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

• Register: ClinicalTrials.gov
• Last refreshed on: 8 January 2018
• Main ID: NCT00647296
• Date of registration: 26/03/2008
• Prospective Registration: No
• Primary sponsor: Knopp Biosciences
• Public title: Safety and Tolerability Study of KNS-760704 in Amyotrophic Lateral Sclerosis (ALS) CL201
• Scientific title: A 2-Part, Randomized, Double-Blind, Safety and Tolerability Study Evaluating KNS-760704 in Patients With Amyotrophic Lateral Sclerosis (ALS)
• Date of first enrolment: March 2008
• Target sample size: 102
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00647296
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Evan Ingersoll, Ph.D.
• Affiliation: Knopp Neurosciences Inc.

Key inclusion & exclusion 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



Age minimum: 21 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Amyotrophic Lateral Sclerosis

Intervention(s)

Drug: KNS-760704

Drug: Placebo

Primary Outcome(s)

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]

Secondary Outcome(s)

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]

Change from baseline to Week 12 in upright and supine vital capacity [Time Frame: 12 weeks]

Changes in cystatin C and neurofilament H [Time Frame: 12 weeks]

Change from Baseline in ALSFRS-R [Time Frame: 12 weeks (part 1) and 28 weeks (part 2)]

Secondary ID(s)

KNS-760704-CL201

Source(s) of Monetary Support

Please refer to primary and secondary sponsors