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

Kastle Therapeutics

Status and phase

Completed

Phase 1

Conditions

Healthy Volunteer

Treatments

Drug: mipomersen

Drug: placebo

Study type

Interventional

Funder types

Industry

Identifiers

NCT01299298

2010-021948-18 (EudraCT Number)

MIPO3700710

Details and patient eligibility

About

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.

Enrollment

20 patients

Sex

Male

Ages

20 to 45 years old

Volunteers

Accepts Healthy Volunteers

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