Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to control TMA as characterized by thrombocytopenia, hemolysis and renal impairment.
ID
Source
Brief title
Condition
- Haemolyses and related conditions
- Immune disorders NEC
- Nephropathies
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Efficacy and safety.
Secondary outcome
Efficacy.
Background summary
Atypical hemolytic uremic syndrome (aHUS) is a very rare disease, with an
estimated incidence of only approximately 3 per million in children less than
18 years of age (1). The prevalence in adults is believed to be even lower.
Most patients afflicted with aHUS develop the disease before 10 years of age,
but initial presentations may also occur in patients between 20-40 years of age
(2). All patients with aHUS exhibit evidence of uncontrolled complement
activation with resulting pro-thrombotic and pro-inflammatory perturbations.
About 50-60% of aHUS cases have known aberrations in complement regulatory
proteins-either mutations or antibodies(3). The development of thrombotic
microangiopathy (TMA) in patients with aHUS represents a pro-coagulant state
and is presumed due to uncontrolled terminal complement
activation that occurs secondary to predominantly proximal alternative
complement pathway activation (4;5). Current treatment for patients with aHUS
is considered inadequate. Eculizumab is a terminal complement inhibitor and its
mechanism of action suggests possible utility in the treatment of this disease.
Study objective
Assess the efficacy and safety of eculizumab in pediatric patients with aHUS to
control TMA as characterized by thrombocytopenia, hemolysis and renal
impairment.
Study design
This is an open-label, non-randomized, single-arm multi-center clinical trial
of eculizumab in pediatric patients (1 month up to 18 years) with body weight
of >= 5kg) with aHUS.
Intervention
Eculizumab. Fixed dosing is based on body weight cohorts. Adjustment of dose to
accommodated patient growth is possible.
Study burden and risks
See section E9.
352 Knotter Drive
Cheshire
US
352 Knotter Drive
Cheshire
US
Listed location countries
Age
Inclusion criteria
1. Patient*s parent/legal guardian must be willing and able to give written informed consent and the patient must be willing to give written informed assent [if applicable as determined by the central Institutional Review Boards/Independent Ethics Committees (IRB/IEC)].
2. Pediatric patients with aHUS. Patients may be newly diagnosed, or with previously diagnosed disease, or post-kidney transplant with the disease.
3. Patients from 1 month up to 18 years of age and body weight >= 5 kg.
4. Patients exhibit Thrombocytopenia, hemolysis and elevated Serum Creatinine.
Exclusion criteria
1. Plasma Therapy for > 5 weeks prior to enrollment.
2. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for end stage renal disease).
3. Prior eculizumab use or hypersensitivity to eculizumab, to murine proteins or to one of the excipients.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2010-020310-28-NL |
| ClinicalTrials.gov | NCT01193348 |
| CCMO | NL33553.091.10 |