A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers

NCT ID: NCT01299298

Last Updated: 2016-08-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-01-31
• Study Completion Date: 2011-04-30

Brief 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. 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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

mipomersen

50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose

Group Type: EXPERIMENTAL

mipomersen

Intervention Type: DRUG

50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) subcutaneous (SC) single dose of study drug

placebo

50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) SC single dose

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

50 mg (cohort A), 100mg (cohort B), or 200mg (cohort C) subcutaneous (SC) single dose of study drug

Interventions

mipomersen

50 mg (cohort A), 100mg (cohort B) or 200mg (cohort C) subcutaneous (SC) single dose of study drug

Intervention Type: DRUG

placebo

50 mg (cohort A), 100mg (cohort B), or 200mg (cohort C) subcutaneous (SC) single dose of study drug

Intervention Type: DRUG

Other Intervention Names

ISIS 301012

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

• 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
• 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

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Ionis Pharmaceuticals, Inc.

INDUSTRY

Sponsor Role: collaborator

Kastle Therapeutics, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Medical Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

FOCUS Clinical Drug Development GmbH

Stresemannallee 6, Neuss, , Germany

Countries

Germany

Other Identifiers

2010-021948-18

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

MIPO3700710

Identifier Type: -

Identifier Source: org_study_id