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

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ClinicalTrials.gov Identifier: NCT03303313
Recruitment Status : Recruiting
First Posted : October 6, 2017
Last Update Posted : May 17, 2018
Sponsor:
Information provided by (Responsible Party):
Alnylam Pharmaceuticals

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 Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
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 : September 19, 2017
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : November 2020


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




Primary Outcome Measures :
  1. The effect of Cemdisiran on platelet count [ Time Frame: Week 32 ]

Secondary Outcome Measures :
  1. The effect of Cemdisiran on hematological response as measured by platelet count [ Time Frame: after 32 weeks of treatment ]
  2. The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH) [ Time Frame: after 32 weeks of treatment ]
  3. The effect of Cemdisiran on hematological response as measured by rescue plasma therapy [ Time Frame: after 32 weeks of treatment ]
  4. The effect of Cemdisiran on LDH response as measured by LDH [ Time Frame: after 32 weeks of treatment ]
  5. The effect of Cemdisiran on LDH response as measured by rescue plasma therapy [ Time Frame: after 32 weeks of treatment ]
  6. The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count [ Time Frame: after 32 weeks of treatment ]
  7. The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH [ Time Frame: after 32 weeks of treatment ]
  8. The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels [ Time Frame: after 32 weeks of treatment ]
  9. The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy [ Time Frame: after 32 weeks of treatment ]
  10. The effect of Cemdisiran on serum creatinine levels [ Time Frame: up to 84 weeks ]
  11. The effect of Cemdisiran on estimated glomerular filtration rate (eGFR) [ Time Frame: up to 84 weeks ]
  12. The effect of Cemdisiran on adverse events (AEs) [ Time Frame: up to 108 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03303313


Contacts
Contact: Alnylam Clinical Trial Information Line 1-877-ALNYLAM clinicaltrials@alnylam.com
Contact: Alnylam Clinical Trial Information Line 1-877-256-9526

Locations
Bosnia and Herzegovina
Clinical Trial Site Recruiting
Sarajevo, Bosnia and Herzegovina
Canada
Clinical Trial Site Recruiting
Calgary, Canada, T2N 2T9
Estonia
Clinical Trial Site Recruiting
Tallinn, Estonia
Clinical Trial Site Recruiting
Tartu, Estonia
Georgia
Clinical Trial Site Recruiting
Tbilisi, Georgia
Latvia
Clinical Trial Site Recruiting
Riga, Latvia
Lithuania
Clinical Trial Site Recruiting
Kaunas, Lithuania
Clinical Trial Site Recruiting
Vilnius, Lithuania
Macedonia, The Former Yugoslav Republic of
Clinical Trial Site Recruiting
Skopje, Macedonia, The Former Yugoslav Republic of
Moldova, Republic of
Clinical Trial Site Recruiting
Chisinau, Moldova, Republic of
Serbia
Clinical Trial Site Recruiting
Belgrade, Serbia
Sweden
Clinical Trial Site Recruiting
Örebro, Sweden
Sponsors and Collaborators
Alnylam Pharmaceuticals
Investigators
Study Director: Nader Najafian, MD Alnylam Pharmaceuticals

Responsible Party: Alnylam Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03303313     History of Changes
Other Study ID Numbers: ALN-CC5-004
First Posted: October 6, 2017    Key Record Dates
Last Update Posted: May 17, 2018
Last Verified: May 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Alnylam Pharmaceuticals:
RNAi therapeutic
Atypical Hemolytic Uremic Syndrome
Hemolysis
Thrombocytopenia
Renal insufficiency
Thrombotic Microangiopathy
aHUS
TMA

Additional relevant MeSH terms:
Syndrome
Hemolysis
Azotemia
Hemolytic-Uremic Syndrome
Atypical Hemolytic Uremic Syndrome
Disease
Pathologic Processes
Uremia
Kidney Diseases
Urologic Diseases
Anemia, Hemolytic
Anemia
Hematologic Diseases
Thrombotic Microangiopathies
Thrombocytopenia
Blood Platelet Disorders