Clinical trial

Personalized Spacing of Eculizumab Infusions Based on Therapeutic Pharmacological Monitoring: Prospective Health-economic Evaluation in Patients in Remission With Atypical Hemolytic Uraemic Syndrome Requiring Long-term Treatment.

Name

DR200088-EspacECU

Description

Eculizumab is an anti-C5 monoclonal antibody approved for rare diseases including atypical haemolytic-uraemic syndrome. The maintenance phase dosing regimen is identical for all adult patients but several studies have shown a high interindividual kinetics variability. A tailored administration of eculizumab based on therapeutic drug monitoring will be compared with real-life administration in adults suffering from an atypical haemolytic uraemic syndrome. The objective is to improve efficiency of eculizumab administration.

Trial arms

Trial start

2021-11-01

Estimated PCD

2023-12-28

Trial end

2024-06-01

Status

Active (not recruiting)

Phase

Early phase I

Treatment

Spacing of Eculizumab infusions

Personalized spacing of eculizumab infusions using a pharmacokinetic population model to estimate eculizumab concentration ( a one-week spacing of eculizumab infusion will be decide if predicted eculizumab concentration in the event of spacing is \> 150 mg/L). Spacing of infusion can be decided every 3 months.

Arms:

Therapeutic drug monitoring

Size

Primary endpoint

Incremental Cost-Utility Ratio (ICUR) at 18 months of a personalized spacing of eculizumab infusions compared to the usual administration scheme without personalization, from the French health insurance perspective.

18 months

Eligibility criteria

Inclusion Criteria: 1. Adults with an atypical Hemolytic and Uremic Syndrome defined by at least 2 of the following parameters: * Thrombopenia (platelet count \< 150 G/L) * Mechanical lytic anemia (Hb \< 10 g/L, Lactate dehydrogenase \> upper limit of normal, undetectable haptoglobin, presence of schistocytes on blood smear) * Acute renal failure 2. Eculizumab treatment * Without project of withdrawal in the next 18 months * Started since at least 6 months * Administrated with an unchanged administration schedule since at least 3 months (initial episode or relapse) 3. Atypical Hemolytic and Uremic Syndrome in remission 4. Patients who give informed consent. Exclusion Criteria: 1. Patients on dialysis. 2. Women treated starting or planning a pregnancy. 3. Patients suffering from a typical or secondary microangiopathic hemolytic anemia (drugs, malignancies, autoimmune disease...) 4. Patients under protection of a judicial authority

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'Randomized (2:1) controlled study, open-label, with Markov modeling.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 40, 'type': 'ACTUAL'}}

Updated at

2024-03-01

1 organization

1 product

2 indications

Organization

University Hospital of Tours

Product

Eculizumab

Indication

Hemolytic Uremic Syndrome

Indication

atypical