Assess the efficacy of eculizumab in adult 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
The proportion of patients with aHUS with complete TMA response.
Secondary outcome
Safety and 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 proteinseither 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 of eculizumab in adult 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 patients *18 years with aHUS.
Intervention
Eculizumab 600 mg, 900 mg or 1200 mg will be administered intravenously.
Study burden and risks
See section E9.
352 Knotter Drive
Cheshire
USA
352 Knotter Drive
Cheshire
USA
Listed location countries
Age
Inclusion criteria
1. Patient must be willing and able to give written informed consent.
2. Patient*s age * 18 years.
3. Patients exhibit thrombocytopenia, hemolysis and elevated Serum Creatinine
Exclusion criteria
1. Chronic dialysis
2. Prior eculizumab use or hypersensitivity to eculizumab
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 | EUCTR2010-020326-18-NL |
| ClinicalTrials.gov | NCT01194973 |
| CCMO | NL33539.058.10 |