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

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT03303313
• Other study ID #: ALN-CC5-004
• Status: Withdrawn
• Phase: Phase 2
• 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
• 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• Status: Withdrawn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

Related Conditions & MeSH terms

• Atypical Hemolytic Uremic Syndrome
• Azotemia
• Hemolysis
• Hemolytic-Uremic Syndrome
• Syndrome

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