CClinicalTrials.gg
Sign In
WithdrawnNCT03303313Updated Oct 1, 2018

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

A Phase 2 interventional study of Cemdisiran in Atypical Hemolytic Uremic Syndrome, sponsored by Alnylam Pharmaceuticals. Withdrawn at 12 sites in 10 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-10-01.

Sponsored by Alnylam Pharmaceuticals · Phase 2, Interventional, and Treatment

Why this study was withdrawn
Study terminated early due to lack of enrollment.
Phase
Phase 2
Study type
Interventional
Enrollment
0
Allocation
Not applicable
Ages
18 Years and older
Sex
All
01

Study summary

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

02

Conditions studied

  • Atypical Hemolytic Uremic Syndrome

Keywords

  • RNAi therapeutic
  • Atypical Hemolytic Uremic Syndrome
  • Hemolysis
  • Thrombocytopenia
  • Renal insufficiency
  • Thrombotic Microangiopathy
  • aHUS
  • TMA
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
0 participants (actual)

Study arms

  • Experimental
    Cemdisiran

    Drug: Cemdisiran

Interventions

  • DrugCemdisiran

    Subcutaneous (sc) injection of Cemdisiran

05

What researchers measure

Primary outcomes

  1. The effect of Cemdisiran on platelet count

    Time frame: Week 32

Secondary outcomes

  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

06

Study locations

12 sites
  • Clinical Trial Site
    Sarajevo, Bosnia and Herzegovina
  • Clinical Trial Site
    Calgary, T2N 2T9, Canada
  • Clinical Trial Site
    Tallinn, Estonia
  • Clinical Trial Site
    Tartu, Estonia
  • Clinical Trial Site
    Tbilisi, Georgia
  • Clinical Trial Site
    Riga, Latvia
  • Clinical Trial Site
    Kaunas, Lithuania
  • Clinical Trial Site
    Vilnius, Lithuania
  • Clinical Trial Site
    Skopje, Macedonia, The Former Yugoslav Republic of
  • Clinical Trial Site
    Chisinau, Moldova, Republic of
  • Clinical Trial Site
    Belgrade, Serbia
  • Clinical Trial Site
    Örebro, Sweden
07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03303313
Lead sponsor
Alnylam Pharmaceuticals
Responsible party
Sponsor
First posted
Oct 6, 2017
Start date
Sep 19, 2017
Primary completion
Sep 12, 2018
Completion
Sep 12, 2018
Last update
Oct 1, 2018

Study contacts

Nader Najafian, MD
study director · Alnylam Pharmaceuticals

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is withdrawn, as verified in Sep 2018. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Azotemia→

Other studies from this sponsor

Alnylam Pharmaceuticals

Start the discussion