A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03303313
• Date of registration: 25/09/2017
• Prospective Registration: No
• Primary sponsor: Alnylam Pharmaceuticals
• Public title: A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
• Scientific title: A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
• Date of first enrolment: September 19, 2017
• Target sample size: 0
• Recruitment status: Withdrawn
• URL: https://clinicaltrials.gov/show/NCT03303313
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Bosnia and Herzegovina, Canada, Estonia, Georgia, Latvia, Lithuania, Macedonia, The Former Yugoslav Republic of, Moldova, Republic of, Serbia, Sweden

Contacts

• Name: Nader Najafian, MD
• Affiliation: Alnylam Pharmaceuticals

Key inclusion & exclusion criteria

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

5. 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

6. Previously vaccinated with meningococcal group ACWY conjugate vaccine and
meningococcal group B vaccine or willingness to receive these vaccinations

7. ADAMTS13 >10% or other proven aHUS-associated mutation

Exclusion Criteria:

1. Clinically significant abnormal laboratory results

2. Positive Shiga toxin producing Escherichia coli test at Screening

3. Suspected secondary aHUS, in the opinion of the Investigator (unless there is a
documented aHUS-associated genetic mutation)

4. Positive direct Coombs test

5. Patients who have received hemodialysis for >3 months

6. Bone marrow transplant recipients

7. Organ transplant recipients, except for kidney transplant recipients with primary aHUS
(confirmed by known genetic mutation and kidney biopsy)

8. Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody
syndrome

9. History of multiple drug allergies or history of allergic reaction to an
oligonucleotide or GalNAc

10. Malignancy (except for non-melanoma skin cancers, cervical in-situ carcinoma, breast
ductal carcinoma in situ, or stage 1 prostate carcinoma) within the last 5 years

11. Patients with a poor prognosis that is expected to limit their life expectancy to less
than 3 months, in the opinion of the Investigator



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Atypical Hemolytic Uremic Syndrome

Intervention(s)

Drug: Cemdisiran

Primary Outcome(s)

The effect of Cemdisiran on platelet count [Time Frame: Week 32]

Secondary Outcome(s)

The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH) [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on hematological response as measured by platelet count [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on adverse events (AEs) [Time Frame: up to 108 weeks]

The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on estimated glomerular filtration rate (eGFR) [Time Frame: up to 84 weeks]

The effect of Cemdisiran on LDH response as measured by rescue plasma therapy [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on hematological response as measured by rescue plasma therapy [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on serum creatinine levels [Time Frame: up to 84 weeks]

The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels [Time Frame: after 32 weeks of treatment]

The effect of Cemdisiran on LDH response as measured by LDH [Time Frame: after 32 weeks of treatment]

Secondary ID(s)

ALN-CC5-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors