Etude : ALXN1210-TMA-313 /

ATTENTION : pour chaque essai clinique, les éléments affichés ci-dessous ne sont pas exhaustifs, et le protocole fourni par le promoteur reste l’unique document à consulter pour mener à bien un essai clinique sur centre. Pour plus d'informations, contactez le référent du territoire concerné.

Acronyme / Nom
Situation thérapeutique
Cadre réglementaire
Présentation de l'étude
Acronyme / Nom : ALXN1210-TMA-313

Situation thérapeutique : Greffe

Traitement :

Cadre réglementaire : RIPH1

Dernière MÀJ : 30/11/2021
CIM10 - Localisation(s)
Informations principales
Titre : A Phase 3, Open-label, Randomized, Multicenter Study of Ravulizumab in Adult and Adolescent Participants who have Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplant (HSCT)

Spécialité : Tissus lymphoïde, hématopoïétique et apparentés
Localisation : C96 - Tumeurs malignes des tissus lymphoïde, hématopoïétique et apparentés, autres et non précisées
Informations complémentaires
Schéma : 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 of the study, the dosing regimen for participants with HSCT-TMA will be confirmed. In Stage 2, participants will be randomized to receive either ravulizumab plus best supportive care or best supportive care only. The treatment period is 26 weeks followed by a 26-week off-treatment follow-up period.

- Experimental: Ravulizumab plus Best Supportive Care
Weight-based doses of ravulizumab will be administered intravenously as loading dose regimen followed by maintenance dosing every 8 weeks.

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

To assess the efficacy of ravulizumab plus BSC versus BSC-only

Safety and tolerability of ALXN1210 and additional efficacy measures

Phase : III

Stade : NA

Informations libres de droit
Critères d'inclusion
Critères de non-inclusion
Informations libres de droit
Critères d'inclusion et de non-inclusion
Critères d'inclusion : 1.12 years of age or older, at the time of signing the informed consent form (ICF)
2.participants who received HSCT within the past 6 months at the time of Screening
3.A TMA diagnosis, based on all of the following criteria occurring simultaneously:
• De novo thrombocytopenia or platelet transfusion refractoriness
• De novo anemia or increase in transfusion requirements
• Either one of the following markers of hemolysis
− LDH > 1.5 × ULN or,
− Presence of schistocytes ≥ 2 high power field (HPF)
• Proteinuria on spot urinalysis
• Presence of hypertension
4.Participants must have HSCT-TMA that persists despite initial management of any triggering condition (persists for at least 72 hours after management of triggering agent/condition)
• Withdrawal or dose reduction of the offending agent (eg, CNIs)
• Treatment of any underlying infection
• Treatment of underlying GVHD
5.Participants must be vaccinated against meningococcal infections if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. Participants < 18 years of age must be re-vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible, according to institutional guidelines for immune reconstitution after HSCT. All participants should be administered coverage with prophylactic antibiotics according to institutional posttransplant infection prophylaxis guidances including coverage against N. meningiditis for at least 2 weeks after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should receive antibiotic prophylaxis coverage against N. meningiditis the entire Treatment Period and for 8 months following the final dose of ravulizumab
6.Male or female
Contraceptive use by men or women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.
7.Capable of giving signed informed consent or assent which includes compliance with the requirements and restrictions listed in the informed consent and in this protocol

Critères de non-inclusion : 1.Known familial or acquired ‘a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13’ (ADAMTS13) deficiency (activity < 5%) .
2.Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
3.Positive direct Coombs test
4.Diagnosis or suspicion of disseminated intravascular coagulation (DIC)
5.Known bone marrow/graft failure
6.Diagnosis of veno-occlusive disease (VOD), regardless of severity
7.Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer,
8.Unresolved meningococcal disease
9.Presence or suspicion of sepsis (treated or untreated) within 7 days prior to Screening
10.Pregnancy or breastfeeding
11.Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab
12.Previously or currently treated with a complement inhibitor
Informations relatives au promoteur
Promoteur :
Alexion Pharmaceuticals
Type de sponsor : Industriel
Alexion Pharmaceuticals - Belgique

Coordonnateur :
Centre investigateur
Informations relatives aux investigateurs
Centre investigateur :
Centre Hospitalier Universitaire de Lille - 2 Avenue Oscar Lambret - 59000 LILLE

Investigateur :
Docteur Valérie Coiteux

Secrétariat de recherche

Statut de l'essai : OUVERT

MAJ : 26/01/2021