A Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

A Phase 2 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01281631

Enrollment

136

Registered

2011-01-24

Start date

2011-02-28

Completion date

2012-09-30

Last updated

2012-11-22

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS

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.

Interventions

DRUGNP001

Low dose of NP001

DRUGPlacebo

normal saline

Study design

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Masking

QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender

ALL

Age

21 Years to 80 Years

Healthy volunteers

Inclusion 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.

Design outcomes

Primary

Measure	Time frame
To evaluate the effects of NP001 on measures of clinical function	over 9 months

Secondary

Measure	Time frame
Safety and tolerability in ALS	duration of study
Pulmonary function and biomarkers	over 9 months

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 5, 2026