Dexpramipexole SAD/MAD Study

NCT01449578 | Completed Phase 1

• 1 other identifier: 223HV103
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

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

  Change from baseline to 11 Days.

Secondary Outcomes (1)

• Dexpramipexole pharmacokinetics time frame in plasma

  pre-dose and 0.5, 1, 2, 3, 4, 6, 8, 12, 16, 24, 36, 48, and 72 hours post-dose

Study Arms (10)

Part A, Treatment 1

EXPERIMENTAL

Dexpramipexole single dose (SAD Dose 1)

Drug: Dexpramipexole

Part A, Treatment 1 placebo

PLACEBO COMPARATOR

Dexpramipexole single dose placebo (SAD Dose 1)

Drug: Dexpramipexole Placebo

Part A, Treatment 2

EXPERIMENTAL

Dexpramipexole single dose (SAD Dose 2)

Drug: Dexpramipexole

Part A, Treatment 2 placebo

PLACEBO COMPARATOR

Dexpramipexole single dose placebo (SAD Dose 2)

Drug: Dexpramipexole Placebo

Part A, Treatment 3

EXPERIMENTAL

Dexpramipexole single dose (SAD Dose 3)

Drug: Dexpramipexole

Part A, Treatment 3 placebo

PLACEBO COMPARATOR

Dexpramipexole single dose placebo (SAD Dose 3)

Drug: Dexpramipexole Placebo

Part B, Treatment 1

EXPERIMENTAL

Dexpramipexole multiple dose (MAD Dose 1)

Drug: Dexpramipexole

Part B, Treatment 1 placebo

PLACEBO COMPARATOR

Dexpramipexole multiple dose placebo (MAD Dose 1)

Drug: Dexpramipexole Placebo

Part B, Treatment 2

EXPERIMENTAL

Dexpramipexole multiple dose (MAD Dose 2)

Drug: Dexpramipexole

Part B, Treatment 2 placebo

PLACEBO COMPARATOR

Dexpramipexole multiple dose placebo (MAD Dose 2)

Drug: Dexpramipexole Placebo

Interventions

Dexpramipexole DRUG

Oral Tablet at varying doses

Also known as: BIIB050

Part A, Treatment 1; Part A, Treatment 2; Part A, Treatment 3; Part B, Treatment 1; Part B, Treatment 2

Dexpramipexole Placebo DRUG

Oral tablet at varying doses

Part A, Treatment 1 placebo; Part A, Treatment 2 placebo; Part A, Treatment 3 placebo; Part B, Treatment 1 placebo; Part B, Treatment 2 placebo

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Knopp Biosciences: lead

Study Sites (1)

Research Site

Overland Park, Kansas, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Dexpramipexole

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Benzothiazoles; Thiazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2011
• First Posted: October 10, 2011
• Study Start: November 1, 2011
• Primary Completion: January 1, 2012
• Study Completion: January 1, 2012
• Last Updated: November 25, 2014

Record last verified: 2014-11

Locations

US (1)