Healthy VolunteerPhase 1NCT01414881
Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects
A Phase 1 study testing mipomersen in people with Healthy Volunteer, run by Kastle Therapeutics, LLC at 1 site in United States.
Trial summary
The primary objective of this Phase I exploratory study is to determine the effects of mipomersen on the hepatic production of apolipoprotein-B (apo B) in very low density lipoprotein (VLDL) compared to baseline levels. Read more about this study
- Who is it for?
- Ages 18 to 75 yearsHealthy volunteers can take part
- Conditions the study is for
- Healthy Volunteer
- What is being tested?
- mipomersen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2011to Nov 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 75 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Non-pregnant, non-lactating, surgically sterile, postmenopausal, abstinent, or the subject or partner is compliant with an acceptable contraceptive regimen for 4 weeks prior to Screening and willing to remain compliant with the contraceptive regimen throughout treatment and for 25 weeks after the last investigational product dose
- Body weight >50 kg, body mass index (BMI) ≤38 kg/m2, and stable weight (i.e., within 5% of mean body weight) for > 8 weeks prior to Screening
- Fasting TG levels of ≤170 mg/dL, fasting serum blood glucose of ≤115 mg/dL, and an HbA1c ≤6.5%
Who cannot join
- Presence of any clinically significant abnormal laboratory profiles, physical exams, vital signs, or ECGs
- History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, infectious, or psychiatric disease
- Malignancy (with the exception of basal or squamous cell carcinoma of the skin if adequately treated and no recurrence for >1 year) at Screening
- History of relevant food and/or drug allergies (i.e., allergy to heparin or any significant food allergy that could preclude a stable diet)
- The subject is receiving prescription lipid-lowering therapies such as statins, bile acid sequestrants, niacin/nicotinic acid, and/or fibrates or over-the-counter (OTC) fish oils, flaxseed, red rice or nutrient supplements that might affect lipid levels
- The subject is unwilling to limit alcohol consumption for the entire duration of the study
- The subject smokes >5 cigarettes per day
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia-Presbyterian Medical Center, MS Care Center
New York, New York
About this study
Official title: A Phase 1 Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects
The primary objective of this Phase I exploratory study is to determine the effects of mipomersen on the hepatic production of apolipoprotein-B (apo B) in very low density lipoprotein (VLDL) compared to baseline levels. The study will consist of a Screening Period, a 1-week Run-in Period to establish a stable diet, an approximate 11-week Treatment Period with Placebo or Mipomersen, and a 25-week Post-Treatment Follow-up Period. The total duration of any given subject's participation will be approximately 40 weeks.
- Conditions
- Healthy Volunteer
- Being tested
- mipomersen, Placebo
- Keywords
- ApoB (Apolipoprotein B), LDL (low density lipoprotein), mipomersen
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mipomersenDrug
mipomersen 200mg subcutaneously (SC) once weekly
PlaceboDrug
Placebo administered subcutaneously (SC) once weekly
| Study group | Role | What they receive |
|---|---|---|
| mipomersen | Experimental | mipomersen 200mg subcutaneously (SC) once weekly Receives: Drug: mipomersen |
| Placebo | Placebo comparator | Placebo administered subcutaneously (SC) once weekly Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent change in the production rate (PR) of very low density lipoprotein (VLDL) apolipoprotein B (apo B)
Measured: through approximately 11 weeks of treatment
Secondary outcomes (7)
Fractional clearance rate (FCR) of VLDL Triglyceride (TG), VLDL apo B, intermediate density lipoprotein (IDL) apo B, and low-density lipoprotein (LDL) apo B
Measured: Through approximately 11 weeks of treatment
Production rate (PR) of VLDL-TG, IDL apo B, LDL apo B
Measured: Through approximately 11 weeks of treatment
Conversion of VLDL apo B to low-density lipoprotein (LDL) apo B
Measured: Through approximately 11 weeks of treatment
Direct removal of VLDL apo B from plasma
Measured: Through approximately 11 weeks of treatment
Post-heparin hepatic lipase and lipoprotein lipase activities in serum
Measured: Through approximately 11 weeks of treatment
Fasting plasma levels of fatty acids and beta-hydroxybutyrate
Measured: Up to 40 weeks
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Measured: Up to 40 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2011 (estimated)
- Study startSep 2011
- Primary completionJun 2012
- Study completionNov 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Healthy VolunteerStudies 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
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kastle Therapeutics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Genzyme, a Sanofi Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01414881
- Sponsor's protocol ID
- MIPO1600810
- Status last verified
- Aug 2016
- First submitted
- 10 Aug 2011
- Last updated
- 3 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects
- ClinicalTrials.gov ID
- NCT01414881
- Acronym / sponsor ID
- MIPO1600810
- Conditions
- Healthy Volunteer
- Keywords
- ApoB (Apolipoprotein B), LDL (low density lipoprotein), mipomersen
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Kastle Therapeutics, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Ionis Pharmaceuticals, Inc.
- Study start
- Sep 2011
- Primary completion
- Jun 2012 (actual)
- Study completion
- Nov 2012 (actual)
- First posted
- 11 Aug 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 primary objective of this Phase I exploratory study is to determine the effects of mipomersen on the hepatic production of apolipoprotein-B (apo B) in very low density lipoprotein (VLDL) compared to baseline levels. The study will consist of a Screening Period, a 1-week Run-in Period to establish a stable diet, an approximate 11-week Treatment Period with Placebo or Mipomersen, and a 25-week Post-Treatment Follow-up Period. The total duration of any given subject's participation will be approximately 40 weeks.
Study design
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| mipomersen | Experimental | Drug: mipomersenmipomersen 200mg subcutaneously (SC) once weekly |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo administered subcutaneously (SC) once weekly |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Non-pregnant, non-lactating, surgically sterile, postmenopausal, abstinent, or the subject or partner is compliant with an acceptable contraceptive regimen for 4 weeks prior to Screening and willing to remain compliant with the contraceptive regimen throughout treatment and for 25 weeks after the last investigational product dose
- Body weight >50 kg, body mass index (BMI) ≤38 kg/m2, and stable weight (i.e., within 5% of mean body weight) for > 8 weeks prior to Screening
- Fasting TG levels of ≤170 mg/dL, fasting serum blood glucose of ≤115 mg/dL, and an HbA1c ≤6.5%
Key exclusion criteria (7 listed)
- Presence of any clinically significant abnormal laboratory profiles, physical exams, vital signs, or ECGs
- History or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, infectious, or psychiatric disease
- Malignancy (with the exception of basal or squamous cell carcinoma of the skin if adequately treated and no recurrence for >1 year) at Screening
- History of relevant food and/or drug allergies (i.e., allergy to heparin or any significant food allergy that could preclude a stable diet)
- The subject is receiving prescription lipid-lowering therapies such as statins, bile acid sequestrants, niacin/nicotinic acid, and/or fibrates or over-the-counter (OTC) fish oils, flaxseed, red rice or nutrient supplements that might affect lipid levels
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 |
|---|---|---|
| mipomersenmipomersen 200mg subcutaneously (SC) once weekly | Drug | mipomersen |
| PlaceboPlacebo administered subcutaneously (SC) once weekly | Drug | Placebo |
Primary outcome
Percent change in the production rate (PR) of very low density lipoprotein (VLDL) apolipoprotein B (apo B)
Time frame: through approximately 11 weeks of treatment
7 secondary outcomes
Fractional clearance rate (FCR) of VLDL Triglyceride (TG), VLDL apo B, intermediate density lipoprotein (IDL) apo B, and low-density lipoprotein (LDL) apo B
Time frame: Through approximately 11 weeks of treatment
Production rate (PR) of VLDL-TG, IDL apo B, LDL apo B
Time frame: Through approximately 11 weeks of treatment
Conversion of VLDL apo B to low-density lipoprotein (LDL) apo B
Time frame: Through approximately 11 weeks of treatment
Direct removal of VLDL apo B from plasma
Time frame: Through approximately 11 weeks of treatment
Post-heparin hepatic lipase and lipoprotein lipase activities in serum
Time frame: Through approximately 11 weeks of treatment
Fasting plasma levels of fatty acids and beta-hydroxybutyrate
Time frame: Up to 40 weeks
Incidence of adverse events (AEs) and serious adverse events (SAEs)
Time frame: Up to 40 weeks
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 3 Aug 2016.
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Screening Protocol
Pain
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.
Medical Monitor study director
Genzyme, a Sanofi Company
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 mipomersen in people with Healthy Volunteer, run by Kastle Therapeutics, LLC 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 3 inclusion and 7 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 3 Aug 2016. 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 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?
Percent change in the production rate (PR) of very low density lipoprotein (VLDL) apolipoprotein B (apo B) (time frame: through approximately 11 weeks of treatment). 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 3 Aug 2016.