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

Neuraltus Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: NP001

Study type: Interventional

Funder types: Industry

Identifiers

NCT01281631

NP001-10-002

Details and patient eligibility

About

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

Full 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.

Enrollment: 136 patients

Sex: All

Ages: 21 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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136 participants in 3 patient groups, including a placebo group

Low dose NP001

Experimental group

Description:

Low drug dose

Treatment:

Drug: NP001

Drug: NP001

High dose NP001

Experimental group

Description:

High drug dose

Treatment:

Drug: NP001

Drug: NP001

Placebo

Placebo Comparator group

Description:

normal saline

Treatment:

Drug: Placebo