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Clinical Trial Details — Status: Withdrawn

Administrative data

NCT number NCT03303313
Other study ID # ALN-CC5-004
Secondary ID
Status Withdrawn
Phase Phase 2
First received
Last updated
Start date September 19, 2017
Est. completion date September 12, 2018

Study information

Verified date September 2018
Source Alnylam Pharmaceuticals
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial Summary

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


Recruitment information / eligibility

Status Withdrawn
Enrollment 0
Est. completion date September 12, 2018
Est. primary completion date September 12, 2018
Accepts healthy volunteers No
Gender All
Age group 18 Years and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Cemdisiran
Subcutaneous (sc) injection of Cemdisiran

Locations

Country Name City State
Bosnia and Herzegovina Clinical Trial Site Sarajevo
Canada Clinical Trial Site Calgary
Estonia Clinical Trial Site Tallinn
Estonia Clinical Trial Site Tartu
Georgia Clinical Trial Site Tbilisi
Latvia Clinical Trial Site Riga
Lithuania Clinical Trial Site Kaunas
Lithuania Clinical Trial Site Vilnius
Macedonia, The Former Yugoslav Republic of Clinical Trial Site Skopje
Moldova, Republic of Clinical Trial Site Chisinau
Serbia Clinical Trial Site Belgrade
Sweden Clinical Trial Site Örebro

Sponsors (1)

Lead Sponsor Collaborator
Alnylam Pharmaceuticals

Countries where clinical trial is conducted

Bosnia and Herzegovina,  Canada,  Estonia,  Georgia,  Latvia,  Lithuania,  Macedonia, The Former Yugoslav Republic of,  Moldova, Republic of,  Serbia,  Sweden, 

Outcome

Type Measure Description Time frame Safety issue
Primary The effect of Cemdisiran on platelet count Week 32
Secondary The effect of Cemdisiran on hematological response as measured by platelet count after 32 weeks of treatment
Secondary The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH) after 32 weeks of treatment
Secondary The effect of Cemdisiran on hematological response as measured by rescue plasma therapy after 32 weeks of treatment
Secondary The effect of Cemdisiran on LDH response as measured by LDH after 32 weeks of treatment
Secondary The effect of Cemdisiran on LDH response as measured by rescue plasma therapy after 32 weeks of treatment
Secondary The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count after 32 weeks of treatment
Secondary The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH after 32 weeks of treatment
Secondary The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels after 32 weeks of treatment
Secondary The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy after 32 weeks of treatment
Secondary The effect of Cemdisiran on serum creatinine levels up to 84 weeks
Secondary The effect of Cemdisiran on estimated glomerular filtration rate (eGFR) up to 84 weeks
Secondary The effect of Cemdisiran on adverse events (AEs) up to 108 weeks
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