Atypical Hemolytic Uremic Syndrome Clinical Trial
Official title:
A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
Verified date | September 2018 |
Source | Alnylam Pharmaceuticals |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
Status | Withdrawn |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 |
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 |
Lead Sponsor | Collaborator |
---|---|
Alnylam Pharmaceuticals |
Bosnia and Herzegovina, Canada, Estonia, Georgia, Latvia, Lithuania, Macedonia, The Former Yugoslav Republic of, Moldova, Republic of, Serbia, Sweden,
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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