Atypical Hemolytic Uremic SyndromePhase 2NCT03303313
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
A Phase 2 study testing Cemdisiran in people with Atypical Hemolytic Uremic Syndrome, run by Alnylam Pharmaceuticals at 12 sites in 10 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Atypical Hemolytic Uremic Syndrome
- What is being tested?
- Cemdisiran
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 12 sitesin 10 countries
- Study dates
- 19 Sep 2017to 12 Sep 2018
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing to provide written informed consent and to comply with the study requirements
- Age 18 years or older
- Clinical diagnosis of primary aHUS
- Clinical thrombotic microangiopathy (TMA) activity
- 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
- Previously vaccinated with meningococcal group ACWY conjugate vaccine and meningococcal group B vaccine or willingness to receive these vaccinations
- ADAMTS13 >10% or other proven aHUS-associated mutation
Who cannot join
- Clinically significant abnormal laboratory results
- Positive Shiga toxin producing Escherichia coli test at Screening
- Suspected secondary aHUS, in the opinion of the Investigator (unless there is a documented aHUS-associated genetic mutation)
- Positive direct Coombs test
- Patients who have received hemodialysis for >3 months
- Bone marrow transplant recipients
Show all 11Show fewer
- Organ transplant recipients, except for kidney transplant recipients with primary aHUS (confirmed by known genetic mutation and kidney biopsy)
- Known history or evidence of systemic lupus erythematosus or antiphospholipid antibody syndrome
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc
- 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
- Patients with a poor prognosis that is expected to limit their life expectancy to less than 3 months, in the opinion of the Investigator
Trial locations and contacts
12 sites in 10 countries.
12 sites in 10 countries
About this study
Official title: A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Being tested
- Cemdisiran
- Keywords
- RNAi therapeutic, Atypical Hemolytic Uremic Syndrome, Hemolysis, Thrombocytopenia, Renal insufficiency, Thrombotic Microangiopathy, aHUS, TMA
- Medical subject headings
- Atypical Hemolytic Uremic Syndrome, Hemolysis, Thrombocytopenia, Renal Insufficiency, Thrombotic Microangiopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CemdisiranDrug
Subcutaneous (sc) injection of Cemdisiran
| Study group | Role | What they receive |
|---|---|---|
| Cemdisiran | Experimental | Not described Receives: Drug: Cemdisiran |
What is being measured
The primary outcome is the main question the study is designed to answer.
The effect of Cemdisiran on platelet count
Measured: Week 32
Secondary outcomes (12)
The effect of Cemdisiran on hematological response as measured by platelet count
Measured: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
Measured: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
Measured: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by LDH
Measured: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
Measured: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
Measured: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
Measured: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
Measured: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
Measured: after 32 weeks of treatment
The effect of Cemdisiran on serum creatinine levels
Measured: up to 84 weeks
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
Measured: up to 84 weeks
The effect of Cemdisiran on adverse events (AEs)
Measured: up to 108 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2017
- Study start19 Sep 2017
- Primary completion12 Sep 2018
- Study completion12 Sep 2018
Why it stopped: Study terminated early due to lack of enrollment.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Atypical Hemolytic Uremic SyndromeStudies of the same condition, open and under way.
- More trials run by Alnylam PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Nader Najafian, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03303313
- Sponsor's protocol ID
- ALN-CC5-004
- Status last verified
- Sep 2018
- First submitted
- 25 Sep 2017
- Last updated
- 1 Oct 2018
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 1 Oct 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic Syndrome
- ClinicalTrials.gov ID
- NCT03303313
- Acronym / sponsor ID
- ALN-CC5-004
- Conditions
- Atypical Hemolytic Uremic Syndrome
- Keywords
- RNAi therapeutic, Atypical Hemolytic Uremic Syndrome, Hemolysis, Thrombocytopenia, Renal insufficiency, Thrombotic Microangiopathy, aHUS, TMA
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Study terminated early due to lack of enrollment.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Sep 2017 (actual)
- Primary completion
- 12 Sep 2018 (actual)
- Study completion
- 12 Sep 2018 (actual)
- First posted
- 6 Oct 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Cemdisiran | Experimental | Drug: Cemdisiran |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Willing to provide written informed consent and to comply with the study requirements
- Age 18 years or older
- Clinical diagnosis of primary aHUS
- Clinical thrombotic microangiopathy (TMA) activity
- 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
Key exclusion criteria (11 listed)
- Clinically significant abnormal laboratory results
- Positive Shiga toxin producing Escherichia coli test at Screening
- Suspected secondary aHUS, in the opinion of the Investigator (unless there is a documented aHUS-associated genetic mutation)
- Positive direct Coombs test
- Patients who have received hemodialysis for >3 months
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CemdisiranSubcutaneous (sc) injection of Cemdisiran | Drug | Cemdisiran |
Primary outcome
The effect of Cemdisiran on platelet count
Time frame: Week 32
12 secondary outcomes
The effect of Cemdisiran on hematological response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by lactate dehydrogenase (LDH)
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on hematological response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on LDH response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by platelet count
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by LDH
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by serum creatinine levels
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on complete Thrombotic microangiopathy (TMA) response as measured by rescue plasma therapy
Time frame: after 32 weeks of treatment
The effect of Cemdisiran on serum creatinine levels
Time frame: up to 84 weeks
The effect of Cemdisiran on estimated glomerular filtration rate (eGFR)
Time frame: up to 84 weeks
The effect of Cemdisiran on adverse events (AEs)
Time frame: up to 108 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Oct 2018.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nader Najafian, MD study director
Alnylam Pharmaceuticals
12 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Cemdisiran in people with Atypical Hemolytic Uremic Syndrome, run by Alnylam Pharmaceuticals at 12 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 1 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Estonia, Lithuania, Bosnia and Herzegovina, Canada, Georgia and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The effect of Cemdisiran on platelet count (time frame: Week 32). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Oct 2018.