Study on Ravulizumab for Patients with Thrombotic Microangiopathy After Stem Cell Transplant

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What is this study about?

This clinical trial is focused on studying a condition called Thrombotic Microangiopathy (TMA), which can occur after a Hematopoietic Stem Cell Transplant (HSCT). TMA is a serious condition that affects the small blood vessels and can lead to complications such as low platelet counts, anemia, and organ damage. The study is testing a treatment called Ravulizumab, which is given as an infusion into a vein. This treatment is being compared to a placebo to see how effective it is in treating TMA in adults and adolescents who have undergone HSCT.

The purpose of the study is to assess how well Ravulizumab works in treating TMA. Participants in the study will receive either Ravulizumab or a placebo. The study will last for several months, during which participants will receive regular infusions and have their health monitored closely. The study aims to see if Ravulizumab can help improve symptoms of TMA and prevent further complications.

Throughout the study, participants will be monitored for changes in their health, including any improvements in organ function and overall survival. The study will also track how quickly participants respond to the treatment and if there are any relapses of TMA. This research is important for finding better ways to treat TMA in patients who have had a stem cell transplant.

1 joining the study

Upon joining the study, eligibility is confirmed based on specific criteria such as age, recent hematopoietic stem cell transplant (HSCT), and a diagnosis of thrombotic microangiopathy (TMA).

Participants must be vaccinated against certain infections and may need to follow specific antibiotic guidelines.

2 treatment initiation

The treatment involves the administration of ravulizumab or a placebo. Ravulizumab is given as a solution for infusion through an intravenous route.

The dosage and frequency of administration are determined by the study protocol.

3 monitoring and assessments

Regular monitoring is conducted to assess the response to treatment. This includes evaluating the response of TMA and any changes in organ function.

Assessments occur at various intervals, including at 26 weeks and 52 weeks, to track changes from the baseline.

4 follow-up period

During the follow-up period, the study continues to monitor for TMA relapse and overall survival.

The follow-up period extends to specific time points, such as Day 100, Week 26, and Week 52.

5 completion of the study

The study is estimated to conclude by June 2025, with all data collected and analyzed to determine the efficacy of ravulizumab compared to the placebo.

Who Can Join the Study?

  • Must be 12 years of age or older at the time of signing the informed consent form.
  • Must have received a hematopoietic stem cell transplant (HSCT) within the past 12 months.
  • Must have a thrombotic microangiopathy (TMA) diagnosis, which includes:
    • New onset of thrombocytopenia (low platelet count) or not responding to platelet transfusions.
    • Any one of the following signs of hemolysis (breakdown of red blood cells):
      • LDH (lactate dehydrogenase) levels higher than normal for age.
      • Presence of schistocytes (fragmented red blood cells) in blood.
    • Proteinuria (protein in urine) on a urine test.
    • New onset of anemia (low red blood cell count) or hypertension (high blood pressure).
  • Must have HSCT-TMA that continues despite initial treatment for at least 72 hours.
  • Must weigh 30 kg or more at the time of screening or within 7 days before screening.
  • Must be vaccinated against meningococcal infections if possible, following guidelines for immune recovery after HSCT. Participants under 18 should also be revaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if possible. All participants should receive preventive antibiotics, including protection against N. meningiditis, for at least 2 weeks after the meningococcal vaccine. If unable to receive the vaccine, antibiotic protection should continue throughout the treatment and for 8 months after the last dose of ravulizumab.
  • Must agree to use contraception if applicable, following local regulations for clinical studies.
  • Must be able to provide signed informed consent or assent, agreeing to follow the study’s requirements and restrictions.

Who Cannot Join the Study?

  • Patients who have had a hematopoietic stem cell transplant but do not have thrombotic microangiopathy. This is a condition where small blood vessels become damaged and form clots.
  • Patients who are not within the specified age range for the study.
  • Patients who are not part of the specified clinical trial groups.
  • Patients who are not considered part of the vulnerable population selected for the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Casa Sollievo Della Sofferenza San Giovanni Rotondo Italy
Centre hospitalier universitaire de Liege Liege Belgium
Azienda Sanitaria Universitaria Friuli Centrale Udine Italy
Hospital General Universitario Gregorio Maranon Madrid Spain
Hospital Universitario Puerta De Hierro De Majadahonda Majadahonda Spain
Geniko Nosokomeio Thessalonikis George Papanikolaou Thessaloniki Greece
Hospital Universitario Virgen De Las Nieves Granada Spain
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona Salerno Italy
AORN San Giuseppe Moscati Avellino Avellino Italy
HELIOS Klinikum Berlin-Buch GmbH Berlin Germany
Evangelismos S.A. Athens Greece
Virgen del Rocío University Hospital Sevilla Spain
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Ospedale San Raffaele S.r.l. Milan Italy
Region Skane Skanes Universitetssjukhus Lund Sweden
Alexandra Hospital Athens Greece
Hospital Clinic De Barcelona Barcelona Spain
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Karolinska University Hospital Solna Sweden
Az St-Jan Brugge-Oostende A.V. Brugge Belgium
Klinikum Chemnitz gGmbH Chemnitz Germany
Hospital Universitario Fundacion Jimenez Diaz Madrid Spain
Universita’ Politecnica Delle Marche Ancona Italy
General University Hospital Of Patras Patras Greece
Centre Hospitalier Universitaire De Nice Nice France
Institut fuer Klinische Transfusionsmedizin und Immungenetik Ulm gGmbH Ulm Germany
Servei De Salut De Les Illes Balears Palma Spain
Centre Hospitalier Universitaire Dinant Godinne Sainte-Elisabeth-UCL-Namur Namur Belgium
Cmdmgzbwk Urnzgxktgmhssp Soocyycii Woluwe-Saint-Lambert Belgium
Utixowznxo Mrbvzto Ctumfg Hxuseahiewrpftjxh Hamburg Germany
Uglihmxhvaun Msyltau Cnwvnql Gpibrnqne Groningen The Netherlands
Hgcurvfe Uxujppkgogjaw Dj La Pmpybyxi Madrid Spain
Igjdkqup Clxtxx Dbhtsnmqfsbrlaekn L'hospitalet De Llobregat Spain
Avgfwin Ovqogbyrrna Ubxigmsxjhwtw Cvxewnhynkcn Dinnq Sxdblw E Delxi Skedbji Dx Tpgxin Turin Italy
Uigjkmviprqxcy Cwkciub Kunfilrox Gdansk Poland
Hbofdguo Dd Lm Sjyxy Chdm I Saqw Pte Barcelona Spain
Usqlcttopx Dyrmk Spyeh De Rbir Lq Shaqareq Rome Italy
Mapfubtklgwqlfurhigucuxacr Hzwbjojjbqgggexn Halle (Saale) Germany
Cqjngo Hvbwrniverm Rohrovgy Dnltfcqmfrnzwl Angers France
Fbhrperxe Psxy Lp Ikrqakuxlbjmu Bpswimaar Dlb Hcpkqjjd Uvbukyzydobid Lc Pvp Madrid Spain
Hoiwhqzm Vfzk drfuumxn Barcelona Spain
Ujowuiguhd Gcisueq Hqzayldp Ajohafd Athens Greece

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
01.10.2020
France France
Not recruiting
01.10.2020
Germany Germany
Not recruiting
01.10.2020
Greece Greece
Not recruiting
01.10.2020
Italy Italy
Not recruiting
01.10.2020
Poland Poland
Not recruiting
01.10.2020
Spain Spain
Not recruiting
01.10.2020
Sweden Sweden
Not recruiting
01.10.2020
The Netherlands The Netherlands
Not recruiting
01.10.2020

Trial locations

Investigated drugs:

Ravulizumab is a medication being studied for its effectiveness in treating a condition called thrombotic microangiopathy (TMA) that can occur after a hematopoietic stem cell transplant (HSCT). This medication works by targeting specific proteins in the immune system to help reduce the damage to blood vessels and improve blood flow. The goal of using ravulizumab in this trial is to see if it can help patients recover from TMA more effectively than not using the medication.

Investigated diseases:

Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy – This condition occurs after a hematopoietic stem cell transplant, where small blood vessels become damaged and clots form in the vessels. It can lead to reduced blood flow to various organs, causing potential damage. The disease affects multiple systems, including the kidneys, heart, lungs, central nervous system, and gastrointestinal tract. Symptoms may include high blood pressure, low platelet count, and signs of organ dysfunction. The progression of the disease can vary, with some individuals experiencing more severe symptoms than others. It is considered a rare disease, and its impact can be significant on the patient’s overall health.

Trial ID:
2023-510107-22-00
Protocol code:
ALXN1210-TMA-313
NCT ID:
NCT04543591
Trial Phase:
Therapeutic confirmatory (Phase III)

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