NCT03303313

Brief Summary

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

Trial Health

37
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Sep 2017

Shorter than P25 for phase_2

Geographic Reach
9 countries

11 active sites

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 19, 2017

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

September 25, 2017

Completed
11 days until next milestone

First Posted

Study publicly available on registry

October 6, 2017

Completed
11 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 12, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 12, 2018

Completed
Last Updated

October 1, 2018

Status Verified

September 1, 2018

Enrollment Period

12 months

First QC Date

September 25, 2017

Last Update Submit

September 27, 2018

Conditions

Atypical Hemolytic Uremic Syndrome

Keywords

RNAi therapeuticAtypical Hemolytic Uremic SyndromeHemolysisThrombocytopeniaRenal insufficiencyThrombotic MicroangiopathyaHUSTMA

Outcome Measures

Primary Outcomes (1)

  • The effect of Cemdisiran on platelet count

    Week 32

Secondary Outcomes (12)

  • The effect of Cemdisiran on hematological response as measured by platelet count

    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

  • +7 more secondary outcomes

Study Arms (1)

Cemdisiran

EXPERIMENTAL
Drug: Cemdisiran

Interventions

CemdisiranDRUG

Subcutaneous (sc) injection of Cemdisiran

Cemdisiran

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

Clinical Trial Site

Sarajevo, Bosnia and Herzegovina

Location

Clinical Trial Site

Calgary, T2N 2T9, Canada

Location

Clinical Trial Site

Tallinn, Estonia

Location

Clinical Trial Site

Tartu, Estonia

Location

Clinical Trial Site

Tbilisi, Georgia

Location

Clinical Trial Site

Riga, Latvia

Location

Clinical Trial Site

Kaunas, Lithuania

Location

Clinical Trial Site

Vilnius, Lithuania

Location

Clinical Trial Site

Chisinau, Moldova

Location

Clinical Trial Site

Skopje, North Macedonia

Location

Clinical Trial Site

Belgrade, Serbia

Location

Clinical Trial Site

Örebro, Sweden

Location

MeSH Terms

Conditions

Atypical Hemolytic Uremic SyndromeHemolysisThrombocytopeniaRenal InsufficiencyThrombotic Microangiopathies

Condition Hierarchy (Ancestors)

Hemolytic-Uremic SyndromeUremiaKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesBlood Platelet DisordersCytopeniaPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Nader Najafian, MD

    Alnylam Pharmaceuticals

    STUDY DIRECTOR
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 25, 2017

First Posted

October 6, 2017

Study Start

September 19, 2017

Primary Completion

September 12, 2018

Study Completion

September 12, 2018

Last Updated

October 1, 2018

Record last verified: 2018-09

Locations

BO(1)NO(1)SE(1)CA(1)LI(2)MO(1)LA(1)ES(2)GE(1)