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Phase 3 Extension Study of Dexpramipexole in ALS (ENVISION)

Primary Purpose

Amyotrophic Lateral Sclerosis

Status

Terminated

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Dexpramipexole

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Subject has the ability to understand the purpose and risks of the study and provide signed and dated informed consent (or have the consent confirmed by a witness if unable to write) and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.
Subject was enrolled in either CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
Subject has completed their last visit in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
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.

Exclusion Criteria:

Subject withdrew prematurely from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
Subject permanently discontinued study treatment in Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) for any reason other than enrollment into this study.
Subject from Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189) has a significant change in medical history (including laboratory tests or a clinically significant condition) that in the opinion of the Investigator would impair the subject's medical fitness for participation and preclude treatment.
Female subject who is pregnant or breastfeeding.
Subject is currently enrolled in any investigational drug study other than Study CL211 (NCT00931944) or Study 223AS302 (NCTO1281189).
Subject is taking pramipexole, other dopamine agonists, any other agent with dopaminergic activity, or any other disallowed concomitant medication.
Subject is unwilling or unable to comply with the requirements of the protocol including the presence of any condition (physical, mental, or social) that is likely to affect the subject's ability to comply with the protocol. At a minimum, subjects who are not able to travel to the study site must be willing to agree to remote blood draws for clinical laboratory evaluations and telephone visits to report Adverse Events, concomitant medications, and Amyotrophic Lateral Sclerosis Functional Rating Scale (revised) (ALSFRS-R) scores.

Sites / Locations

Barrow Neurological Institute - St. Joseph's Hospital
University of California at San Francisco - Fresno
University of California, Irvine
University of California, Davis
California Pacific Medical Center
Hospital for Special Care
Mayo Clinic - Jacksonville
University of Miami Miller School of Medicine
University of South Florida Medical Center
Emory University
Northwestern University
Indiana University
University of Iowa
University of Kansas Medical Center
Johns Hopkins University School of Medicine
Massachusetts General Hospital
St. Mary's Health Care
Hennepin County Medical Center
Mayo Clinic - Rochester
Washington University School of Medicine
Neurology Associates, P.C.
University of Nevada School of Medicine
Dartmouth-Hitchcock Medical Center
Columbia University
Research Foundation of the State University of New York
Carolinas Medical Center
Duke University Medical Center
Wake Forest University
The Cleveland Clinic Foundation
Ohio State University
Providence ALS Center
Penn State Hershey Medical Center
Drexel University College of Medicine
ALS Center at Penn
University of Pittsburgh Medical Center
Vanderbilt University Medical Center
Texas Neurology
Methodist Neurological Institute
University of Texas Health Sciences Center
University of Utah
University of Virginia Health System
University of Washington
Prince of Wales Hospital
Westmead Hospital
Royal Brisbane and Women's Hospital
Calvary Health Care Bethlehem
AZ St-Lucas
UZ Leuven
Univ of Calgary / Foothills MC
CHUM - Hopital Notre Dame
Mcgill University
London Health Sciences Centre
Sunnybrook and Women's College and Health Sciences Centre
University of British Columbia
CHRU de Lille - Hôpital Roger Salengro
Centre Hospitalier La Timone
CHU Gui de Chauliac
CHU de Nice - Hôpital de l'Archet 1
Hôpital La Pitié Salpétrière
Charité - Universitätsmedizin Berlin
Bergmannsheil Gmbh
Medizinische Hochschule Hannover (MHH)
Universitätsklinikum Jena
University of Ulm, RKU
Beaumont Hospital
Academisch Medisch Centrum
UMC St. Radboud
Universitair Medisch Centrum Utrecht
Hospital Universitario de Bellvitge
Hospital Vall d'Hebron
Hospital La Paz
Hospital Carlos III
Sahlgrenska Universitetssjukhuset
Karolinska Universitetssjukhuset, Solna
Queen Elizabeth Hospital
Walton Centre for Neurology & Neurosurgery
Kings College Hospital NHS Foundation Trust
Newcastle University Hospital - Clinical Ageing Research Unit
John Radcliffe Hospital
Sheffield Institute for Transnational Neuroscience

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Dexpramipexole

Arm Description

Dexpramipexole open-label

Outcomes

Primary Outcome Measures

Number of Subjects Who Reported an Adverse Event

The number of subjects who reported an adverse event during the study

Number of Subjects Who Experienced a Serious Adverse Event

The number of subjects enrolled who reported a serious adverse event during the study

Number of Subjects Who Discontinued the Study Treatment Due to an Adverse Event

The number of subjects enrolled who discontinued the study treatment due to an adverse event during the study

Number of Participants With Potentially Clinically Significant Vital Sign Results

Number of Participants with Potentially Clinically Significant Vital Sign Abnormalities. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

Number of Participants With Potentially Clinically Significant Hematology Results

Number of Participants with Potentially Clinically Significant Hematology Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

Number of Participants With Potentially Clinically Significant Blood Chemistry Results

Number of Participants with Potentially Clinically Significant Blood Chemistry Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

Number of Participants With Potentially Clinically Significant ECG Results

Number of Participants with Potentially Clinically Significant ECG Results. Percentages based on number of patients with at least one non-missing post-baseline value in each treatment group. Patients are only counted once per criterion per laboratory test.

Secondary Outcome Measures

Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to End of Study

The ALSFRS-R (ALS functional rating scale with respiratory component) is a validated scale which measures 4 functional domains, comprising respiratory function, bulbar function, gross motor skills, and fine motor skills. There are a total of 12 questions, each scored from 0 to 4 for a total possible score between 0 to 48, with higher scores representing better function. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis ALSFRS-R score. Units for slope are change per month in units on the ALSFRS-R scale.

Slope of Sniff Nasal Inspiratory Pressure (SNIP) From Baseline to End of Study

SNIP is a test of inspiratory force (sternocleidomastoid and diaphragm) measured via a nasal cannula and is used to assess respiratory muscle weakness and to monitor changes in respiratory muscle strength over time. During the SNIP maneuver, the patient is asked to perform a strong, sharp, maximal sniff, whereby nasal pressure is measured via nasal cannula. The maximum recorded value after several attempts, with rest in between attempts, was use in the analysis.

Death up to 6 Months

Kaplan-Meier estimate of percentage of subjects who died up to 6 months

Percentage of Participants With Death or Death Equivalent up to 6 Months

Kaplan-Meier estimate of percentage of subjects who died or had a death equivalent event (tracheostomy or permanent assisted ventilation [PAV], defined as use of noninvasive ventilation [NIV] for ≥22 hours per day for ≥10 days) up to 6 months

Full Information

NCT ID

NCT01622088

First Posted

May 3, 2012

Last Updated

April 8, 2022

Sponsor

Knopp Biosciences

Collaborators

Biogen

1. Study Identification

Unique Protocol Identification Number

NCT01622088

Brief Title

Phase 3 Extension Study of Dexpramipexole in ALS

Acronym

ENVISION

Official Title

An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Terminated

Why Stopped

The Initial Phase 3 Study (NCTO1281189) did not meet its primary efficacy endpoint.

Study Start Date

June 2012 (undefined)

Primary Completion Date

February 2013 (Actual)

Study Completion Date

February 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Knopp Biosciences

Collaborators

Biogen

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

The purpose of the study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.

Detailed Description

Amyotrophic Lateral Sclerosis (ALS) is a rapidly progressive, degenerative disease of motor neurons in the brain and spinal cord that leads to muscle atrophy and spasticity in limb and bulbar muscles resulting in weakness and loss of ambulation, oropharyngeal dysfunction, weight loss, and ultimately respiratory failure. The purpose of this study is to collect long-term safety data from subjects with Amyotrophic Lateral Sclerosis (ALS) exposed to dexpramipexole.

6. Conditions and Keywords

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

Amyotrophic Lateral Sclerosis

Keywords

ALS, Motor Neuron Disease, Amyotrophic Lateral Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

616 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Dexpramipexole

Arm Type

Experimental

Arm Description

Dexpramipexole open-label

Intervention Type

Drug

Intervention Name(s)

Dexpramipexole

Other Intervention Name(s)

BIIB050

Intervention Description

Oral tablet 150 mg given twice daily (BID)

Primary Outcome Measure Information:

Title

Number of Subjects Who Reported an Adverse Event

Description

The number of subjects who reported an adverse event during the study

Time Frame