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

Sponsor

Alnylam Pharmaceuticals (Industry)

Overall Status

Withdrawn

CT.gov ID

NCT03303313

Collaborator

(none)

Enrollment

Locations

Arm

11.8

Actual Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Atypical Hemolytic Uremic Syndrome	Drug: Cemdisiran	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

0 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Sep 19, 2017

Actual Primary Completion Date :

Sep 12, 2018

Actual Study Completion Date :

Sep 12, 2018

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: Cemdisiran

Drug: Cemdisiran

Subcutaneous (sc) injection of Cemdisiran

Outcome Measures

Primary Outcome Measures

The effect of Cemdisiran on platelet count [Week 32]

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Clinical Trial Site	Sarajevo	Bosnia and Herzegovina
2	Clinical Trial Site	Calgary	Canada	T2N 2T9
3	Clinical Trial Site	Tallinn	Estonia
4	Clinical Trial Site	Tartu	Estonia
5	Clinical Trial Site	Tbilisi	Georgia
6	Clinical Trial Site	Riga	Latvia
7	Clinical Trial Site	Kaunas	Lithuania
8	Clinical Trial Site	Vilnius	Lithuania
9	Clinical Trial Site	Skopje	Macedonia, The Former Yugoslav Republic of
10	Clinical Trial Site	Chisinau	Moldova, Republic of
11	Clinical Trial Site	Belgrade	Serbia
12	Clinical Trial Site	Örebro	Sweden