucatu, 18618-970
Research Site
Ribeirão Preto, 14049-901
Research Site
Rio de de Janeiro, 20211030
Research Site
Salvador, 40170010
Research Site
São Paulo, 01308-050
Research Site
São Paulo, 05403-000
Turkey (Türkiye)5 sites
Research Site
Adana, 01330
Research Site
Istanbul, 34093
Research Site
Istanbul, 34096
Research Site
Istanbul, 34899
Research Site
Izmir, 35100
Belgium4 sites
Research Site
Antwerp, 2020
Research Site
Brussels, 1200
Research Site
Hasselt, 3500
Research Site
Leuven, 3000
Italy4 sites
Research Site
Catania, 95123
Research Site
Milan, 20122
Research Site
Roma, 00168
Research Site
Rome, 161
Russia4 sites
Research Site
Moscow, 125167
Research Site
Moscow, 125284
Research Site
Saint Petersburg, 197089
Research Site
Yekaterinburg, 620102
About this study
Official title: A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab
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.
Read the detailed description
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.
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Being tested
- Ravulizumab OBDS, Ravulizumab
- Keywords
- PNH
- Medical subject headings
- Hemoglobinuria, Paroxysmal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ravulizumab OBDSCombination product
The ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector.
RavulizumabBiological
Also called ALXN1210
Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight.
| Study group | Role | What they receive |
|---|---|---|
| Ravulizumab SC Treatment Group | Experimental | 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. Receives: Combination Product: Ravulizumab OBDS; Biological: Ravulizumab |
| Ravulizumab IV Treatment Group | Active comparator | 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. Receives: Combination Product: Ravulizumab OBDS; Biological: Ravulizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Ctrough Serum Concentration of Ravulizumab
Measured: Predose at Day 71
Secondary outcomes (15)
Ctrough Serum Concentration of Ravulizumab at Day 351
Measured: Predose at Day 351
Free Serum Complement Component 5 (C5) Concentrations at Day 71
Measured: Predose at Day 71
Free Serum Complement Component 5 (C5) Concentrations at Day 351
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: Baseline up to Day 351
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Nov 2018
- Study start19 Feb 2019
- Primary completion2 Feb 2021
- Study completion31 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Paroxysmal Nocturnal HemoglobinuriaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 139 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03748823
- Sponsor's protocol ID
- ALXN1210-PNH-303
- Other IDs
- 2017-002370-39, 2017
- Status last verified
- Aug 2024
- First submitted
- 19 Nov 2018
- Last updated
- 19 Sep 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- Yes
- Individual data sharing
- No
Publications
Yenerel MN, Sicre de Fontbrune F, Piatek C, Sahin F, Fureder W, Ortiz S, Ogawa M, Ozol-Godfrey A, Sierra JR, Szer J. Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up. Adv Ther. 2023 Jan;40(1):211-232. doi: 10.1007/s12325-022-02339-3. Epub 2022 Oct 22.
PubMed 36272026
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Randomized, Parallel-Group, Multicenter, Open-Label, Pharmacokinetic, Noninferiority Study of Ravulizumab Administered Subcutaneously Versus Intravenously in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria Currently Treated With Eculizumab
- ClinicalTrials.gov ID
- NCT03748823
- Acronym / sponsor ID
- ALXN1210-PNH-303
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria
- Keywords
- PNH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 139 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Feb 2019 (actual)
- Primary completion
- 2 Feb 2021 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 21 Nov 2018 (actual)
- Results
- Posted 20 Oct 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Ravulizumab SC Treatment Group | Experimental | Combination Product: Ravulizumab OBDS, Biological: RavulizumabIn 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. |
| Ravulizumab IV Treatment Group | Active comparator | Combination Product: Ravulizumab OBDS, Biological: RavulizumabIn 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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male or female ≥18 years of age
- Treated with eculizumab for PNH for at least 3 months prior to Day 1
- LDH level ≤1.5 × upper limit of normal (ULN) at screening
- PNH diagnosis confirmed by documented high-sensitivity flow cytometry.
- Documented meningococcal vaccination not more than 3 years prior to, or at the time of, initiating study treatment.
Key exclusion criteria (6 listed)
- More than 1 LDH value > 2 × ULN within the 3 months prior to study entry
- History of bone marrow transplantation.
- History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic disease that, in the opinion of the Investigator or Sponsor, would preclude participation.
- Unstable medical conditions (for example, myocardial ischemia, active gastrointestinal bleed, severe congestive heart failure, anticipated need for major surgery within 6 months of randomization, coexisting chronic anemia unrelated to PNH).
- Females who are pregnant, breastfeeding or who have a positive pregnancy test at screening or Day 1.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ravulizumab OBDSThe ravulizumab OBDS is a biological-device combination product consisting of a prefilled cartridge containing ravulizumab SC and an on-body injector. | Combination product | Ravulizumab IV Treatment Group, Ravulizumab SC Treatment Group |
| RavulizumabAlso: ALXN1210Administered by IV infusion. Ravulizumab IV doses will be based on participant body weight. | Biological | Ravulizumab IV Treatment Group, Ravulizumab SC Treatment Group |
Primary outcome
Ctrough Serum Concentration of Ravulizumab
Time frame: Predose at Day 71
15 secondary outcomes
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
Time frame: Baseline, Day 71
Percent Change From Baseline in LDH Levels at Day 351
Time frame: Baseline, Day 351
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Subscale Version 4 Score at Day 71
Time frame: Baseline, Day 71
Change From Baseline in FACIT-Fatigue Scale Version 4 Score at Day 351
Time frame: Baseline, Day 351
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 71
Time frame: Baseline, Day 71
Change From Baseline in Treatment Administration Satisfaction Questionnaire (TASQ) Score at Day 351
Time frame: Baseline, Day 351
Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 71
Time frame: Baseline up to Day 71
Percentage of Participants Who Experienced Breakthrough Hemolysis up to Day 351
Time frame: Baseline up to Day 351
Percentage of Participants Who Achieved Transfusion Avoidance up to Day 71
Time frame: Baseline up to Day 71
Percentage of Participants Who Achieved Transfusion Avoidance up to Day 351
Time frame: Baseline up to Day 351
Percentage of Participants Who Maintained Stabilized Hemoglobin (SHg) up to Day 71
Time frame: Baseline up to Day 71
Percentage of Participants Who Maintained SHg up to Day 351
Time frame: Baseline up to Day 351
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36272026Yenerel MN, Sicre de Fontbrune F, Piatek C, Sahin F, Fureder W, Ortiz S, Ogawa M, Ozol-Godfrey A, Sierra JR, Szer J. Phase 3 Study of Subcutaneous Versus Intravenous Ravulizumab in Eculizumab-Experienced Adult Patients with PNH: Primary Analysis and 1-Year Follow-Up. Adv Ther. 2023 Jan;40(1):211-232. doi: 10.1007/s12325-022-02339-3. Epub 2022 Oct 22.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Paroxysmal Nocturnal Hemoglobinuria
Enrolling by invitationObservational study
An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea
Hemoglobinuria, Paroxysmal
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 3
A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works
Paroxysmal Nocturnal Hemoglobinuria
Not yet recruitingPhase 3
A Study of Efficacy and Safety of FXS6837 in Anti-C5 Treated PNH Patients.
Paroxysmal Nocturnal Hemoglobinuria
RecruitingPhase 2
Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis
Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
51 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Ravulizumab OBDS and Ravulizumab in people with Paroxysmal Nocturnal Hemoglobinuria, run by Alexion Pharmaceuticals, Inc. at 51 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 51 sites in France, Spain, Brazil, Turkey (Türkiye), Belgium and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Oct 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Ctrough Serum Concentration of Ravulizumab (time frame: Predose at Day 71). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03748823, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Sep 2024.