EVIDENCE - EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT

• Register: EUCTR
• Last refreshed on: 14 May 2018
• Main ID: EUCTR2015-003135-35-IT
• Date of registration: 20/04/2018
• Prospective Registration: Yes
• Primary sponsor: ALEXION PHARMACEUTICALS INCORPORATED
• Public title: EVIDENCE - EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT
• Scientific title: EVIDENCE - EVALUATION OF POTENTIAL PREDICTORS OF DISEASE PROGRESSION IN PATIENTS WITH aHUS, INCLUDING GENETICS, BIOMARKERS, AND TREATMENT - EVIDENCE
• Target sample size: 330
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-003135-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: Cross over: Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): yes

Countries of recruitment

Australia, Belgium, Canada, Czech Republic, Germany, Israel, Italy, Norway, Poland, Russian Federation, Spain, United Arab Emirates, United Kingdom, United States

Contacts

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

• Name: European Clinical Trial Information
• Affiliation: Alexion Europe SAS

Key inclusion & exclusion criteria

Inclusion criteria:
1. Currently receiving eculizumab treatment in the M11-001 aHUS Registry
2. Two normal platelet counts at least 4 weeks apart
3. Two normal LDH levels at least 4 weeks apart
4. Willing, committed, and able to return for all clinic visits and complete all study related procedures
5. Patient or patient's parent/legal guardian must be willing and able to give written informed consent. Patient (if minor) must be willing to give written informed assent (if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees [IRB/IEC]).
Are the trial subjects under 18? yes
Number of subjects for this age range: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 155
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion criteria:
1. Any prior intentional discontinuation of eculizumab treatment
2. On chronic dialysis (defined as =3 months on dialysis)
3. Currently participating in another complement inhibitor trial
4. With life expectancy of <6 months
5. Patient or patient's parent/legal guardian unable to give written informed consent. Patient (if minor) unable to give written informed assent (if applicable as determined by the central Institutional Review
Boards/Independent Ethics Committees [IRB/IEC]).


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

atypical Hemolytic-Uremic Syndrome (aHUS)
MedDRA version: 20.0 Level: LLT Classification code 10019515 Term: Hemolytic uremic syndrome System Organ Class: 100000004851

Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Intervention(s)

Trade Name: SOLIRIS - 300 MG CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO 1 FLACONCINO (VETRO) 30 ML (10 MG/ML)
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Eculizumab
CAS Number: 219685-50-4
Current Sponsor code: /
Other descriptive name: /
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 300-

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: From week 2 up to month 47

Primary end point(s): Rate of TMA manifestations, where TMA includes:
1. Hematologic or renal events due to aHUS
2. Extra-renal clinical signs and symptoms of aHUS
3. Tissue (eg, kidney transplant) biopsy demonstrating TMA due to aHUS

Main Objective: To assess TMA disease manifestations in patients with aHUS with or without ongoing eculizumab treatment in a real-world setting.

Secondary Objective: To evaluate potential clinical predictors of disease manifestation and progression, including clinical characteristics and genetic profiling to evaluate disease activity and relationship to treatment as determined by levels of biomarkers associated with complement activation, and the use of associated supportive interventions such as PE/PI, dialysis, blood transfusions, and renal transplantation.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: From week 2 up to month 47

Secondary end point(s): • Change in estimated Glomerular Filtration Rate (eGFR) over time
• Rate of TMA manifestations
• Incidence of PE/PI

Secondary ID(s)

ECU-aHUS-403

2015-003135-35-DE

NCT02614898

Source(s) of Monetary Support

Alexion Pharmaceuticals Inc