Back to results

Dexpramipexole SAD/MAD Study

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Completed

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Dexpramipexole

Dexpramipexole Placebo

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - 55 Years (Adult)All SexesAccepts Healthy Volunteers

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.

Sites / Locations

Research Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm 6

Arm 7

Arm 8

Arm 9

Arm 10

Arm Type

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Experimental

Placebo Comparator

Arm Label

Part A, Treatment 1

Part A, Treatment 1 placebo

Part A, Treatment 2

Part A, Treatment 2 placebo

Part A, Treatment 3

Part A, Treatment 3 placebo

Part B, Treatment 1

Part B, Treatment 1 placebo

Part B, Treatment 2

Part B, Treatment 2 placebo

Arm Description

Dexpramipexole single dose (SAD Dose 1)

Dexpramipexole single dose placebo (SAD Dose 1)

Dexpramipexole single dose (SAD Dose 2)

Dexpramipexole single dose placebo (SAD Dose 2)

Dexpramipexole single dose (SAD Dose 3)

Dexpramipexole single dose placebo (SAD Dose 3)

Dexpramipexole multiple dose (MAD Dose 1)

Dexpramipexole multiple dose placebo (MAD Dose 1)

Dexpramipexole multiple dose (MAD Dose 2)

Dexpramipexole multiple dose placebo (MAD Dose 2)

Outcomes

Primary Outcome Measures

Safety and tolerability based on medical review of adverse events & results of vital sign measurements, electrocardiogram (ECGs), physical examinations and clinical laboratory tests.

Secondary Outcome Measures

Dexpramipexole pharmacokinetics time frame in plasma

Full Information

NCT ID

NCT01449578

First Posted

September 22, 2011

Last Updated

November 24, 2014

Sponsor

Knopp Biosciences

1. Study Identification

Unique Protocol Identification Number

NCT01449578

Brief Title

Dexpramipexole SAD/MAD Study

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

November 2014

Overall Recruitment Status

Completed

Study Start Date

November 2011 (undefined)

Primary Completion Date

January 2012 (Actual)

Study Completion Date

January 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Knopp Biosciences

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

63 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Part A, Treatment 1

Arm Type

Experimental

Arm Description

Dexpramipexole single dose (SAD Dose 1)

Arm Title

Part A, Treatment 1 placebo

Arm Type

Placebo Comparator

Arm Description

Dexpramipexole single dose placebo (SAD Dose 1)

Arm Title

Part A, Treatment 2

Arm Type

Experimental

Arm Description

Dexpramipexole single dose (SAD Dose 2)

Arm Title

Part A, Treatment 2 placebo

Arm Type

Placebo Comparator

Arm Description

Dexpramipexole single dose placebo (SAD Dose 2)

Arm Title

Part A, Treatment 3

Arm Type

Experimental

Arm Description

Dexpramipexole single dose (SAD Dose 3)

Arm Title

Part A, Treatment 3 placebo

Arm Type

Placebo Comparator

Arm Description

Dexpramipexole single dose placebo (SAD Dose 3)

Arm Title

Part B, Treatment 1

Arm Type

Experimental

Arm Description

Dexpramipexole multiple dose (MAD Dose 1)

Arm Title

Part B, Treatment 1 placebo

Arm Type

Placebo Comparator

Arm Description

Dexpramipexole multiple dose placebo (MAD Dose 1)

Arm Title

Part B, Treatment 2

Arm Type

Experimental

Arm Description

Dexpramipexole multiple dose (MAD Dose 2)

Arm Title

Part B, Treatment 2 placebo

Arm Type

Placebo Comparator

Arm Description

Dexpramipexole multiple dose placebo (MAD Dose 2)

Intervention Type

Drug

Intervention Name(s)

Dexpramipexole

Other Intervention Name(s)

BIIB050

Intervention Description

Oral Tablet at varying doses

Intervention Type

Drug

Intervention Name(s)

Dexpramipexole Placebo

Intervention Description

Oral tablet at varying doses

Primary Outcome Measure Information:

Title

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.

Secondary Outcome Measure Information:

Title

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

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

55 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

Research Site

City

Overland Park

State/Province

Kansas

Country

United States

12. IPD Sharing Statement

Learn more about this trial

Dexpramipexole SAD/MAD Study

We'll reach out to this number within 24 hrs