Safety and Efficacy Study of NP001 in Patients With Amyotrophic Lateral Sclerosis (ALS) and Systemic Inflammation

Neuraltus Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: Placebo; Drug: NP001

Study type: Interventional

Funder types: Industry

Identifiers

NCT02794857

NP001-10-003

Details and patient eligibility

About

This study evaluates NP001 in patients with amyotrophic lateral sclerosis (ALS) and evidence of systemic inflammation. Half of participants will receive NP001 and the other half will receive placebo.

Full description

This is a randomized, double-blind, placebo-controlled study of NP001 in subjects with ALS and evidence of elevated systemic inflammation. Subjects will be allocated (1:1) to NP001 and placebo. Drug or placebo will be given intravenously.

Enrollment: 138 patients

Sex: All

Ages: 21 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key Inclusion Criteria:

• Diagnosis of clinically possible, clinically probable (with or without laboratory support), or clinically definite ALS (using the revised El Escorial Criteria)
• Forced vital capacity greater than or equal to 65% of that predicted for age and height
• Onset of ALS-related weakness less than 3 years prior to first dose of study drug
• Plasma high sensitivity C-reactive protein (hs-CRP) concentration of greater than or equal to 0.113 mg/dL at pre-screening/screening
• Stable dose (greater than 30 days) 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

Key Exclusion Criteria:

• Life expectancy of less than 6 months
• Tracheotomy or be using ventilatory assistance, including Bi-level Positive Airway Pressure (BiPAP) or Continuous Positive Airway Pressure (CPAP)
• Active pulmonary disease
• Gastrostomy
• Stem cell therapy
• Immune modulator therapy or participation in studies of other agents within 12 weeks of pre-screening/screening
• Unstable medical condition other than ALS

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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138 participants in 2 patient groups, including a placebo group

NP001

Experimental group

Description:

NP001 2 mg/kg by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Treatment:

Drug: NP001

Placebo

Placebo Comparator group

Description:

Normal saline by intravenous administration for 5 consecutive days in Month 1 and for 3 consecutive days in Months 2 through 6

Treatment:

Drug: Placebo