Clinical trial NCT01281631: A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Official title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Lead sponsor: Neuraltus Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2011-01-24
Enrollment: 136
Sex: ALL
Minimum age (years): 21
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 2 randomized, double-blind, placebo-controlled, multicenter study of NP001 in subjects with ALS.

Detailed description: This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS conducted in multiple centers. Drug or placebo will be given intravenously. Approximately 105 subjects will be enrolled. Subjects will be allocated (1:1:1) to placebo and 2 dose levels of NP001.

Eligibility criteria: Subjects with sporadic or familial ALS classified as definite, probable, or laboratory-supported probable ALS according to the revised El Escorial criteria. A list of key criteria is listed below:

Inclusion Criteria:

* Onset of symptoms less than 3 years prior to study entry.
* Forced Vital Capacity (FVC) at least 70% of that predicted for age and height.
* Stable dose of riluzole if undergoing treatment with this agent.
* For females: Not be of childbearing potential or agree to use adequate birth control during the study.

Exclusion Criteria:

* Unstable medical condition(s) other than ALS.
* Life expectancy of less than 6 months.
* Require life-sustaining interventions for the 6 months following randomization.
* Have a tracheotomy or be using ventilatory assistance \[including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)\].
* Active pulmonary disease.
* Immune modulator therapy within 12 weeks of study entry or participation in studies of other agents within the last 4 weeks prior to the randomization.

Primary outcomes:
measure: To evaluate the effects of NP001 on measures of clinical function
timeFrame: over 9 months
Secondary outcomes:
measure: Safety and tolerability in ALS
timeFrame: duration of study
measure: Pulmonary function and biomarkers
timeFrame: over 9 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic, Scottsdale
state: Arizona
city: Irvine
country: United States
facility: UC, Irvine
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: San Francisco
country: United States
facility: California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic, Jacksonville
state: Florida
city: Atlanta
country: United States
facility: The Emory Clinic
state: Georgia
city: Kansas City
country: United States
facility: University of Kansas Medical Center, Landon Center on Aging
state: Kansas
city: Lexington
country: United States
facility: University of Kentucky, Department of Neurology
state: Kentucky
city: Charlestown
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: New York
country: United States
facility: Columbia University
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University, Syracuse
state: New York
city: Charlotte
country: United States
facility: Carolinas Medical Center
state: North Carolina
city: Durham
country: United States
facility: Duke University, Dept of Neurology
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: Providence ALS Center
state: Oregon
city: Houston
country: United States
facility: Methodist Hospital Research Institute, Methodist Neurologic Institute
state: Texas
city: Centralia
country: United States
facility: Providence Saint Peter Hospital
state: Washington
Study officials:
affiliation: Forbes Norris ALS Treatment and Research, California Pacific Medical Center
name: Robert G. Miller, MD
role: PRINCIPAL_INVESTIGATOR
References: