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Ravulizumab bij trombotische microangiopathie na hematopoëtische stamceltransplantatie

2 november 2026 bijgewerkt door: Alexion Pharmaceuticals, Inc.

Een gerandomiseerde, dubbelblinde, placebogecontroleerde, multicenter fase 3-studie van Ravulizumab bij volwassen en adolescente deelnemers met trombotische microangiopathie (TMA) na hematopoëtische stamceltransplantatie (HSCT)

This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of ravulizumab in adult and adolescent participants with hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). In Stage 1, an open-label, single-arm period, the dosing regimen will be confirmed. In Stage 2, participants will be randomized to receive either blinded ravulizumab plus best supportive care or matching placebo plus best supportive care. The treatment period is 26 weeks (open-label for Stage 1, and randomized, double-blind, and placebo-controlled for Stage 2) followed by a 26-week follow-up period.

Studie Overzicht

Toestand

Voltooid

Conditie

Interventie / Behandeling

Studietype

Ingrijpend

Inschrijving (Werkelijk)

148

Fase

  • Fase 3

Contacten en locaties

In dit gedeelte vindt u de contactgegevens van degenen die het onderzoek uitvoeren en informatie over waar dit onderzoek wordt uitgevoerd.

Studie Locaties

  • Australië
      • Parkville, Australië, 3050
        • Research Site
  • België
      • Bruges, België, 8000
        • Research Site
      • Brussels, België, 1200
        • Research Site
      • Chênée, België, 4032
        • Research Site
      • Yvoir, België, 5530
        • Research Site
  • Brazilië
      • Cerqueira César, Brazilië, 05403-000
        • Research Site
      • Florianópolis, Brazilië, 88034-000
        • Research Site
      • Jaú, Brazilië, 17210-080
        • Research Site
      • Porto Alegre, Brazilië, 90110-270
        • Research Site
      • Porto Alegre, Brazilië, 90035-903
        • Research Site
      • Rio de Janeiro, Brazilië, 20230-130
        • Research Site
      • São José do Rio Preto, Brazilië, 15090-000
        • Research Site
      • São Paulo, Brazilië, 05.403-010
        • Research Site
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 4N1
        • Research Site
  • China
      • Suzhou, China, 215006
        • Research Site
      • Tianjin, China, 300020
        • Research Site
  • Duitsland
      • Hamburg, Duitsland, 20246
        • Research Site
      • Ulm, Duitsland, 89081
        • Research Site
  • Frankrijk
      • Angers, Frankrijk, 49033
        • Research Site
      • La Tronche, Frankrijk, 38043
        • Research Site
      • Nice, Frankrijk, 06200
        • Research Site
  • Griekenland
      • Athens, Griekenland, 12462
        • Research Site
      • Pátrai, Griekenland, 26504
        • Research Site
      • Thessaloniki, Griekenland, 57010
        • Research Site
  • Israël
      • Halfa, Israël, 31096
        • Research Site
      • Ramat Gan, Israël, 52621
        • Research Site
  • Italië
      • Roma, Italië, 00168
        • Research Site
      • Udine, Italië, 33100
        • Research Site
  • Japan
      • Akita, Japan, 010-8543
        • Research Site
      • Anjo, Japan, 446-8602
        • Research Site
      • Chiba, Japan, 260-8677
        • Research Site
      • Fukushima, Japan, 960-1295
        • Research Site
      • Isehara-shi, Japan, 259-1193
        • Research Site
      • Kurashiki-shi, Japan, 710-8602
        • Research Site
      • Minatoku, Japan, 105-8470
        • Research Site
      • Okayama, Japan, 700-8558
        • Research Site
      • Osaka, Japan, 545-8586
        • Research Site
      • Osakasayama-shi, Japan, 589-8511
        • Research Site
      • Sapporo, Japan, 060-8638
        • Research Site
      • Suita-shi, Japan, 565-0871
        • Research Site
      • Tsukuba, Japan, 305-8576
        • Research Site
      • Wakayama, Japan, 641-8510
        • Research Site
  • Nederland
      • Groningen, Nederland, 9713 GZ
        • Research Site
  • Spanje
      • Barcelona, Spanje, 08036
        • Research Site
      • Granada, Spanje, 18014
        • Research Site
      • L'Hospitalet de Llobregat, Spanje, 08908
        • Research Site
      • Madrid, Spanje, 28034
        • Research Site
      • Madrid, Spanje, 28046
        • Research Site
      • Madrid, Spanje, 28040
        • Research Site
      • Madrid, Spanje, 28007
        • Research Site
      • Málaga, Spanje, 29010
        • Research Site
      • Pamplona, Spanje, 31008
        • Research Site
      • Salamanca, Spanje, 37007
        • Research Site
      • Seville, Spanje, 41013
        • Research Site
  • Verenigd Koninkrijk
      • London, Verenigd Koninkrijk, W12 0HS
        • Research Site
      • Nottingham, Verenigd Koninkrijk, NG5 1PB
        • Research Site
  • Verenigde Staten
    • Florida
      • Tampa, Florida, Verenigde Staten, 33612
        • Research Site
    • Michigan
      • Grosse Pointe Farms, Michigan, Verenigde Staten, 48236
        • Research Site
    • North Carolina
      • Durham, North Carolina, Verenigde Staten, 27705
        • Research Site
    • Pennsylvania
      • Pittsburgh, Pennsylvania, Verenigde Staten, 15232
        • Research Site
    • Washington
      • Seattle, Washington, Verenigde Staten, 98109
        • Research Site
  • Zuid -Korea
      • Goyang-si, Zuid -Korea, 10408
        • Research Site
      • Seoul, Zuid -Korea, 03080
        • Research Site
      • Seoul, Zuid -Korea, 03722
        • Research Site
      • Seoul, Zuid -Korea, 06351
        • Research Site
  • Zweden
      • Huddinge, Zweden, 141 57
        • Research Site

Deelname Criteria

Onderzoekers zoeken naar mensen die aan een bepaalde beschrijving voldoen, de zogenaamde geschiktheidscriteria. Enkele voorbeelden van deze criteria zijn iemands algemene gezondheidstoestand of eerdere behandelingen.

Geschiktheidscriteria

Leeftijden die in aanmerking komen voor studie

12 jaar tot 100 jaar (Kind, Volwassen, Oudere volwassene)

Accepteert gezonde vrijwilligers

Nee

Geslachten die in aanmerking komen voor studie

Allemaal

Beschrijving

Inclusion Criteria:

  1. 12 years of age or older at time of consent/assent.
  2. Received HSCT within the past 12 months.
  3. Diagnosis of TMA that persists for at least 72 hours after initial management of any triggering agent/condition.
  4. A TMA diagnosis based on meeting the laboratory-based criteria during the Screening Period and/or ≤14 days prior to the Screening Period.
  5. Body weight ≥ 30 kilograms at Screening or ≤7 days prior to the start of the Screening Period (date of consent).
  6. Female participants of childbearing potential and male participants with female partners of childbearing potential must use highly effective contraception.
  7. Participants must be vaccinated against meningococcal infections if clinically feasible. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis. Participants <18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible.
  8. Participants or their legally authorized representative must be capable of giving signed informed consent or assent.

Exclusion Criteria:

  1. Thrombotic thrombocytopenic purpura (TTP) evidenced by ADAMTS13 deficiency
  2. Known Shiga toxin-related hemolytic uremic syndrome as demonstrated by positive test.
  3. Positive direct Coombs test indicative of a clinically significant immune-mediated hemolysis not due to TMA.
  4. Clinical diagnosis of disseminated intravascular coagulation (DIC).
  5. Known bone marrow/graft failure for the current HSCT.
  6. Diagnosis of veno-occlusive disease which is unresolved at the time of Screening.
  7. Human immunodeficiency virus (HIV) infection.
  8. Unresolved meningococcal disease.
  9. Presence of sepsis requiring vasopressor support.
  10. Pregnancy or breastfeeding.
  11. Hypersensitivity to murine proteins or to one of the excipients of ravulizumab.
  12. Any ongoing or history of medical or psychological conditions unrelated to HSCT-TMA that could increase the risk to the participant or confound the outcome of the study.
  13. Respiratory failure requiring mechanical ventilation.
  14. Acute and/or chronic heart failure with an ejection fraction ≤ 40%.
  15. Previously or currently treated with a complement inhibitor.
  16. Participation in an interventional treatment study of any therapy for TMA.

Studie plan

Dit gedeelte bevat details van het studieplan, inclusief hoe de studie is opgezet en wat de studie meet.

Hoe is de studie opgezet?

Ontwerpdetails

  • Primair doel: Behandeling
  • Toewijzing: Gerandomiseerd
  • Interventioneel model: Parallelle opdracht
  • Masker: Verviervoudigen

Wapens en interventies

Deelnemersgroep / Arm
Interventie / Behandeling
Experimenteel: Ravulizumab

In Stage 1, all participants will receive open-label ravulizumab plus Best Supportive Care (BSC).

In Stage 2, participants will receive blinded ravulizumab plus Best Supportive Care (BSC).
Biologisch: Ravulizumab

Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.

Andere namen:
  • Ultomiris, ALXN1210
Ander: Best supportive care

Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).
Placebo-vergelijker: Placebo

In Stage 2, participants randomized to the placebo arm will receive matching placebo plus BSC.
Ander: Placebo

Matching placebo

Ander: Best supportive care

Participants will receive medications, therapies, and interventions per standard hospital treatment protocols (unless specifically prohibited by the protocol).

Wat meet het onderzoek?

Primaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Stage 2: Proportion of Participants With Event-free Survival During the 26-week Treatment Period
Tijdsspanne: Up to Week 26

Event-free survival was defined as the time from randomization until the first of the 2 following events: death (due to any cause) and clinical worsening up through Week 26. Clinical worsening was defined as meeting 2 or more of the following criteria: doubling of serum creatinine from baseline or new requirement of dialysis; increase in lactate dehydrogenase (LDH) >25% compared to baseline; reduction in platelets of >25% compared to baseline; reduction in hemoglobin >10% compared to baseline; increased requirement for platelets or red blood cells at any time. The criteria for clinical worsening had to be confirmed by 2 independent samples collected at least 8 hours apart (ideally 24 hours apart).
Up to Week 26

Secundaire uitkomstmaten

Uitkomstmaat
Maatregel Beschrijving
Tijdsspanne
Stage 2: Overall Survival
Tijdsspanne: Up to Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period)

The Kaplan-Meier method was used to measure overall survival estimate. For Day 100 and Week 26, overall survival was calculated from randomization to death (due to any cause), up to Day 100/Week 26. Participants who survived were censored at the earliest of an additional hematopoietic stem cell transplant (HSCT), Day 100/Week 26, or their last known date alive. For Week 52, overall survival was calculated from randomization to death (due to any cause), up to Week 52. Participants who survived were censored at the earlier of an additional HSCT, or their last known date alive up to Week 52.
Up to Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period)
Stage 2: Non-relapse Mortality
Tijdsspanne: Baseline through Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period)

Cumulative incidence for each group was estimated using a competing risks model. Non-relapse mortality was defined as a participant's death due to any cause during the study, with the exception of death due to underlying disease (malignant or non-malignant reason for HSCT) progression or relapse.
Baseline through Day 100 (Treatment Period), Week 26 (Treatment Period), and Week 52 (Follow-up Period)
Stage 2: Mean Number of Thrombotic Microangiopathy (TMA) Response Criteria Met During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

The TMA response parameters included: platelet response, normalization of LDH and absence of schistocytes, and renal response. A participant could have met 0, 1, 2, or 3 TMA response criteria during the 26-week Treatment Period. If the participants met more than 1 TMA response criterion, the number of responses overlapping for at least 24 hours was counted. Least squares mean determined from a general linear model adjusted by acute GVHD status and geographic region, assuming normal data distribution.
Baseline through Week 26
Stage 2: Percentage of Participants With Hematologic Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Clinical laboratory assessments were performed according to the protocol and assessed against the following predetermined criteria for hematologic response: if baseline platelet count was ≤50000/cubic millimeter (mm^3), then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count had to be >50000/mm^3, then there must be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; normalization of LDH and absence of schistocytes (defined as ≤1% or a result of "none", per the International Council for Standardization in Hematology recommendations for schistocyte counting).
Baseline through Week 26
Stage 2: Change From Baseline in the Estimated Glomerular Filtration Rate (eGFR) at Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

The eGFR was calculated based on serum creatinine using the Chronic Kidney Disease Epidemiology Collaboration (CKD EPI) formula. Serum creatinine was collected according to the protocol. Data reported as milliliters/minute/1.73 square meters (mL/min/1.73 m^2).
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Percentage of Participants With TMA Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

The criteria for TMA response included a hematological response and a renal response. Hematological Response: if baseline platelet count was ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; normalization of LDH and absence of schistocytes. Renal response: at least 50% reduction of proteinuria from baseline. Participants must have met each of the response criteria, with each parameter meeting the requirements at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. A participant could meet 0, 1, 2, or 3 TMA response criteria during the treatment period. If the participant met more than 1 TMA response criterion, the number of responses overlapping for at least 24 hours was counted.
Baseline through Week 26
Stage 2: Cumulative Proportion of Participants Who Experienced a Complete TMA Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Cumulative proportion of participants who experienced a complete TMA response was estimated using a competing risks model based on the time (days) to complete TMA response. Time to complete TMA response was defined as the days from randomization to the first day during the 26-week Treatment Period on which a participant met all criteria for a complete TMA response. The date of complete TMA response was defined as the first date on which all individual response criteria were met simultaneously.
Baseline through Week 26
Stage 2: Cumulative Proportion of Participants Experiencing Each Individual Component of TMA During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Cumulative proportion of participants experiencing individual TMA components included those for platelet count, LDH, and protein/creatinine ratio. For platelet count: if baseline platelet count was ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days. For LDH: normalization of LDH and absence of schistocytes. For protein/creatinine ratio: at least 50% reduction of proteinuria from baseline.
Baseline through Week 26
Stage 2: Cumulative Proportion of Participants Experiencing a Hematologic Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Clinical laboratory assessments were performed according to the protocol and assessed against the following predetermined criteria for hematologic response: if baseline platelet count was ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days.
Baseline through Week 26
Stage 2: Percentage of Participants With Hemoglobin Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Hemoglobin response was defined as the ability to maintain hemoglobin ≥10 grams/deciliter (g/dL) without any support. The criterion had to be met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between, and without any support during the prior 7 days.
Baseline through Week 26
Stage 2: Percentage of Participants With Platelet Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

Platelet response was defined as: if baseline platelet count was ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days. The criteria had to be met at 2 separate assessments obtained at least 24 hours apart, and any measurement in between.
Baseline through Week 26
Stage 2: Percentage of Participants With Partial Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

The criteria for TMA response included a hematological response and a renal response. Hematological response: if baseline platelet count was ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if the baseline platelet count was >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; normalization of LDH and absence of schistocytes. Renal response: at least 50% reduction of proteinuria from baseline. Partial response occurred when the participant met ≥1, but not all, criteria for TMA response.
Baseline through Week 26
Stage 2: Percentage of Participants With Loss of TMA Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

For participants that met the criteria for TMA response during the 26-week Treatment Period, loss of TMA response was defined as when the participant failed to meet the criteria for 1 or more components of TMA response at a subsequent visit during the 26-week Treatment Period. At least 1 parameter must have failed to meet the response criteria at 2 separate assessments obtained at least 24 hours apart, and any measurement in between.
Baseline through Week 26
Stage 2: Duration of TMA Response
Tijdsspanne: Baseline through Week 52

This analysis includes data for each participant after achieving TMA response under the composite strategy through Weeks 26 and 52. The criteria for TMA response included a hematological response and a renal response. Time was calculated as the number of days from TMA response under the composite strategy to first loss of response or intercurrent event in the treatment period, or TMA relapse in the follow-up period. Participants must have met each of the response criteria, with each parameter meeting the requirements at 2 separate assessments obtained at least 24 hours apart, and any measurement in between. Participants with TMA response who did not experience these events were censored at their end of study date. The estimate is calculated based on the Kaplan-Meier method. Non-responders were counted as an event with a duration of zero day.
Baseline through Week 52
Stage 2: Change From Baseline in Haptoglobin at Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Protocol-specified blood samples were taken for the analysis of haptoglobin in plasma at designated time points. Results are reported as grams/liter (g/L).
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in Platelets at Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Protocol-specified blood samples were taken for the analyses of platelets in plasma at designated time points.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in LDH at Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Protocol-specified blood samples were taken for the analyses of LDH in plasma at designated time points. Results are reported as units/liter (U/L).
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in Hemoglobin at Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Protocol-specified blood samples were taken for the analyses of hemoglobin in plasma at designated time points.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Percentage of Participants With Modified TMA Response During the 26-week Treatment Period
Tijdsspanne: Baseline through Week 26

The modified criteria for TMA response included a hematological response and a renal response. Hematological Response: if baseline platelet count ≤50000/mm^3, then the absolute platelet count had to be >50000/mm^3 without platelet transfusion support during the prior 7 days; if baseline platelet count >50000/mm^3, then there had to be a ≥50% increase in platelet count compared to baseline value without platelet transfusion support during the prior 7 days; at least 50% reduction of LDH from baseline and absence of schistocytes or normalization of LDH and absence of schistocytes. Renal response: at least 50% reduction of proteinuria from baseline. Participants must have met each response criterion, with each parameter meeting the requirement at 2 separate assessments obtained at least 24 hours apart, and any measurement in between.
Baseline through Week 26
Stage 2: Change From Baseline in Number of Participants With TMA-associated Organ Dysfunction in the Renal System Through Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Renal dysfunction was defined as doubling of serum creatinine from baseline or requirement of dialysis. Requirements for dialysis included: at the baseline visit, if there was any dialysis within 5 days prior to the first dose of study treatment; at the post-baseline visit, if there was any dialysis within 5 days prior to the visit. Participants categorized as those experiencing no symptoms at baseline and those experiencing symptoms at baseline.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in Number of Participants With TMA-associated Organ Dysfunction in the Cardiopulmonary System Through Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Cardiopulmonary dysfunction was defined as the presence of cardiopulmonary symptoms (excluding hypertension) or use of any mechanical ventilation. Cardiopulmonary symptoms included, but were not limited to, pleural effusion, pulmonary edema, pericardial effusion, and serositis. The presence of cardiopulmonary symptoms at baseline and post-baseline visits were assessed and recorded on the electronic case report form (eCRF). Use of any ventilatory or respiratory support, including mechanical ventilation, was reported on the participant's Nonpharmacologic Therapies and Procedures eCRF page. Participants categorized as those experiencing no symptoms at baseline and those experiencing symptoms at baseline.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in Number of Participants With TMA-associated Organ Dysfunction in the Central Nervous System Through Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Central nervous system dysfunction was defined as confirmed posterior reversible encephalopathy syndrome, or any signs of headache, confusion, visual loss, or seizures at baseline and post-baseline visits, as recorded on the eCRF. Participants categorized as those experiencing no symptoms at baseline and those experiencing symptoms at baseline.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Change From Baseline in Number of Participants With TMA-associated Organ Dysfunction in the Gastrointestinal System Through Week 26 and Week 52
Tijdsspanne: Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)

Gastrointestinal dysfunction was defined as presence of signs and symptoms of gastrointestinal involvement at baseline and post-baseline visits, including diarrhea, vomiting, pain, and bleeding as recorded on the eCRF. Participants categorized as those experiencing no symptoms at baseline and those experiencing symptoms at baseline.
Baseline, Week 26 (Treatment Period), Week 52 (Follow-up Period)
Stage 2: Percentage of Participants With TMA Relapse
Tijdsspanne: Baseline through Week 52

For participants that met the criteria for TMA response during the 26-week Treatment Period, TMA relapse was defined as evidence of worsening hematologic and renal dysfunction due to TMA during the post-treatment Follow-up Period and required treatment intervention, as determined by the investigator.
Baseline through Week 52

Medewerkers en onderzoekers

Hier vindt u mensen en organisaties die betrokken zijn bij dit onderzoek.

Sponsor

Alexion Pharmaceuticals, Inc.

Studie record data

Deze datums volgen de voortgang van het onderzoeksdossier en de samenvatting van de ingediende resultaten bij ClinicalTrials.gov. Studieverslagen en gerapporteerde resultaten worden beoordeeld door de National Library of Medicine (NLM) om er zeker van te zijn dat ze voldoen aan specifieke kwaliteitscontrolenormen voordat ze op de openbare website worden geplaatst.

Bestudeer belangrijke data

Studie start (Werkelijk)

10 december 2020

Primaire voltooiing (Werkelijk)

19 september 2025

Studie voltooiing (Werkelijk)

20 maart 2026

Studieregistratiedata

Eerst ingediend

17 augustus 2020

Eerst ingediend dat voldeed aan de QC-criteria

2 september 2020

Eerst geplaatst (Werkelijk)

10 september 2020

Updates van studierecords

Laatste update geplaatst (Werkelijk)

4 november 2026

Laatste update ingediend die voldeed aan QC-criteria

2 november 2026

Laatst geverifieerd

1 november 2026

Meer informatie

Termen gerelateerd aan deze studie

Trefwoorden

Aanvullende relevante MeSH-voorwaarden

Andere studie-ID-nummers

  • ALXN1210-TMA-313
  • 2020-000144-61 (EudraCT-nummer)

Plan Individuele Deelnemersgegevens (IPD)

Bent u van plan om gegevens van individuele deelnemers (IPD) te delen?

NEE

Informatie over medicijnen en apparaten, studiedocumenten

Bestudeert een door de Amerikaanse FDA gereguleerd geneesmiddel

Ja

Bestudeert een door de Amerikaanse FDA gereguleerd apparaatproduct

Nee

Deze informatie is zonder wijzigingen rechtstreeks van de website clinicaltrials.gov gehaald. Als u verzoeken heeft om uw onderzoeksgegevens te wijzigen, te verwijderen of bij te werken, neem dan contact op met register@clinicaltrials.gov. Zodra er een wijziging wordt doorgevoerd op clinicaltrials.gov, wordt deze ook automatisch bijgewerkt op onze website .

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