Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) (CL201)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Knopp Biosciences
ClinicalTrials.gov Identifier:
NCT00647296
First received: March 26, 2008
Last updated: August 8, 2013
Last verified: August 2013

March 26, 2008
August 8, 2013
March 2008
September 2009   (final data collection date for primary outcome measure)
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 ]
To evaluate the safety and tolerability of oral administration of 3 dosage levels of KNS-760704 compared to placebo for 12 weeks in patients with ALS (Part 1) [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
Complete list of historical versions of study NCT00647296 on ClinicalTrials.gov Archive Site
  • 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 ]
  • To evaluate the safety and tolerability of oral administration of 2 dosage levels of KNS-760704 for 28 weeks in ALS patients (Part 2) [ Time Frame: 28 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the effects of KNS-760704 on measures of clinical function [ Time Frame: 12 weeks and 28 weeks ] [ Designated as safety issue: Yes ]
  • To evaluate the reliability and clinical predictive value of upright and supine vital capacity [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • To evaluate the effects of KNS-760704 on selected protein levels in blood and cerebrospinal fluid [ Time Frame: 12 weeks and 28 weeks ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS)
A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)

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.

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.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Amyotrophic Lateral Sclerosis
  • Drug: KNS-760704
    Oral tablet 2 x 12.5 mg twice daily for 12 weeks
  • Drug: KNS-760704
    Oral tablet 2 x 37.5 mg twice daily for 12 weeks
  • Drug: KNS-760704
    Oral tablet 2 x 75 mg twice daily for 12 weeks
  • Drug: Placebo
    Placebo oral tablet (2 tabs twice daily) for 12 weeks
  • Placebo Comparator: matched placebo
    Intervention: Drug: Placebo
  • Experimental: low-dose KNS-760704
    Intervention: Drug: KNS-760704
  • Experimental: mid-dose KNS-760704
    Intervention: Drug: KNS-760704
  • Experimental: high-dose KNS-760704
    Intervention: Drug: KNS-760704
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
102
October 2009
September 2009   (final data collection date for primary outcome measure)

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
Both
21 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00647296
KNS-760704-CL201
No
Knopp Biosciences
Knopp Biosciences
Not Provided
Study Director: Evan Ingersoll, Ph.D. Knopp Neurosciences Inc.
Knopp Biosciences
August 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP