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

September 27, 2018 updated by: Alnylam Pharmaceuticals

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

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

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cemdisiran
Drug: Cemdisiran
Subcutaneous (sc) injection of Cemdisiran

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
The effect of Cemdisiran on platelet count
Time Frame: Week 32
Week 32

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Alnylam Pharmaceuticals

Investigators

  • Study Director: Nader Najafian, MD, Alnylam Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 19, 2017

Primary Completion (Actual)

September 12, 2018

Study Completion (Actual)

September 12, 2018

Study Registration Dates

First Submitted

September 25, 2017

First Submitted That Met QC Criteria

October 2, 2017

First Posted (Actual)

October 6, 2017

Study Record Updates

Last Update Posted (Actual)

October 1, 2018

Last Update Submitted That Met QC Criteria

September 27, 2018

Last Verified

September 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ALN-CC5-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Undecided

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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