Dexpramipexole SAD/MAD Study

November 24, 2014 updated by: Knopp Biosciences

A Randomized, Blinded, Placebo-Controlled Ascending Dose Study of the Safety and Pharmacokinetics of Dexpramipexole in Healthy Volunteers

This Phase 1 study will explore the safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

Preclinical and clinical data to date support the exploration of doses of dexpramipexole higher than 150 mg twice daily for their effectiveness in slowing the progression of ALS. Exploration of doses of dexpramipexole higher than 150 mg twice daily is justified by preclinical and clinical data that suggest that higher doses could potentially be more effective in slowing the progression of ALS than the dose of dexpramipexole currently being explored in Phase 3 studies (150 mg twice daily).

This is a Phase 1, single-center, blinded, randomized, placebo controlled, ascending-dose study consisting of 2 parts; Part A (single-ascending dose [SAD]) and Part B (multiple ascending dose [MAD]). The study will explore safety, tolerability, and pharmacokinetics of single doses ranging from 300 to 600 mg and multiple daily doses ranging from 225 mg to 300 mg BID dexpramipexole in healthy volunteers.

Study Type

Interventional

Enrollment (Actual)

63

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Kansas
      • Overland Park, Kansas, United States
        • Research Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 55 years (Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Must give written informed consent.
  • Adult males/females aged 18 to 55 years inclusive and between 19 and 30 kg/m2 body mass index (BMI), inclusive at screening.
  • Subjects who are healthy as determined by prestudy medical history, physical examination and 12-lead ECG.
  • Subjects of childbearing potential must practice effective contraception during the study and be willing and able to continue contraception for 1 month (females) or 3 months (males) after their last dose of study treatment.
  • Normal systemic blood pressure defined as a systolic blood pressure of 90 to 140 mmHg and a diastolic blood pressure of 50 to 90 mmHg.

Exclusion Criteria:

  • History of cardiovascular disease (e.g., hypertension, arrhythmia, heart failure, Long QT Syndrome, or other conditions/diseases causing prolongation of the QT/QTc interval).
  • A prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval >450 ms before study treatment administration) at screening, admission or pre-dose on Day 1.
  • Any clinically important abnormalities in resting ECG that may interfere with the interpretation of QTc interval changes at screening, admission or pre-dose on Day 1.
  • Prior exposure to dexpramipexole.
  • Treatment with pramipexole or any dopamine agonist within 1 year.
  • Treatment with another investigational drug or approved therapy for investigational use within 30 days, or 5 half-lives (whichever is longer), or in follow up for any other drug, biologic, or device study.
  • Currently active infection or serious infection (e.g., pneumonia, septicemia) within the 2 months prior to Day -2 as determined by the Investigator.
  • Female subjects who are pregnant, currently breastfeeding, or attempting to conceive during the study.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Single Group Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part A, Treatment 1
Dexpramipexole single dose (SAD Dose 1)
Drug: Dexpramipexole
Oral Tablet at varying doses
Other Names:
  • BIIB050
Placebo Comparator: Part A, Treatment 1 placebo
Dexpramipexole single dose placebo (SAD Dose 1)
Drug: Dexpramipexole Placebo
Oral tablet at varying doses
Experimental: Part A, Treatment 2
Dexpramipexole single dose (SAD Dose 2)
Drug: Dexpramipexole
Oral Tablet at varying doses
Other Names:
  • BIIB050
Placebo Comparator: Part A, Treatment 2 placebo
Dexpramipexole single dose placebo (SAD Dose 2)
Drug: Dexpramipexole Placebo
Oral tablet at varying doses
Experimental: Part A, Treatment 3
Dexpramipexole single dose (SAD Dose 3)
Drug: Dexpramipexole
Oral Tablet at varying doses
Other Names:
  • BIIB050
Placebo Comparator: Part A, Treatment 3 placebo
Dexpramipexole single dose placebo (SAD Dose 3)
Drug: Dexpramipexole Placebo
Oral tablet at varying doses
Experimental: Part B, Treatment 1
Dexpramipexole multiple dose (MAD Dose 1)
Drug: Dexpramipexole
Oral Tablet at varying doses
Other Names:
  • BIIB050
Placebo Comparator: Part B, Treatment 1 placebo
Dexpramipexole multiple dose placebo (MAD Dose 1)
Drug: Dexpramipexole Placebo
Oral tablet at varying doses
Experimental: Part B, Treatment 2
Dexpramipexole multiple dose (MAD Dose 2)
Drug: Dexpramipexole
Oral Tablet at varying doses
Other Names:
  • BIIB050
Placebo Comparator: Part B, Treatment 2 placebo
Dexpramipexole multiple dose placebo (MAD Dose 2)
Drug: Dexpramipexole Placebo
Oral tablet at varying doses

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety and tolerability based on medical review of adverse events & results of vital sign measurements, electrocardiogram (ECGs), physical examinations and clinical laboratory tests.
Time Frame: Change from baseline to 11 Days.
Change from baseline to 11 Days.

Secondary Outcome Measures

Outcome Measure
Time Frame
Dexpramipexole pharmacokinetics time frame in plasma
Time Frame: pre-dose and 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose
pre-dose and 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Knopp Biosciences

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2011

Primary Completion (Actual)

January 1, 2012

Study Completion (Actual)

January 1, 2012

Study Registration Dates

First Submitted

September 22, 2011

First Submitted That Met QC Criteria

October 7, 2011

First Posted (Estimate)

October 10, 2011

Study Record Updates

Last Update Posted (Estimate)

November 25, 2014

Last Update Submitted That Met QC Criteria

November 24, 2014

Last Verified

November 1, 2014

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 223HV103

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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