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Completed

NCT Number: NCT00931944

Open-Label, Safety and Tolerability Extension Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL211)

This is an open-label, multi-center study designed to extend the evaluation of the safety, tolerability, and clinical effects of oral administration of KNS-760704 in patients with ALS.

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Key information

Conditions

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

Age range

21 year–80 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States

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About this study

Patients who complete the Part 2 Week 28 visit in study KNS-760704-CL201 and patients with ALS who were actively receiving RTPB [(6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydro-chloride monohydrate] under Research IND #60,948 were eligible to participate in this study.

Eligible patients received 1 tablet of KNS-760704 (150 mg) every 12 hours (Q12H) (300 mg total daily dose) for up to 180 weeks.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patient has provided signed informed consent for this trial before the commencement of any study-related procedure
  • Patient is actively participating in Knopp Protocol KNS-760704-CL201 and has completed the Part 2 Week 28 visit in that study or patient is actively receiving RTPB under Research IND #60,948

Exclusion criteria

  • Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948.
  • Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)
  • Patient was not taking RTPB under Research IND #60,948 prior to April 30, 2009 (applies to patients enrolled under Research IND #60,948 only)

Treatment and study plan

KNS-760704

Drug

150 mg Q12H KNS-760704 given orally (300 mg total daily dose)

Other names: dexpramipexole

Primary outcomes

  1. Number of Participants With Potentially Clinically Significant Hematology Results

    Time frame: 180 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.

  2. Number of Participants With Potentially Clinically Significant Liver Enzyme Abnormalities

    Time frame: 180 weeks

    Number of participants with potentially clinically significant liver enzyme abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.

  3. Number of Participants With Potentially Clinically ECG Abnormalities

    Time frame: 180 weeks

    Number of participants with potentially clinically significant ECG abnormalities for the safety population are presented. Percentages are based on the number of patients in the safety population who had at least one non-missing, post-baseline value.

  4. Number of Participants With Potentially Clinically Significant Vital Sign Abnormalities

    Time frame: 180 weeks

    Number of participants with potentially clinically significant vital sign abnormalities for the safety population are presented. Percentages are based on the number of patients with at least one non-missing, post-baseline value for each parameter.

Secondary outcomes

  1. Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 12

    Time frame: 12 weeks

    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. Therefore, the score ranges from 0 to 48 with higher scores representing better function.

  2. Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 24

    Time frame: 24 weeks

    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. Therefore, the score ranges from 0 to 48 with higher scores representing better function.

  3. Change in ALSFRS-R (ALS Functional Rating Scale With Respiratory Component) Total Score From Baseline to Week 48

    Time frame: 48 weeks

    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. Therefore, the score ranges from 0 to 48 with higher scores representing better function.

  4. Change in Upright Vital Capacity From Baseline to Week 12

    Time frame: 12 weeks

    Change in Percent Predicted Upright Vital Capacity from Baseline to Week 12. A negative change indicates clinical worsening.

  5. Change in Upright Vital Capacity From Baseline to Week 24

    Time frame: 24 weeks

    Change in Percent Predicted Upright Vital Capacity from Baseline to Week 24. A negative change indicates clinical worsening.

  6. Change in Upright Vital Capacity From Baseline to Week 48

    Time frame: 48 weeks

    Change in Percent Predicted Upright Vital Capacity from Baseline to Week 48. A negative change indicates clinical worsening.

  7. Change in McGill Single-Item Scale (SIS) From Baseline to Week 12

    Time frame: 12 weeks

    The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.

  8. Change in McGill Single-Item Scale (SIS) From Baseline to Week 24

    Time frame: 24 weeks

    The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.

  9. Change in McGill Single-Item Scale (SIS) From Baseline to Week 48

    Time frame: 48 weeks

    The McGill SIS consists of a single question designed to assess the improvement of or the rate of deterioration of the subject's quality-of-life. Subjects rated their quality of life over the prior 2 days on a scale of 0 (very bad) to 10 (excellent). Decreases from Baseline indicate deterioration of a subject's quality of life.

  10. Number of Subjects With Feeding Tube Placed During the Study.

    Time frame: 144 weeks

    Number of participants who had a feeding tube placed during the study.

Sponsors and collaborators

Lead sponsor

Knopp Biosciences

Industry

Registry information

Official study title

An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Acronym: CL211

Important dates

Study start
2009
Primary completion
2013
Study completion
2013
First posted
Jul 2, 2009
Registry last updated
Aug 16, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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