Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)

NCT01091142 | Completed Phase 1

• 1 other identifier: NP001 Single Dose - ALS
• Study Type: interventional
• Target: 56
• Locations: 1 country
• Sites: 3

Brief Summary

Primary objectives: To assess the safety and tolerability of ascending doses of NP001 compared to placebo in subjects with ALS. Secondary objective: To explore the effects of NP001 on biomarkers potentially relevant to ALS.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability of NP001 compared to placebo in subjects with ALS

  6 mo.

Secondary Outcomes (1)

• To explore the effects of NP001 on biomarkers potentially relevant to ALS

  6 mo.

Study Arms (2)

NP001

EXPERIMENTAL

Drug: NP001

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

NP001 DRUG

Cohort 1: 0.3 mg/kg NP001(6:2 active:placebo) Cohort 2: 1.1 mg/kg NP001(6:2 active:placebo) Cohort 3: 2.1 mg/kg NP001(6:2 active:placebo) Cohort 4: 4.3 mg/kg NP001(6:2 active:placebo)

NP001

Placebo DRUG

Cohort 1: placebo Cohort 2: placebo Cohort 3: placebo Cohort 4: placebo

Placebo

Eligibility Criteria

• Age: 21 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males and females, age 21 years - 75 years
• Subjects must be in generally sound health as appropriate for their ALS diagnosis.
• Subjects must have a clinical diagnosis of laboratory-supported probable or definite ALS, according to modified El Escorial criteria.
• Subjects receiving riluzole must be on a stable dose for at least 30 days prior to enrollment.
• Women of childbearing age must be non-lactating and surgically sterile or using an effective method of birth control and have a negative pregnancy test prior to dosing with study medication.
• Subjects must understand the study and be willing to adhere to protocol requirements as evidenced by provision of written informed consent.
• Subject must be willing and able to give signed informed consent that has been approved by the Institutional Review Board (IRB).
• Subjects must be willing to have an intravenous infusion.
• Subjects must have suitable veins for IV access as determined by examination.

You may not qualify if:

• Subjects should not require nor are expected to require life sustaining interventions for the next six months or longer. (e.g. invasive ventilation).
• Subjects must not have:
• presence of a tracheotomy or invasive ventilation. Nocturnal non-invasive ventilation system (e.g. C-PAP) is allowed.
• a diagnosis of neurologic disease known to mimic the muscle atrophy or weakness seen in ALS including MS, muscular dystrophy, spinal stenosis, peripheral neuropathy, inherited neuropathies or neuromuscular diseases, foramen magnum or brainstem tumor, or toxic conditions.
• an active pulmonary disease under treatment including uncontrolled asthma, chronic obstructive pulmonary disease (pneumonia, bronchitis, etc.), pulmonary fibrosis, pulmonary infection in the last 2 months, or history of aspiration that may expose the subject to increased risk by participating in this trial as determined by the Investigator.
• a history of unstable medical illness in the 3 months prior to screening including any emergent hospitalizations.
• renal disease based on screening estimated creatinine clearance (eCcr) \< 50 mL/minute (Cockcroft Gault estimate using ideal body weight) where:
• evidence of elevated alanine aminotransferase greater than 3 times the upper limit of normal.
• evidence of anemia, thrombocytopenia, or neutropenia (screening hematocrit \<33%, platelet count \< lower limit of normal for the site laboratory, or neutrophil count less than 1,500/mm3).
• any condition that requires periodic red blood cell transfusions, erythropoietin or any blood dyscrasias undergoing active treatment in the past year.
• clinical laboratory parameters that are clinically significant in the opinion of the Investigator.
• systolic blood pressure in excess of 160 mmHg nor less than 100 mmHG or a diastolic blood pressure above 98 mmHg.
• a history of G6PD deficiency (Glucose-6-phosphate dehydrogenase deficiency) determined by subject report.
• a current history of hepatitis or HIV determined by subject report.
• Subjects must not be using systemic immunosuppressants including steroids and chemotherapeutic agents. Inhaled steroids, eye drops and local topical use are permitted with concurrence of Medical Monitor.

Sponsors & Collaborators

• Neuraltus Pharmaceuticals, Inc.: lead

Study Sites (3)

California Pacific Medical Center

San Francisco, California, 94115, United States

Location

University of Kansas Medical Center - Landon Center on Aging

Kansas City, Kansas, 66160, United States

Location

Department of Neurology; University of Kentucky Medical Center

Lexington, Kentucky, 40536, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Robert G. Miller, MD

  Forbes Norris ALS Treatment and Research, California Pacific Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY

Study Record Dates

• First Submitted: March 19, 2010
• First Posted: March 23, 2010
• Study Start: July 1, 2010
• Primary Completion: September 1, 2010
• Last Updated: October 6, 2010

Record last verified: 2010-10

Locations

US (3)