Elevated LDL-Cholesterol (LDL-C)Phase 1NCT01437059
Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
A Phase 1 study testing ALN-PCS02 and Sterile Normal Saline (0.9% NaCl) in people with Elevated LDL-Cholesterol (LDL-C), run by Alnylam Pharmaceuticals at 2 sites in United Kingdom.
Trial summary
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C). Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Elevated LDL-Cholesterol (LDL-C)
- What is being tested?
- ALN-PCS02 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
- 32 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- Sep 2011to Sep 2012
- 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 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Elevated LDL-C of >3.0 mmol/L and <5.7 mmol/L
- Fasting triglyceride concentration ≤2.8 mmol/L
- Body weight >60.0 kg; body mass index (BMI) between 19.00 kg/m2 and <35.00 kg/m2
- Adequate blood counts, liver and renal function
- May not received any lipid lowering drug/agent within the 30 days prior to the screening
- Non-smokers for at least 3 months
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- Women of child-bearing potential must have a negative pregnancy test, cannot be breast feeding, and must use an adequate method of birth control
- Males agree to use appropriate contraception
- Willing and able to comply with protocol-required visit schedule and visit requirements and provide written informed consent
Who cannot join
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
- Received prescription drugs within 4 weeks of first dosing
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- Subjects who have donated more than 500 mL of blood within the 3 months prior to ALN-PCS02 or placebo administration;
- Received megadose vitamin therapy or dietary supplements within 4 weeks prior to screening
- Subjects who have used prescription drugs within 4 weeks of first dosing
- Considered unfit for the study by the Principal Investigator
- Employee or family member of the sponsor or the clinical study site personnel
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Clinical Site
Leeds, LS2 9LH
Clinical Site
London, SE1 1YR
About this study
Official title: A Phase 1, Randomized, Single-blind, Placebo-Controlled, Single Ascending Dose, Safety, Tolerability and Pharmacokinetics Study of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (LDL-C).
- Conditions
- Elevated LDL-Cholesterol (LDL-C)
- Being tested
- ALN-PCS02, Sterile Normal Saline (0.9% NaCl)
- Medical subject headings
- Hyperlipoproteinemia Type II
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ALN-PCS02Drug
Dose levels between 15 and 400 μg/kg by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)Drug
Calculated volume to match active comparator
| Study group | Role | What they receive |
|---|---|---|
| ALN-PCS02 | Active comparator | Not described Receives: Drug: ALN-PCS02 |
| 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 proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Measured: Up to 28 days
Secondary outcomes (3)
Pharmacokinetics (PK) of ALN-PCS02 (Cmax, tmax, t1/2, AUC0-last, CL).
Measured: Up to 180 days
Effect of ALN-PCS02 on Circulating PCSK9 Levels (Determination of % Lowering of PCSK9 to pretreatment/Baseline PCSK9 Level).
Measured: Up to 28 days
Effect of ALN-PCS02 on Circulating LDL-c Levels (Determination of % Lowering of LDL-c to pretreatment/Baseline LDL-c Level).
Measured: Up to 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Sep 2011 (estimated)
- Study startSep 2011
- Primary completionMar 2012
- Study completionSep 2012
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 Elevated LDL-Cholesterol (LDL-C)Studies 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
- 32 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alnylam Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Amy Simon, MD, Study director, Alnylam Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01437059
- Sponsor's protocol ID
- ALN-PCS02-001
- Status last verified
- Oct 2012
- First submitted
- 19 Sep 2011
- Last updated
- 12 Oct 2012
- Results posted
- No
- Data monitoring committee
- No
Publications
Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, Nochur SV, Kotelianski V, Horton J, Mant T, Chiesa J, Ritter J, Munisamy M, Vaishnaw AK, Gollob JA, Simon A. Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial. Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3.
PubMed 24094767
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2012. 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, Safety, Tolerability and Pharmacokinetics Study of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)
- ClinicalTrials.gov ID
- NCT01437059
- Acronym / sponsor ID
- ALN-PCS02-001
- Conditions
- Elevated LDL-Cholesterol (LDL-C)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 32 participants (actual)
- Lead sponsor
- Alnylam PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2011
- Primary completion
- Mar 2012 (actual)
- Study completion
- Sep 2012 (actual)
- First posted
- 20 Sep 2011 (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 determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-PCS02 in subjects with Elevated LDL-Cholesterol (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-PCS02 | Active comparator | Drug: ALN-PCS02 |
| Sterile Normal Saline (0.9% NaCl) | Placebo comparator | Drug: Sterile Normal Saline (0.9% NaCl) |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Elevated LDL-C of >3.0 mmol/L and <5.7 mmol/L
- Fasting triglyceride concentration ≤2.8 mmol/L
- Body weight >60.0 kg; body mass index (BMI) between 19.00 kg/m2 and <35.00 kg/m2
- Adequate blood counts, liver and renal function
- May not received any lipid lowering drug/agent within the 30 days prior to the screening
Key exclusion criteria (11 listed)
- Known hepatitis B surface antigen (HBsAg), hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) infection
- Multiple drug allergies or know sensitivity to oligonucleotide
- History of drug abuse and/or alcohol abuse
- Receiving an investigational agent within 3 months prior to study drug administration
- Subjects with safety laboratory test results deemed clinical significant by the Investigator;
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-PCS02Dose levels between 15 and 400 μg/kg by intravenous (IV) infusion | Drug | ALN-PCS02 |
| Sterile Normal Saline (0.9% NaCl)Calculated volume to match active comparator | Drug | Sterile Normal Saline (0.9% NaCl) |
Primary outcome
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Time frame: Up to 28 days
3 secondary outcomes
Pharmacokinetics (PK) of ALN-PCS02 (Cmax, tmax, t1/2, AUC0-last, CL).
Time frame: Up to 180 days
Effect of ALN-PCS02 on Circulating PCSK9 Levels (Determination of % Lowering of PCSK9 to pretreatment/Baseline PCSK9 Level).
Time frame: Up to 28 days
Effect of ALN-PCS02 on Circulating LDL-c Levels (Determination of % Lowering of LDL-c to pretreatment/Baseline LDL-c Level).
Time frame: Up to 28 days
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 12 Oct 2012.
- Derived from this study: PubMed 24094767Fitzgerald K, Frank-Kamenetsky M, Shulga-Morskaya S, Liebow A, Bettencourt BR, Sutherland JE, Hutabarat RM, Clausen VA, Karsten V, Cehelsky J, Nochur SV, Kotelianski V, Horton J, Mant T, Chiesa J, Ritter J, Munisamy M, Vaishnaw AK, Gollob JA, Simon A. Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial. Lancet. 2014 Jan 4;383(9911):60-68. doi: 10.1016/S0140-6736(13)61914-5. Epub 2013 Oct 3.
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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.
Amy Simon, 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-PCS02 and Sterile Normal Saline (0.9% NaCl) in people with Elevated LDL-Cholesterol (LDL-C), 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 9 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: finished, as of the sponsor's last update on 12 Oct 2012. 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 proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation. (time frame: Up to 28 days). 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 NCT01437059, 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 12 Oct 2012.