HypercholesterolemiaPhase 1NCT00927459
Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Pharmacodynamics (PD) of Liposomal siRNA in Subjects With High Cholesterol
A Phase 1 study testing PRO-040201 in people with Hypercholesterolemia, run by Arbutus Biopharma Corporation at 1 site in United States.
Trial summary
This study is a Phase 1, single-center, placebo-controlled, single-blind, first-in-human, single-ascending dose study in male and female subjects with high cholesterol. A maximum of 32 subjects is planned for enrollment in this study. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Hypercholesterolemia
- What is being tested?
- PRO-040201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2009to Jan 2010
- 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
- Fasting, stable LDL-C ≥ 160 mg/dL
- Fasting, stable triglyceride < 400 mg/dL
- BMI between 22 and 35 kg/m2, inclusive
- Females must be of non-child bearing potential
- Males of reproductive potential must agree to practice effective contraception throughout the study and for 3 months following infusion
Who cannot join
- Clinically significant endocrine, hematologic, renal, hepatic, pulmonary, uncontrolled psychiatric, or neurologic disease
- Cancer within 5 years prior to screening
- History of congestive heart failure or chronic heart failure
- Uncontrolled cardiac arrhythmias
- History of coronary heart disease
- Clinically significant abnormal baseline ECG
Show all 17Show fewer
- History of additional risk factors for torsades de pointes
- Hepatitis B, C, or HIV positive
- Current diagnosis or known history of liver disease
- A marked baseline prolongation of QT/QTc interval
- Known history of fibromyalgia, myopathy, myositis, rhabdomyolysis, any unexplained muscle pain, or a creatine phosphokinase (CPK) >3 x upper limit of normal (ULN) at screening
- Alanine aminotransferase, AST, GGT, or total bilirubin >2 x ULN at screening
- Serum creatinine > 1.5 mg/dL
- Systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥ 100 mmHg on 2 occasions during screening
- Concomitant use of medications that prolongs the QT/QTc interval
- Treatment with lipid lowering therapy within 30 days prior to screening
- Use of investigational drug within 3 months prior to screening
Trial locations and contacts
1 site in 1 country.
United States1 site
Medpace Clinical Pharmacology Unit
Cincinnati, Ohio, 45212
About this study
Official title: A Placebo-Controlled, Single-Blind, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PRO-040201 in Male and Female Subjects With Hypercholesterolemia
This study is a Phase 1, single-center, placebo-controlled, single-blind, first-in-human, single-ascending dose study in male and female subjects with high cholesterol. A maximum of 32 subjects is planned for enrollment in this study.
- Conditions
- Hypercholesterolemia
- Being tested
- PRO-040201, Placebo
- Keywords
- cholesterol, coronary artery disease
- Medical subject headings
- Hypercholesterolemia, Coronary Artery Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PRO-040201Drug
Single dose IV infusion
PlaceboDrug
placebo
| Study group | Role | What they receive |
|---|---|---|
| PRO-040201 | Experimental | PRO-040201 with placebo control in each cohort Receives: Drug: PRO-040201 |
| Placebo | Placebo comparator | PRO-040201 with placebo control in each cohort Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and tolerability of PRO-040201
Measured: 29 days
Secondary outcomes (2)
Pharmacokinetics of PRO-040201 in Humans
Measured: 48 hours
Pharmacodynamics of PRO-040201 in Humans
Measured: 29 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jun 2009 (estimated)
- Study startJun 2009
- Primary completionDec 2009
- Study completionJan 2010
Why it stopped: Potential for immune stimulation to interfere with further dose escalation.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HypercholesterolemiaStudies of the same condition, open and under way.
- 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
- Single group
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Arbutus Biopharma Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Douglas Logan, MD, Principal investigator, Medpace Clinical Pharmacology Unit
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00927459
- Sponsor's protocol ID
- TKM-ApoB-001
- Status last verified
- Jan 2010
- First submitted
- 23 Jun 2009
- Last updated
- 22 Jan 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 22 Jan 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo-Controlled, Single-Blind, Single-Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PRO-040201 in Male and Female Subjects With Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT00927459
- Acronym / sponsor ID
- TKM-ApoB-001
- Conditions
- Hypercholesterolemia
- Keywords
- cholesterol, coronary artery disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Potential for immune stimulation to interfere with further dose escalation.
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Arbutus Biopharma CorporationIndustry (pharma, biotech, devices)
- Study start
- Jun 2009
- Primary completion
- Dec 2009 (actual)
- Study completion
- Jan 2010 (actual)
- First posted
- 25 Jun 2009 (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.
This study is a Phase 1, single-center, placebo-controlled, single-blind, first-in-human, single-ascending dose study in male and female subjects with high cholesterol. A maximum of 32 subjects is planned for enrollment in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| PRO-040201 | Experimental | Drug: PRO-040201PRO-040201 with placebo control in each cohort |
| Placebo | Placebo comparator | Drug: PlaceboPRO-040201 with placebo control in each cohort |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Fasting, stable LDL-C ≥ 160 mg/dL
- Fasting, stable triglyceride < 400 mg/dL
- BMI between 22 and 35 kg/m2, inclusive
- Females must be of non-child bearing potential
- Males of reproductive potential must agree to practice effective contraception throughout the study and for 3 months following infusion
Key exclusion criteria (17 listed)
- Clinically significant endocrine, hematologic, renal, hepatic, pulmonary, uncontrolled psychiatric, or neurologic disease
- Cancer within 5 years prior to screening
- History of congestive heart failure or chronic heart failure
- Uncontrolled cardiac arrhythmias
- History of coronary heart disease
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 |
|---|---|---|
| PRO-040201Single dose IV infusion | Drug | PRO-040201 |
| Placeboplacebo | Drug | Placebo |
Primary outcome
Safety and tolerability of PRO-040201
Time frame: 29 days
2 secondary outcomes
Pharmacokinetics of PRO-040201 in Humans
Time frame: 48 hours
Pharmacodynamics of PRO-040201 in Humans
Time frame: 29 days
Results and safety
The study was stopped early (reason given: Potential for immune stimulation to interfere with further dose escalation.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jan 2010.
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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.
Douglas Logan, MD principal investigator
Medpace Clinical Pharmacology Unit
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PRO-040201 in people with Hypercholesterolemia, run by Arbutus Biopharma Corporation at 1 site in United States.
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 5 inclusion and 17 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: stopped early, as of the sponsor's last update on 22 Jan 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 was stopped early (reason given: Potential for immune stimulation to interfere with further dose escalation.). Results are not available in the source record.
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?
Safety and tolerability of PRO-040201 (time frame: 29 days). 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 22 Jan 2010.