search
Back to results

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

Primary Purpose

Atypical Hemolytic Uremic Syndrome

Status
Withdrawn
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Cemdisiran
Sponsored by
Alnylam Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Atypical Hemolytic Uremic Syndrome focused on measuring RNAi therapeutic, Atypical Hemolytic Uremic Syndrome, Hemolysis, Thrombocytopenia, Renal insufficiency, Thrombotic Microangiopathy, aHUS, TMA

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site
  • Clinical Trial Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Cemdisiran

Arm Description

Outcomes

Primary Outcome Measures

The effect of Cemdisiran on platelet count

Secondary Outcome Measures

The effect of Cemdisiran on hematological response as measured by platelet count
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
The effect of Cemdisiran on LDH response as measured by LDH
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
The effect of Cemdisiran on serum creatinine levels
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
The effect of Cemdisiran on adverse events (AEs)

Full Information

First Posted
September 25, 2017
Last Updated
September 27, 2018
Sponsor
Alnylam Pharmaceuticals
search

1. Study Identification

Unique Protocol Identification Number
NCT03303313
Brief Title
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
Official Title
A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
September 2018
Overall Recruitment Status
Withdrawn
Why Stopped
Study terminated early due to lack of enrollment.
Study Start Date
September 19, 2017 (Actual)
Primary Completion Date
September 12, 2018 (Actual)
Study Completion Date
September 12, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Alnylam Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Atypical Hemolytic Uremic Syndrome
Keywords
RNAi therapeutic, Atypical Hemolytic Uremic Syndrome, Hemolysis, Thrombocytopenia, Renal insufficiency, Thrombotic Microangiopathy, aHUS, TMA

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cemdisiran
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Cemdisiran
Intervention Description
Subcutaneous (sc) injection of Cemdisiran
Primary Outcome Measure Information:
Title
The effect of Cemdisiran on platelet count
Time Frame
Week 32
Secondary Outcome Measure Information:
Title
The effect of Cemdisiran on hematological response as measured by platelet count
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on LDH response as measured by LDH
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
Time Frame
after 32 weeks of treatment
Title
The effect of Cemdisiran on serum creatinine levels
Time Frame
up to 84 weeks
Title
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
Time Frame
up to 84 weeks
Title
The effect of Cemdisiran on adverse events (AEs)
Time Frame
up to 108 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nader Najafian, MD
Organizational Affiliation
Alnylam Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
Sarajevo
Country
Bosnia and Herzegovina
Facility Name
Clinical Trial Site
City
Calgary
ZIP/Postal Code
T2N 2T9
Country
Canada
Facility Name
Clinical Trial Site
City
Tallinn
Country
Estonia
Facility Name
Clinical Trial Site
City
Tartu
Country
Estonia
Facility Name
Clinical Trial Site
City
Tbilisi
Country
Georgia
Facility Name
Clinical Trial Site
City
Riga
Country
Latvia
Facility Name
Clinical Trial Site
City
Kaunas
Country
Lithuania
Facility Name
Clinical Trial Site
City
Vilnius
Country
Lithuania
Facility Name
Clinical Trial Site
City
Skopje
Country
Macedonia, The Former Yugoslav Republic of
Facility Name
Clinical Trial Site
City
Chisinau
Country
Moldova, Republic of
Facility Name
Clinical Trial Site
City
Belgrade
Country
Serbia
Facility Name
Clinical Trial Site
City
Örebro
Country
Sweden

12. IPD Sharing Statement

Plan to Share IPD
Undecided

Learn more about this trial

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

We'll reach out to this number within 24 hrs