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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00931944
Recruitment Status : Completed
First Posted : July 2, 2009
Results First Posted : August 16, 2021
Last Update Posted : August 16, 2021
Sponsor:
Information provided by (Responsible Party):
Knopp Biosciences

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: KNS-760704 Phase 2

Detailed Description:

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.

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 74 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label, Safety and Tolerability, Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
Study Start Date : July 2009
Actual Primary Completion Date : July 2013
Actual Study Completion Date : July 2013

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: KNS-760704 300 mg/day
Open-label KNS-760704 (150 mg Q12H)
 Drug: KNS-760704
150 mg Q12H KNS-760704 given orally (300 mg total daily dose)
Other Name: dexpramipexole


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   21 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Patient has provided signed informed consent for this trial before the commencement of any study-related procedure
  2. 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:

  1. Patient did not participate in Knopp Protocol KNS-760704-CL201 or patient did not receive RTPB under Research IND #60,948.
  2. Patient discontinued from KNS-760704-CL201 for any reason other than enrollment into this study (applies to patients enrolled in KNS-760704-CL201 only)
  3. 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)
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00931944


Locations
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Sponsors and Collaborators
Knopp Biosciences
Investigators
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Principal Investigator: Merit Cudkowicz, M.D., MSc NeuroClinical Trials Unit (Massachusetts General Hospital)
More Information
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Responsible Party: Knopp Biosciences
ClinicalTrials.gov Identifier: NCT00931944    
Other Study ID Numbers: KNS-760704-CL211
First Posted: July 2, 2009    Key Record Dates
Results First Posted: August 16, 2021
Last Update Posted: August 16, 2021
Last Verified: July 2021
Keywords provided by Knopp Biosciences:
ALS
Amyotrophic Lateral Sclerosis
Lou Gehrig
Lou Gehrig's
Lou Gehrig's disease
 Motor Neuron Disease
Nervous System Diseases
KNS-760704
RTPB
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
 Metabolic Diseases
Pramipexole
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antiparkinson Agents
Anti-Dyskinesia Agents
Dopamine Agonists
Dopamine Agents
Neurotransmitter Agents