Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL201)
This study has been completed.
Sponsor:
Knopp Neurosciences
Information provided by:
Knopp Neurosciences
ClinicalTrials.gov Identifier:
NCT00647296
First received: March 26, 2008
Last updated: February 7, 2011
Last verified: February 2011
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Purpose
This is a 2-part study of KNS-760704 in patients with ALS.
- Part 1 is a randomized, placebo-controlled, multi-center study to evaluate the safety, tolerability, and clinical effects of oral administration of 3 dosage levels of KNS 760704 vs. placebo for 12 weeks.
- Part 2 is 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 KNS-760704 for up to 76 weeks.
| Condition | Intervention | Phase |
|---|---|---|
|
Amyotrophic Lateral Sclerosis |
Drug: KNS-760704 Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS) |
Resource links provided by NLM:
Further study details as provided by Knopp Neurosciences:
Primary Outcome Measures:
- Number of subjects experiencing unacceptable safety or tolerability events after oral administration of KNS-760704 compared to placebo for 12 weeks in subjects with ALS (Part 1) [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
Secondary Outcome Measures:
- Number of subjects experiencing unacceptable safety or tolerability events after oral administration of KNS-760704 compared to placebo for up to 76 weeks in subjects with ALS (Part 2) [ Time Frame: 76 weeks ] [ Designated as safety issue: Yes ]
- Change from Baseline in ALSFRS-R [ Time Frame: 12 weeks (part 1) and 28 weeks (part 2) ] [ Designated as safety issue: Yes ]
- Change from baseline to Week 12 in upright and supine vital capacity [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
- Changes in cystatin C and neurofilament H [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
| Enrollment: | 102 |
| Study Start Date: | March 2008 |
| Study Completion Date: | October 2009 |
| Primary Completion Date: | September 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Placebo Comparator: matched placebo |
Drug: Placebo
Placebo oral tablet (2 tabs twice daily) for 12 weeks
|
| Experimental: low-dose KNS-760704 |
Drug: KNS-760704
Oral tablet 2 x 12.5 mg twice daily for 12 weeks
|
| Experimental: mid-dose KNS-760704 |
Drug: KNS-760704
Oral tablet 2 x 37.5 mg twice daily for 12 weeks
|
| Experimental: high-dose KNS-760704 |
Drug: KNS-760704
Oral tablet 2 x 75 mg twice daily for 12 weeks
|
Detailed Description:
This is a double-blind, randomized, placebo-controlled study evaluating the safety and efficacy of KNS-760704 compared to placebo. The study will be conducted in 2 parts.
In Part 1, approximately 80 eligible patients will be randomized to 1 of 4 treatment groups for 12 weeks of treatment: placebo; low-dose; mid-dose; or high-dose KNS-760704. Participants meeting eligibility requirements will be enrolled at approximately 20 centers in the U.S. In addition to the visit to determine eligibility and the first visit to take study drug, participants will be required to make 5 additional research clinic visits in Part 1.
Participants who complete all 12 weeks of Part 1 will be eligible for randomization to Part 2. The duration of Part 2 of the study is 76 weeks. Subjects will receive 1 of 2 active treatment groups for 72 weeks (low-dose or high-dose KNS-760704) and placebo for the remaining 4-week period in Part 2. Participants will not be told when the 4 weeks of placebo treatment will be given. During Part 2, participants will be required to make 12 research clinic visits, including the baseline visit.
Eligibility| Ages Eligible for Study: | 21 Years to 80 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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 VC > 65% of predicted for age, height, and gender
Exclusion Criteria:
- 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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00647296
Show 21 Study Locations
Show 21 Study LocationsSponsors and Collaborators
Knopp Neurosciences
Investigators
| Study Director: | Evan Ingersoll, Ph.D. | Knopp Neurosciences Inc. |
More Information
No publications provided
| Responsible Party: | Jim Mather / Sr. Director, Clinical Development, Knopp Neurosciences Inc. |
| ClinicalTrials.gov Identifier: | NCT00647296 History of Changes |
| Other Study ID Numbers: | KNS-760704-CL201 |
| Study First Received: | March 26, 2008 |
| Last Updated: | February 7, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Knopp Neurosciences:
|
ALS Amyotrophic Lateral Sclerosis Lou Gehrig Lou Gehrig's |
Lou Gehrig's disease Motor Neuron Disease Nervous System Diseases KNS-760704 |
Additional relevant MeSH terms:
|
Amyotrophic Lateral Sclerosis Sclerosis Motor Neuron Disease Spinal Cord Diseases Central Nervous System Diseases Nervous System Diseases Neurodegenerative Diseases TDP-43 Proteinopathies Neuromuscular Diseases Proteostasis Deficiencies Metabolic Diseases Pathologic Processes Pramipexol |
Antioxidants Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Protective Agents Physiological Effects of Drugs Antiparkinson Agents Anti-Dyskinesia Agents Central Nervous System Agents Therapeutic Uses Dopamine Agonists Dopamine Agents Neurotransmitter Agents |
ClinicalTrials.gov processed this record on May 22, 2013