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Clinical Trials/NCT03303313
NCT03303313WithdrawnPhase 2

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

Alnylam Pharmaceuticals1 site in 1 countryStarted: September 19, 2017Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 2
Status
Withdrawn
Sponsor
Locations
1
Primary Endpoint
The effect of Cemdisiran on platelet count
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
18 Years to — (Adult, Older Adult)
Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • •Willing to provide written informed consent and to comply with the study requirements
  • •Age 18 years or older
  • •Clinical diagnosis of primary aHUS
  • •Clinical thrombotic microangiopathy (TMA) activity
  • •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
  • •Previously vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations
  • •ADAMTS13 >10% or other proven aHUS-associated mutation

Exclusion Criteria

  • •Clinically significant abnormal laboratory results
  • •Positive Shiga toxin producing Escherichia coli test at Screening
  • •Suspected secondary aHUS, in the opinion of the Investigator (unless there is a documented aHUS-associated genetic mutation)
  • •Positive direct Coombs test
  • •Patients who have received hemodialysis for >3 months
  • •Bone marrow transplant recipients
  • •Organ transplant recipients, except for kidney transplant recipients with primary aHUS (confirmed by known genetic mutation and kidney biopsy)
  • •Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody syndrome
  • •History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
  • •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
  • •Patients with a poor prognosis that is expected to limit their life expectancy to less than 3 months, in the opinion of the Investigator

Arms & Interventions

Cemdisiran

Experimental

Intervention: Cemdisiran (Drug)

Outcomes

Primary Outcomes

The effect of Cemdisiran on platelet count

Time Frame: Week 32

Secondary Outcomes

  • The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy(after 32 weeks of treatment)
  • The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)(up to 84 weeks)
  • The effect of Cemdisiran on hematological response as measured by platelet count(after 32 weeks of treatment)
  • The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count(after 32 weeks of treatment)
  • The effect of Cemdisiran on serum creatinine levels(up to 84 weeks)
  • The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH(after 32 weeks of treatment)
  • The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels(after 32 weeks of treatment)
  • The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)(after 32 weeks of treatment)
  • The effect of Cemdisiran on hematological response as measured by rescue plasma therapy(after 32 weeks of treatment)
  • The effect of Cemdisiran on LDH response as measured by LDH(after 32 weeks of treatment)
  • The effect of Cemdisiran on LDH response as measured by rescue plasma therapy(after 32 weeks of treatment)
  • The effect of Cemdisiran on adverse events (AEs)(up to 108 weeks)

Investigators

Sponsor
Alnylam Pharmaceuticals
Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (1)

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