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

NCT01299298 | Completed Phase 1 DMC

• 2 other identifiers: MIPO37007102010-021948-18
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (3)

• Maximum plasma concentration (Cmax)

  plasma PK parameters

  Baseline up to Day 36 Post-Treatment

• Time to maximal concentration (Tmax)

  plasma PK parameters

  Baseline up to Day 36 Post-Treatment

• Area Under the Curve (AUC)

  plasma PK parameters

  Baseline up to Day 36 Post-Treatment

Secondary Outcomes (1)

• Number of Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

  Up to Day 36 Post-Treatment

Study Arms (2)

mipomersen

EXPERIMENTAL

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

Drug: mipomersen

placebo

PLACEBO COMPARATOR

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

Drug: placebo

Interventions

mipomersen DRUG

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

Also known as: ISIS 301012

mipomersen

placebo DRUG

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

placebo

Eligibility Criteria

• Age: 20 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Kastle Therapeutics, LLC: lead
• Ionis Pharmaceuticals, Inc.: collaborator

Study Sites (1)

FOCUS Clinical Drug Development GmbH

Stresemannallee 6, Neuss, 41460, Germany

Location

MeSH Terms

Interventions

mipomersen

Study Officials

• Medical Monitor

  Genzyme, a Sanofi Company

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 9, 2011
• First Posted: February 18, 2011
• Study Start: January 1, 2011
• Primary Completion: April 1, 2011
• Study Completion: April 1, 2011
• Last Updated: August 3, 2016

Record last verified: 2016-08

Locations

DE (1)