Amyotrophic Lateral SclerosisPhase 2CL201NCT00647296

Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)

A Phase 2 study testing Dexpramipexole 50 mg/day, Dexpramipexole 150 mg/day and Dexpramipexole 300 mg/day in people with Amyotrophic Lateral Sclerosis, run by Knopp Biosciences at 21 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

This was a 2-part study of dexpramipexole in patients with ALS. Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of dexpramipexole vs. placebo for 12 weeks. Read more about this study

Who is it for?: Ages 21 to 80 years
Conditions the study is for: Amyotrophic Lateral Sclerosis
What is being tested?: Dexpramipexole 50 mg/day, Dexpramipexole 150 mg/day and Dexpramipexole 300 mg/day
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 194 participants
Where: 21 sitesUnited States
Study dates: 9 Apr 2008to 4 Sep 2009
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 21 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients with diagnosis of familial or sporadic ALS, defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria
Patients with ALS symptom onset < 24 months from randomization
Patients with upright vital capacity (VC) > 65% of predicted for age, height, and gender

Who cannot join

Patients in whom causes of neuromuscular weakness other than ALS have not been excluded
Patients without clinical evidence of upper motor neuron dysfunction
Patients with clinically suspected ALS according to the World Federation of Neurology El Escorial criteria
Patients with prior exposure to KNS-760704 or the R(+) enantiomer of pramipexole (i.e., R(+)-pramipexole)
Patients taking other investigational agents (including lithium) within 30 days of randomization or during the study

Trial locations and contacts

21 sites in 1 country.

City, postcode or hospital

21 sites in 1 country

United States21 sites

University of Colorado Health Sciences Center
Aurora, Colorado, 80045
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21228
Massachusettes General Hospital
Boston, Massachusetts, 02129
University of Virginia Health System
Charlottesville, Virginia, 22908
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
University of Kansas Medical Center
Kansas City, Kansas, 66160
Bryan LGH Medical Center East
Lincoln, Nebraska, 68506
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
UCLA, Dept. of Neurology - Neuromuscular/ALS Research Center
Los Angeles, California, 90095
University of Miami Miller School of Medicine
Miami, Florida, 33136
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Columbia University, Lou Gehrig MDA/ALS Research Center
New York, New York, 10032
Drexel University College Of Medicine
Philadelphia, Pennsylvania, 19102
University of Pittsburgh School of Medicine
Pittsburgh, Pennsylvania, 15213
Oregon Health Sciences University
Portland, Oregon, 97239
University of Utah
Salt Lake City, Utah, 84132
University of Texas Health Sciences Center of San Antonio
San Antonio, Texas, 78229
The Forbes Norris MDA/ALS Research Center
San Francisco, California, 94115
University of Washington
Seattle, Washington, 98195
Washington University School of Medicine
St Louis, Missouri, 63110
SUNY Upstate Medical University
Syracuse, New York, 13210

Other trials in United States

About this study

Official title: A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)

This was a 2-part study of dexpramipexole in patients with ALS.

Part 1 was a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of dexpramipexole vs. placebo for 12 weeks.

Part 2 was a randomized, double-blind, 2-arm, parallel group, extension study evaluating the safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole for up to 72 weeks.

Read the detailed description

This study was a two-part, multicenter, double-blind study in subjects with ALS to evaluate the safety and tolerability of dexpramipexole treatment, as well as the preliminary effects on measures of clinical function and mortality of dexpramipexole treatment.

In part 1, 102 subjects with ALS were randomized at 20 US sites to receive placebo, dexpramipexole at 50 mg/day; dexpramipexole at 150 mg/day; or dexpramipexole at 300 mg/day for 12 weeks. Participants who completed Part 1 were eligible to enroll into Part 2.

Part 2 was a randomized, double-blind, 2-arm, parallel-group, extension study evaluating the longer-term safety, tolerability, and clinical effects of oral administration of 2 dosage levels of dexpramipexole. In part 2, following a 4-week, placebo washout, continuing subjects received dexpramipexole at 50 mg/day or 300 mg/day as double-blind treatment for up to 72 additional weeks (Part 2 duration was up to a total of 76 weeks, including the 4 week placebo portion).

Conditions: Amyotrophic Lateral Sclerosis
Being tested: Placebo, Dexpramipexole 50 mg/day, Dexpramipexole 150 mg/day, Dexpramipexole 300 mg/day
Keywords: ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig, Lou Gehrig's, Lou Gehrig's disease, Motor Neuron Disease, Nervous System Diseases, KNS-760704, BIIB050
Medical subject headings: Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Nervous System Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PlaceboDrug
Placebo: 2 tablets taken orally twice daily
Dexpramipexole 50 mg/dayDrug
Also called KNS-760704, BIIB050
Dexpramipexole: 2 x 12.5 mg tablets taken orally twice daily
Dexpramipexole 150 mg/dayDrug
Also called KNS-760704, BIIB050
Dexpramipexole: 2 x 37.5 mg tablets taken orally twice daily
Dexpramipexole 300 mg/dayDrug
Also called KNS-760704, BIIB050
Dexpramipexole: 2 x 75 mg tablets taken orally twice daily

Study groups in NCT00647296
Study group	Role	What they receive
Part 1: Placebo or Dexpramipexole	Placebo comparator	During Part 1, subjects received twice daily doses of dexpramipexole (50 mg/day, 150 mg/day, or 300 mg/day) or matching placebo for approximately 12 weeks. Receives: Drug: Placebo; Drug: Dexpramipexole 50 mg/day; Drug: Dexpramipexole 150 mg/day; Drug: Dexpramipexole 300 mg/day
Part 2: Placebo washout	Experimental	At the beginning of Part 2, subjects received twice daily doses of placebo for approximately 4 weeks. Receives: Drug: Placebo
Part 2: Dexpramipexole	Experimental	Following the Part 2 placebo washout, subjects received dexpramipexole (50 mg/day or 300 mg/day), subjects received twice daily doses of placebo for up to 18 months. Receives: Drug: Dexpramipexole 50 mg/day; Drug: Dexpramipexole 300 mg/day

What is being measured

The primary outcome is the main question the study is designed to answer.

Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. 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.
Measured: 12 weeks
Part 1: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group
Number of Participants with Potentially Clinically Significant Blood Chemistry Results by Treatment Group. 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.
Measured: 12 weeks
Part 1: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Measured: 12 weeks
Part 1: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Measured: 12 weeks

Secondary outcomes (14)

Part 1: Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 12 by Treatment Group
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 of 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.
Measured: 12 weeks
Part 1: Slope of Upright Vital Capacity From Baseline to Week 12 by Treatment Group
Slope of change in Upright Vital Capacity (percent predicted upright vital capacity) from Baseline to Week 12. A negative change/slope indicates clinical worsening. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis as percent predicted upright vital capacity. Units for slope are change per month in percent predicted upright vital capacity.
Measured: 12 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Hematology
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. 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.
Measured: 4 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Blood Chemistry Results
Number of Participants with Potentially Clinically Significant Blood Chemistry Results by Treatment Group. 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.
Measured: 4 weeks
Part 2 Placebo Washout: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Measured: 4 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Vital Sign Measurements
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Measured: 4 weeks
Part 2 Placebo Washout: Absolute Change in ALSFRS-R Total Score
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 of 48, with higher scores representing better function.
Units are points on the ALSFRS-R score as an absolute change from the baseline of the placebo washout to week 4 of the placebo washout.
Measured: 4 weeks
Part 2 Placebo Washout: Absolute Change in Upright Vital Capacity (Percent Predicted) From Baseline to End of Placebo Washout (Week 4)
Absolute change in Upright Vital Capacity From Baseline to Week 4. Units are percent of predicted Upright Vital Capacity. A negative change indicates clinical worsening.
Measured: 4 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. 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 parameter.
Measured: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group
Number of Participants with Potentially Clinically Significant Blood Chemistry Results by Treatment Group. 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 parameter.
Measured: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. 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 parameter.
Measured: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. 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 parameter.
Measured: up to 76 weeks
Part 2 Double-Blind Treatment: Slope of the ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 28 by Treatment Group
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 of 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 n units on the ALSFRS-R scale.
Measured: 28 weeks
Part 2 Double-Blind Treatment: Slope of Percent Predicted Upright Vital Capacity From Baseline by Treatment Group
Slope of Upright Vital Capacity (percent predicted) through Week 28. A negative change indicates clinical worsening. Slope is calculated using a linear mixed effects model with x-axis time in months and y-axis percent predicted upright vital capacity. Units for slope are change per month in percent predicted upright vital capacity.
Measured: Baseline of randomized phase of Part 2 to week 28 of randomized phase of Part 2

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

9 Apr 20084 Sep 2009

First posted31 Mar 2008 (estimated)
Study start9 Apr 2008
Primary completion31 Jul 2009
Study completion4 Sep 2009

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind
Who is masked: Participant, Care provider, Investigator, Outcomes assessor
Enrolment: 194 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00647296
Sponsor's protocol ID: KNS-760704-CL201
Acronym: CL201
Status last verified: Mar 2021
First submitted: 26 Mar 2008
Last updated: 8 Jul 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No

Publications

Cudkowicz M, Bozik ME, Ingersoll EW, Miller R, Mitsumoto H, Shefner J, Moore DH, Schoenfeld D, Mather JL, Archibald D, Sullivan M, Amburgey C, Moritz J, Gribkoff VK. The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nat Med. 2011 Nov 20;17(12):1652-6. doi: 10.1038/nm.2579.
PubMed 22101764

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)

All Amyotrophic Lateral Sclerosis trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
ClinicalTrials.gov ID: NCT00647296
Acronym / sponsor ID: CL201 / KNS-760704-CL201
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig, Lou Gehrig's, Lou Gehrig's disease, Motor Neuron Disease, Nervous System Diseases, KNS-760704
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 194 participants (actual)
Lead sponsor: Knopp BiosciencesIndustry (pharma, biotech, devices)
Study start: 9 Apr 2008 (actual)
Primary completion: 31 Jul 2009 (actual)
Study completion: 4 Sep 2009 (actual)
First posted: 31 Mar 2008 (estimated)
Results: Posted 8 Jul 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This was a 2-part study of dexpramipexole in patients with ALS.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Quadruple blind (participant, care provider, investigator, outcomes assessor)
Number of arms: 3

Arm	Type	Receives
Part 1: Placebo or Dexpramipexole	Placebo comparator	Drug: Placebo, Drug: Dexpramipexole 50 mg/day, Drug: Dexpramipexole 150 mg/day, Drug: Dexpramipexole 300 mg/dayDuring Part 1, subjects received twice daily doses of dexpramipexole (50 mg/day, 150 mg/day, or 300 mg/day) or matching placebo for approximately 12 weeks.
Part 2: Placebo washout	Experimental	Drug: PlaceboAt the beginning of Part 2, subjects received twice daily doses of placebo for approximately 4 weeks.
Part 2: Dexpramipexole	Experimental	Drug: Dexpramipexole 50 mg/day, Drug: Dexpramipexole 300 mg/dayFollowing the Part 2 placebo washout, subjects received dexpramipexole (50 mg/day or 300 mg/day), subjects received twice daily doses of placebo for up to 18 months.

Who may qualify

Ages: 21 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

Patients with diagnosis of familial or sporadic ALS, defined as meeting the possible, laboratory-supported probable, probable, or definite criteria for a diagnosis of ALS according to the World Federation of Neurology El Escorial criteria
Patients with ALS symptom onset < 24 months from randomization
Patients with upright vital capacity (VC) > 65% of predicted for age, height, and gender

Key exclusion criteria (5 listed)

Patients in whom causes of neuromuscular weakness other than ALS have not been excluded
Patients without clinical evidence of upper motor neuron dysfunction
Patients with clinically suspected ALS according to the World Federation of Neurology El Escorial criteria
Patients with prior exposure to KNS-760704 or the R(+) enantiomer of pramipexole (i.e., R(+)-pramipexole)
Patients taking other investigational agents (including lithium) within 30 days of randomization or during the study

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
PlaceboPlacebo: 2 tablets taken orally twice daily	Drug	Part 1: Placebo or Dexpramipexole, Part 2: Placebo washout
Dexpramipexole 50 mg/dayAlso: KNS-760704, BIIB050Dexpramipexole: 2 x 12.5 mg tablets taken orally twice daily	Drug	Part 1: Placebo or Dexpramipexole, Part 2: Dexpramipexole
Dexpramipexole 150 mg/dayAlso: KNS-760704, BIIB050Dexpramipexole: 2 x 37.5 mg tablets taken orally twice daily	Drug	Part 1: Placebo or Dexpramipexole
Dexpramipexole 300 mg/dayAlso: KNS-760704, BIIB050Dexpramipexole: 2 x 75 mg tablets taken orally twice daily	Drug	Part 1: Placebo or Dexpramipexole, Part 2: Dexpramipexole

Primary outcome

Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group
Time frame: 12 weeks
Number of Participants with Potentially Clinically Significant Hematology Results by Treatment Group. 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.
Part 1: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group
Time frame: 12 weeks
Number of Participants with Potentially Clinically Significant Blood Chemistry Results by Treatment Group. 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.
Part 1: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group
Time frame: 12 weeks
Number of Participants with Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.
Part 1: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group
Time frame: 12 weeks
Number of Participants with Potentially Clinically Significant Vital Sign Measurements by Treatment Group. Percentages are based on the number of patients with at least one non-missing post-baseline value in each treatment group.

14 secondary outcomes

Part 1: Slope of ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 12 by Treatment Group
Time frame: 12 weeks
Part 1: Slope of Upright Vital Capacity From Baseline to Week 12 by Treatment Group
Time frame: 12 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Hematology
Time frame: 4 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Blood Chemistry Results
Time frame: 4 weeks
Part 2 Placebo Washout: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings
Time frame: 4 weeks
Part 2 Placebo Washout: Number of Participants With Potentially Clinically Significant Vital Sign Measurements
Time frame: 4 weeks
Part 2 Placebo Washout: Absolute Change in ALSFRS-R Total Score
Time frame: 4 weeks
Part 2 Placebo Washout: Absolute Change in Upright Vital Capacity (Percent Predicted) From Baseline to End of Placebo Washout (Week 4)
Time frame: 4 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group
Time frame: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group
Time frame: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group
Time frame: up to 76 weeks
Part 2 Double-Blind Treatment: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group
Time frame: up to 76 weeks
Part 2 Double-Blind Treatment: Slope of the ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) From Baseline to Week 28 by Treatment Group
Time frame: 28 weeks
Part 2 Double-Blind Treatment: Slope of Percent Predicted Upright Vital Capacity From Baseline by Treatment Group
Time frame: Baseline of randomized phase of Part 2 to week 28 of randomized phase of Part 2

Results and safety

Results posted to the registry on 8 Jul 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Placebo Twice Daily	27	26
50 mg/Day Dexpramipexole	23	22
150 mg/Day Dexpramipexole	26	26
300 mg/Day Dexpramipexole	26	24

Primary outcome: Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Placebo n = 27	50 mg/Day n = 23	150 mg/Day n = 26	300 mg/Day n = 26
White Blood Cell Count (x10^3/uL) < 2000/uL	0	0	0	0
Neutrophils (x10^3/uL) < 1000/uL	0	0	0	0
Eosinophils (x10^3/uL) > 5000/uL	0	0	0	0
Hemoglobin (g/dL) < 8 g/dL	0	0	0	0
Platelets (x10^3/uL) < 50,000/uL	0	0	0	0

Population analysed: Safety Population: Data from all enrolled subjects who were randomized in Part 1 and received at least 1 dose of study drug.

Primary outcome: Part 1: Number of Participants With Potentially Clinically Significant Blood Chemistry Results by Treatment Group

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Placebo n = 27	50 mg/Day n = 23	150 mg/Day n = 26	300 mg/Day n = 26
Creatine Kinase (U/L) >= 10xULN	0	0	0	1
Total Bilirubin (mg/dL) > 1.5xULN	0	0	0	0
ALT (SGPT) (U/L) > 3xULN	0	0	0	2
AST (SGOT) (U/L) > 3xULN	0	0	0	1
Alkaline Phosphatase (U/L) > 1.5xULN	0	0	0	1
BUN (mg/dL) > 5xULN	0	0	0	0
Creatinine (mg/dL) > 3xULN	0	0	0	0
Sodium (mEq/L) low < 123 mEq/L	0	0	0	0

Population analysed: Safety Population: Data from all enrolled subjects who were randomized in Part 1 and received at least 1 dose of study drug.

Primary outcome: Part 1: Number of Participants With Treatment Emergent Potentially Clinically Significant Electrocardiogram (ECG) Findings by Treatment Group

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Placebo n = 27	50 mg/Day n = 23	150 mg/Day n = 26	300 mg/Day n = 26
Rhythm Sinus Tachycardia > 120 bpm	0	0	0	0
Rhythm Sinus Bradycardia < 40 bpm	0	0	0	0
Rhythm Sinus Block/Sinus Arrest	0	0	0	0
Rhythm Junctional Rhythm	0	0	0	0
Rhythm Atrial Flutter	0	0	0	0
Rhythm Atrial Fibrillation	0	0	0	1
Rhythm Supraventricular	0	0	0	0
Rhythm Tachycardia > 100 bpm	0	0	0	0

Population analysed: Safety Population: Data from all enrolled subjects who were randomized in Part 1 and received at least 1 dose of study drug.

Primary outcome: Part 1: Number of Participants With Potentially Clinically Significant Vital Sign Measurements by Treatment Group

Time frame: 12 weeks. Count of participants. Unit: Participants

Measure	Placebo n = 27	50 mg/Day n = 23	300 mg/Day n = 26
Systolic Blood Pressure Increase of > 20 mmHg from baseline and >= 180mmHg	0	2	0
Systolic Blood Pressure Decrease of < -20 mmHg from baseline and <= 90 mmHg	2	0	0
Diastolic Blood Pressure Increase of > 15 mmHg from baseline and >= 105 mmHg	0	1	0
Diastolic Blood Pressure Decrease of < -15 mmHg from baseline and <= 50 mmHg	1	1	0
Pulse Increase of > 15 bpm from baseline and >= 120	0	1	0
Pulse Decrease of < -15 bpm from baseline and <= 50	0	0	1
Body Weight Increase of > 7% lbs from baseline	0	0	0
Body Weight Decrease of < -7% lbs from baseline	3	2	1

Population analysed: Safety Population: Data from all enrolled subjects who were randomized in Part 1 and received at least 1 dose of study drug.

14 secondary outcomes are reported in the full results.

Adverse events

Time frame: Part 1: up to 12 weeks; Part 2 Placebo washout: up to 4 weeks; Part 2 Treatment period: up to 18 months. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part 1: Placebo	0 of 27 (0%)	25 of 27 (92.6%)	0 of 27
Part 1: Dexpramipexole (50 mg/Day)	2 of 23 (8.7%)	19 of 23 (82.6%)	0 of 23
Part 1: Dexpramipexole (150 mg/Day)	0 of 26 (0%)	25 of 26 (96.2%)	0 of 26
Part 1: Dexpramipexole (300 mg/Day)	3 of 26 (11.5%)	23 of 26 (88.5%)	0 of 26
Part 2: Placebo Washout	5 of 97 (5.2%)	46 of 97 (47.4%)	3 of 97
Part 2: Dexpramipexole (50 mg/Day)	14 of 48 (29.2%)	46 of 48 (95.8%)	15 of 48
Part 2: Dexpramipexole (300 mg/Day)	12 of 44 (27.3%)	41 of 44 (93.2%)	9 of 44

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Jul 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 22101764Cudkowicz M, Bozik ME, Ingersoll EW, Miller R, Mitsumoto H, Shefner J, Moore DH, Schoenfeld D, Mather JL, Archibald D, Sullivan M, Amburgey C, Moritz J, Gribkoff VK. The effects of dexpramipexole (KNS-760704) in individuals with amyotrophic lateral sclerosis. Nat Med. 2011 Nov 20;17(12):1652-6. doi: 10.1038/nm.2579.

Related trials for Amyotrophic Lateral Sclerosis

RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
Not yet recruiting
A Study on the Addition of Alsum30 for the Treatment of Amyotrophic Lateral Sclerosis
Amyotrophic Lateral Sclerosis (ALS), Bacillus Coagulans
Active, not recruiting
Functional Outcomes and Control Using Synchron BCI - Australia
Neurologic Disorder, Neurologic Diseases
Not yet recruitingPhase 1
Safety, Tolerability, and Pharmacodynamic Exploration of Orelabrutinib in Patients With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
Clinical Outcome Assessment for AT & BCI
Spinal Cord Injury, ALS (Amyotrophic Lateral Sclerosis)

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

The record lists no contact. Use the official record to reach the sponsor.

21 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 8 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 21 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 8 Jul 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Part 1: Number of Participants With Potentially Clinically Significant Hematology Results by Treatment Group (time frame: 12 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00647296, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jul 2021.