HypercholesterolemiaPhase 1NCT02314442
A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)
A Phase 1 study testing ALN-PCSSC and Sterile Normal Saline (0.9% NaCl) in people with Hypercholesterolemia, run by Alnylam Pharmaceuticals at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-PCSSC in subjects with elevated LDL-C. Read more about this study
- Who is it for?
- Ages 18 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypercholesterolemia
- What is being tested?
- ALN-PCSSC and Sterile Normal Saline (0.9% NaCl)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Dec 2014to Nov 2015
- Phase
- Phase 1
- 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 to 60 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Adequate complete blood counts, liver and renal function
- Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
- Male subjects agree to use appropriate contraception
- Willing to provide written informed consent and willing to comply with study requirements.
- Non-smokers and non-nicotine users for at least 90 days before screening
- On stable statin co-medication [for designated multiple dose cohorts only]
Who cannot join
- Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
- Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
- History of multiple drug allergies or intolerance to subcutaneous injection
- Received any medication or nutraceutical to alter serum lipids within 30 days before screening (non-statin cohorts only)
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Covance Clinical Research Unit
Leeds
Richmond Pharmacology
London
About this study
Official title: A Phase 1, Randomized, Single Blind, Placebo Controlled, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN PCSSC in Subjects With Elevated Low Density Lipoprotein Cholesterol
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-PCSSC in subjects with elevated LDL-C.
- Conditions
- Hypercholesterolemia
- Being tested
- ALN-PCSSC, Sterile Normal Saline (0.9% NaCl)
- Keywords
- LDL-C, RNAi therapeutic
- Medical subject headings
- Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-PCSSCDrug
Single or multiple doses of ALN-PCSSC by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)Drug
calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| ALN-PCSSC | Active comparator | Not described Receives: Drug: ALN-PCSSC |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Not described Receives: Drug: Sterile Normal Saline (0.9% NaCl) |
What is being measured
The primary outcome is the main question the study is designed to answer.
The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
Measured: Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose
Secondary outcomes (3)
The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2)
Measured: Part 1 (SAD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 42, 56 days post-dose; Part 2 (MD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 29, 35, 42, 56, 84 days post-dose
The effect of ALN-PCSSC on serum levels of LDL-C
Measured: Through the sooner of final follow up visit or 180 days post final dose
The effect of ALN-PCSSC on plasma levels of PCSK9
Measured: Through the sooner of final follow up visit or 180 days post final dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2014 (estimated)
- Study startDec 2014
- Primary completionMay 2015
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for HypercholesterolemiaStudies 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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- The Medicines Company
- Responsible party
- Sponsor
- Investigators
- Robert Kauffman, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02314442
- Sponsor's protocol ID
- ALN-PCSSC-001
- Status last verified
- Dec 2015
- First submitted
- 5 Dec 2014
- Last updated
- 17 Dec 2015
- Results posted
- No
- Data monitoring committee
- No
Publications
Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. A Highly Durable RNAi Therapeutic Inhibitor of PCSK9. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13.
PubMed 27959715
Study at a glance
From the registry record, last updated by the sponsor on 17 Dec 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Single Blind, Placebo Controlled, Single Ascending Dose and Multiple Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN PCSSC in Subjects With Elevated Low Density Lipoprotein Cholesterol
- ClinicalTrials.gov ID
- NCT02314442
- Acronym / sponsor ID
- ALN-PCSSC-001
- Conditions
- Hypercholesterolemia
- Keywords
- LDL-C, RNAi therapeutic
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- The Medicines Company
- Study start
- Dec 2014
- Primary completion
- May 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 11 Dec 2014 (estimated)
- 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, pharmacokinetics and pharmacodynamics of ALN-PCSSC in subjects with elevated LDL-C.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ALN-PCSSC | Active comparator | Drug: ALN-PCSSC |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Adequate complete blood counts, liver and renal function
- Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization
- Male subjects agree to use appropriate contraception
- Willing to provide written informed consent and willing to comply with study requirements.
- Non-smokers and non-nicotine users for at least 90 days before screening
Key exclusion criteria (4 listed)
- Any uncontrolled or serious disease, or any medical or surgical condition, that may interfere with participation in the clinical study and/or put the subject at significant risk
- Received an investigational agent within 90 days before the first dose of study drug or are in follow-up of another clinical study
- History of multiple drug allergies or intolerance to subcutaneous injection
- Received any medication or nutraceutical to alter serum lipids within 30 days before screening (non-statin cohorts only)
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 |
|---|---|---|
| ALN-PCSSCSingle or multiple doses of ALN-PCSSC by subcutaneous (sc) injection | Drug | ALN-PCSSC |
| Sterile Normal Saline (0.9% NaCl)calculated volume to match active comparator | Drug | Sterile Normal Saline (0.9% NaCl) |
Primary outcome
The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
Time frame: Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose
3 secondary outcomes
The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2)
Time frame: Part 1 (SAD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 42, 56 days post-dose; Part 2 (MD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 29, 35, 42, 56, 84 days post-dose
The effect of ALN-PCSSC on serum levels of LDL-C
Time frame: Through the sooner of final follow up visit or 180 days post final dose
The effect of ALN-PCSSC on plasma levels of PCSK9
Time frame: Through the sooner of final follow up visit or 180 days post final dose
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Dec 2015.
- Derived from this study: PubMed 27959715Fitzgerald K, White S, Borodovsky A, Bettencourt BR, Strahs A, Clausen V, Wijngaard P, Horton JD, Taubel J, Brooks A, Fernando C, Kauffman RS, Kallend D, Vaishnaw A, Simon A. A Highly Durable RNAi Therapeutic Inhibitor of PCSK9. N Engl J Med. 2017 Jan 5;376(1):41-51. doi: 10.1056/NEJMoa1609243. Epub 2016 Nov 13.
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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.
Robert Kauffman, MD study director
Alnylam Pharmaceuticals
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing ALN-PCSSC and Sterile Normal Saline (0.9% NaCl) in people with Hypercholesterolemia, run by Alnylam Pharmaceuticals at 2 sites in United Kingdom.
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 6 inclusion and 4 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: finished, as of the sponsor's last update on 17 Dec 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. 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. Some participants in this study may receive a placebo. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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 safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation (time frame: Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02314442, which also lists 1 linked publication.
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 17 Dec 2015.