An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injectio

Phase 1

Conditions: Atypical Hemolytic Uremic Syndrome
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]

Registration Number: EUCTR2017-001082-24-LV

Lead Sponsor: Alnylam Pharmaceuticals, Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 24

Inclusion Criteria

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

Exclusion Criteria

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the effect of ALN-CC5 on platelet response in adult patients with aHUS;<br> Secondary Objective: 1. To evaluate the effect of ALN-CC5 on hematological response<br> 2. To assess the effect of ALN-CC5 on thrombotic microangiopathy (TMA) response<br> 3. To assess the effect of ALN-CC5 on renal function parameters<br> 4. To evaluate the safety and tolerability of ALN-CC5<br> ;Primary end point(s): Proportion of patients with a platelet response at Week 32, with platelet response defined as platelet count >lower limit of normal (LLN) AND no rescue plasma therapy within the preceding 90 days.;Timepoint(s) of evaluation of this end point: At week 32.

Secondary Outcome Measures

Name	Time	Method

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