A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome

Alnylam Pharmaceuticals, Inc.0 个研究点目标入组 24 人开始时间: 2017年6月20日最近更新: 2019年2月28日

适应症

试验速览

暂无简介。

•1. Willing to provide written informed consent and to comply with the study requirements
•2. Age 18 years or older
•3. Clinical diagnosis of primary aHUS
•4. Clinical thrombotic microangiopathy (TMA) activity as defined by:
•a. Evidence of low platelet count and
•b. Evidence of hemolysis and
•c. Evidence of impaired renal function
•5. ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) >10%
•6. Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must be willing to use a highly effective method of contraception
•7. Vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations as well as prophylactic antibiotic treatment for at least 2 weeks after completing the recommended vaccination series or longer, if required by local standard of care.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 22
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 2

•1. Abnormal liver function tests
•2. Positive Shiga toxin producing Escherichia coli test
•3. Secondary aHUS
•4. Positive direct Coombs test
•5. Prior eculizumab exposure within 3 months
•6. On dialysis for 3 months or longer
•7. Transplant recipient
•8. Patients considered non-eculizumab responders. except for those with known C5 polymorphisms
•9. Patients who have received hemodialysis for >3 months
•10. Patients who require hemodialysis or plasma exchange more frequently than every 24 hours
•11. Clinical laboratory test results or concomitant conditions considered clinically relevant and unacceptable in the opinion of the Investigator

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