ResearchStudyIsLowInterventionTrial: false

Artifact Author: Computable Publishing®: CTIS-to-FEvIR Converter:

url: https://fevir.net/resources/ResearchStudy/346485

identifier: FEvIR Object Identifier/https://fevir.net/FOI/346485, https://euclinicaltrials.eu/ctis/2024-516402-32-00 (use: official, ), https://clinicaltrials.gov/NCT05726916 (use: official, )

name: CTIS_2024_516402_32_00_FHIR_Transform

title: Eculizumab in hypertensive emergency-associated hemolytic uremic syndrome: a randomized multicenter controlled trial (HYPERSHU)

label

type: Public title

value: Eculizumab in hypertensive emergency-associated hemolytic uremic syndrome: a randomized multicenter controlled trial (HYPERSHU)

label

type: Short title

value: APHP211039

label

type: French

value: Eculizumab dans le syndrome hémolytique et urémique associé à l'urgence hypertensive: essai contrôlé multicentrique randomisé (HYPERSHU)

label

type: French of public title

value: Eculizumab dans le syndrome hémolytique et urémique associé à l'urgence hypertensive: essai contrôlé multicentrique randomisé (HYPERSHU)

citeAs:

relatesTo

type: Transforms

target: https://euclinicaltrials.eu/ctis-public-api/retrieve/2024-516402-32-00

relatesTo

type: Transformed With

target: null @ https://fevir.net

status: Active

condition: Adult patients with aHUS associated with HE and severe kidney involvement (needing dialysis or serum creatinine ≥ 354μM), Atypical haemolytic uraemic syndrome

period: 2024-02-13 --> 2027-02-13

associatedParty

name: DGOS - French Ministry of Health

role: Funding source

associatedParty

name: Assistance Publique Hopitaux De Paris

role: Sponsor

party: Assistance Publique Hopitaux De Paris

associatedParty

name: Dr El Karoui Khalil

role: public contact

party: Dr El Karoui Khalil khalil.el-karoui@aphp.fr

associatedParty

name: Dr El Karoui Khalil

role: scientific contact

party: Dr El Karoui Khalil khalil.el-karoui@aphp.fr

ProgressStatuses

Recruitments

objective

name: To demonstrate the efficacy of early Eculizumab administration added on standard of care BP control on dialysis-dependence at month 6 in HEaHUS patients with severely impaired initial renal function.

type: Primary

objective

name: To evaluate the role of complement biomarkers in predicting therapeutic response

type: Secondary

objective

name: To evaluate the frequency of complement genetic rare variants in this population

type: Secondary

objective

name: To evaluate the efficacy of early eculizumab administration added on standard of care BP control on 3-month (W13) and 12 months renal replacement therapy risk

type: Secondary

objective

name: To evaluate the frequency of severe infections in both groups

type: Secondary

objective

name: To evaluate the time to resolution of hemolysis and acute dialysis

type: Secondary

objective

name: To evaluate the frequency of histological lesions and their prognosis (if kidney biopsy is performed)

type: Secondary

objective

name: To evaluate the medico-economic impact of Eculizumab

type: Secondary

objective

name: Objective of the first ancillary study: A first ancillary study will include a pangenomic analysis aimed at identifying new genetic variants associated with HE-aHUS. This ancillary study will be based on the residual genetic samples already scheduled in the biocollection. This ancillary study will be coordinated by Pr L. Mesnard (Nephrology Department, Tenon Hospital, Paris) and performed according to an already available funding

type: Secondary

objective

name: Objective of any other potential ancillary study: biological collection For any other ancillary study: residual samples will be stored for a biological collection to identify new potential genetic and pathophysiological determinants of HE-aHUS (Department of Immunobiology, HEGP, Dr Frémeaux-Bacchi).

type: Secondary

objective

name: Primary Endpoints

type: Primary

OutcomeMeasures

-	Name	Type	Endpoint
*	Response to therapy, defined by the absence of any of the following events: i) at 6-month follow-up: persistent renal replacement therapy, eGFR<15ml/mn/1,73m2, or patient death; ii) at W2 : persistent hemolysis despite well conducted antihypertensive therapy. Any Eculizumab rescue in the control group will be considered as a failure.	Primary	CTIS2024-516402-32-00 Primary Outcome 1

objective

name: Secondary Endpoints

type: Secondary

outcomeMeasure

name: Prognosis role of repeat complement biomarkers (W1, W4, W13)

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 1

outcomeMeasure

name: Frequency of Complement genetic rare variants

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 2

outcomeMeasure

name: Renal replacement therapy rates at months 3 and 12 follow-up

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 3

outcomeMeasure

name: Frequency of severe infections (defined by the need for hospitalization)

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 4

outcomeMeasure

name: Time to resolution of haemolysis

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 5

outcomeMeasure

name: Time to resolution of renal replacement therapy

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 6

outcomeMeasure

name: Frequency and prognostic role of kidney lesions (glomerular and arteriolar microthromboses), glomerulosclerosis and kidney fibrosis if kidney biopsy is performed

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 7

outcomeMeasure

name: Costs relating to renal replacement therapy (or lack of), Eculizumab and other antihypertensive treatments, hospitalizations

type: Secondary

endpoint: CTIS2024-516402-32-00 Secondary Outcome 8

Generated Narrative: Organization #organization-554686

name: Assistance Publique Hopitaux De Paris

description:

Hospital/Clinic/Other health care facility

Generated Narrative: PractitionerRole #contact-190857

organization: Assistance Publique Hopitaux De Paris

display: Dr El Karoui Khalil khalil.el-karoui@aphp.fr

Contacts

-	Name	Telecom
*	Dr El Karoui Khalil	khalil.el-karoui@aphp.fr, +33156016317

Generated Narrative: PractitionerRole #contact-190858

organization: Assistance Publique Hopitaux De Paris

display: Dr El Karoui Khalil khalil.el-karoui@aphp.fr

Contacts

-	Name	Telecom
*	Dr El Karoui Khalil	khalil.el-karoui@aphp.fr, +33156016317