Healthy VolunteerPhase 1NCT01299298
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers
A Phase 1 study testing mipomersen in people with Healthy Volunteer, run by Kastle Therapeutics, LLC at 1 site in Germany.
Trial summary
This Phase 1 study is being conducted to evaluate 3 increasing subcutaneous (SC) doses (50, mg, 100 mg or 200mg) of mipomersen in Japanese healthy volunteers. Eligible subjects will receive a single study injection of either mipomersen or placebo. Read more about this study
- Who is it for?
- Ages 20 to 45 years, men onlyHealthy 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 siteGermany
- Study dates
- Jan 2011to Apr 2011
- 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 20 to 45 years
- Men only
- Healthy volunteers accepted
- Adult
Who may be able to join
- First generation Japanese (born in Japan of Japanese parents and Japanese grandparents), lived no more than 5 years outside of Japan, with no significant change in lifestyle or habits, including diet, while living outside of Japan.
- Surgically sterile, abstinent or subject or partner compliant with acceptable contraceptive during and 24 weeks after the last study drug dose
- Body weight >50 kg and body mass index between 18 and 30 kg/m2 inclusive
Who cannot join
- Clinically significant cardiovascular, pulmonary, hepatic, renal, haematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, infectious, or psychiatric disease
- Clinically significant abnormal findings on the physical examination, ECG, blood pressure, heart rate, medical history, or clinical laboratory results at Screening or before dosing
- Positive test for human immunodeficiency virus (HIV), hepatitis B or C.
- High sensitivity C-reactive protein (hsCRP) >5 mg/L
- History of or current malignancy (with the exception of basal or squamous cell carcinoma of the skin if adequately treated and no recurrence for > 1 year)
- Evidence of acute or ongoing chronic inflammatory condition or infection
Show all 14Show fewer
- History of rash, impetigo, or drug allergies
- Alcohol and/or drug abuse
- Smoking more than 10 cigarettes per day
- Planned dental work up to and including Day 8 procedures
- Treatment with another investigational drug, biological agent, or device within 4 weeks of Screening or 5 half-lives of the study agent, whichever is longer
- Use of prescribed medications within 4 weeks or over-the counter medications (including dietary supplements and herbal remedies) within 14 days before the first study drug dose, or use of any concomitant medications (prescribed or over the counter) through Day 8 of the study without Investigator and Sponsor approval. Vaccinations are not allowed beginning 3 weeks prior to the first dose of study drug until completion of the safety follow-up period
- Previous exposure to oligonucleotide-based drug therapy
- Donated 50 to 499 mL of blood within 30 days prior to consent, or >499 mL within 60 days
Trial locations and contacts
1 site in 1 country.
Germany1 site
FOCUS Clinical Drug Development GmbH
Stresemannallee 6, Neuss, 41460
About this study
Official title: A Prospective, Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen Administered Subcutaneously to Japanese Healthy Subjects
This Phase 1 study is being conducted to evaluate 3 increasing subcutaneous (SC) doses (50, mg, 100 mg or 200mg) of mipomersen in Japanese healthy volunteers. Eligible subjects will receive a single study injection of either mipomersen or placebo. Subjects will be enrolled into 1 of 3 treatment cohorts (Cohorts A, B, and C) in a dose-escalation design. Dose-escalation will proceed only if there is an acceptable safety profile from the previous dosing level.
- Conditions
- Healthy Volunteer
- Being tested
- mipomersen, placebo
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
mipomersenDrug
Also called ISIS 301012
50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) subcutaneous (SC) single dose of study drug
placeboDrug
50 mg (cohort A), 100mg (cohort B), or 200mg (cohort C) subcutaneous (SC) single dose of study drug
| Study group | Role | What they receive |
|---|---|---|
| mipomersen | Experimental | 50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose Receives: Drug: mipomersen |
| placebo | Placebo comparator | 50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum plasma concentration (Cmax)
plasma PK parameters
Measured: Baseline up to Day 36 Post-Treatment
Time to maximal concentration (Tmax)
plasma PK parameters
Measured: Baseline up to Day 36 Post-Treatment
Area Under the Curve (AUC)
plasma PK parameters
Measured: Baseline up to Day 36 Post-Treatment
Secondary outcomes (1)
Number of Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Measured: Up to Day 36 Post-Treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2011 (estimated)
- Study startJan 2011
- Primary completionApr 2011
- Study completionApr 2011
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- 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
- NCT01299298
- Sponsor's protocol ID
- MIPO3700710
- Other IDs
- 2010-021948-18
- Status last verified
- Aug 2016
- First submitted
- 9 Feb 2011
- Last updated
- 3 Aug 2016
- Results posted
- No
- Data monitoring committee
- Yes
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 Prospective, Randomized, Double-Blind, Placebo-Controlled, Dose-Escalation, Phase 1 Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen Administered Subcutaneously to Japanese Healthy Subjects
- ClinicalTrials.gov ID
- NCT01299298
- Acronym / sponsor ID
- MIPO3700710
- Conditions
- Healthy Volunteer
- 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
- Jan 2011
- Primary completion
- Apr 2011 (actual)
- Study completion
- Apr 2011 (actual)
- First posted
- 18 Feb 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.
This Phase 1 study is being conducted to evaluate 3 increasing subcutaneous (SC) doses (50, mg, 100 mg or 200mg) of mipomersen in Japanese healthy volunteers. Eligible subjects will receive a single study injection of either mipomersen or placebo. Subjects will be enrolled into 1 of 3 treatment cohorts (Cohorts A, B, and C) in a dose-escalation design. Dose-escalation will proceed only if there is an acceptable safety profile from the previous dosing level.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| mipomersen | Experimental | Drug: mipomersen50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose |
| placebo | Placebo comparator | Drug: placebo50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose |
Who may qualify
- Ages
- 20 to 45 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- First generation Japanese (born in Japan of Japanese parents and Japanese grandparents), lived no more than 5 years outside of Japan, with no significant change in lifestyle or habits, including diet, while living outside of Japan.
- Surgically sterile, abstinent or subject or partner compliant with acceptable contraceptive during and 24 weeks after the last study drug dose
- Body weight >50 kg and body mass index between 18 and 30 kg/m2 inclusive
Key exclusion criteria (14 listed)
- Clinically significant cardiovascular, pulmonary, hepatic, renal, haematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, infectious, or psychiatric disease
- Clinically significant abnormal findings on the physical examination, ECG, blood pressure, heart rate, medical history, or clinical laboratory results at Screening or before dosing
- Positive test for human immunodeficiency virus (HIV), hepatitis B or C.
- High sensitivity C-reactive protein (hsCRP) >5 mg/L
- History of or current malignancy (with the exception of basal or squamous cell carcinoma of the skin if adequately treated and no recurrence for > 1 year)
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 |
|---|---|---|
| mipomersenAlso: ISIS 30101250 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) subcutaneous (SC) single dose of study drug | Drug | mipomersen |
| placebo50 mg (cohort A), 100mg (cohort B), or 200mg (cohort C) subcutaneous (SC) single dose of study drug | Drug | placebo |
Primary outcome
Maximum plasma concentration (Cmax)
Time frame: Baseline up to Day 36 Post-Treatment
plasma PK parameters
Time to maximal concentration (Tmax)
Time frame: Baseline up to Day 36 Post-Treatment
plasma PK parameters
Area Under the Curve (AUC)
Time frame: Baseline up to Day 36 Post-Treatment
plasma PK parameters
1 secondary outcome
Number of Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: Up to Day 36 Post-Treatment
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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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 Germany.
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 14 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 Germany. 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?
Maximum plasma concentration (Cmax) (time frame: Baseline up to Day 36 Post-Treatment). The study lists 3 primary outcomes in all. 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.