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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01091142
Recruitment Status : Completed
First Posted : March 23, 2010
Last Update Posted : October 6, 2010
Sponsor:
Information provided by:
Neuraltus Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE March 19, 2010
First Posted Date  ICMJE March 23, 2010
Last Update Posted Date October 6, 2010
Study Start Date  ICMJE July 2010
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 22, 2010)
Safety and tolerability of NP001 compared to placebo in subjects with ALS [ Time Frame: 6 mo. ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 22, 2010)
To explore the effects of NP001 on biomarkers potentially relevant to ALS [ Time Frame: 6 mo. ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Single-Ascending-Dose Safety/Tolerability of NP001 in Amyotrophic Lateral Sclerosis (ALS)
Official Title  ICMJE Single-Ascending-Dose Safety and Tolerability Study of NP001 in Subjects With Amyotrophic Lateral Sclerosis (ALS)
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.

Detailed Description This study is a double-blind, placebo-controlled single ascending dose safety and tolerability study. Approximately 32-56 subjects with clinical diagnosis of ALS according to modified El Escorial criteria are planned to receive a single dose of study drug, NP001.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Amyotrophic Lateral Sclerosis
Intervention  ICMJE
  • Drug: NP001
    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)
  • Drug: Placebo
    Cohort 1: placebo Cohort 2: placebo Cohort 3: placebo Cohort 4: placebo
Study Arms  ICMJE
  • Experimental: NP001
    Intervention: Drug: NP001
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Estimated Enrollment  ICMJE
 (submitted: July 19, 2010)
56
Original Estimated Enrollment  ICMJE
 (submitted: March 22, 2010)
42
Study Completion Date  ICMJE Not Provided
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.
  • Subjects must not have a hematologic disorder such as autoimmune anemia, or hemolytic anemia of any type including paroxysmal nocturnal hemoglobinuria or myoglobinuria.
  • Subjects must not have a history of unexplained jaundice determined by subject report.
  • Subjects must not have received IV Immunoglobulin (IG) within 30 days of the planned initial dose of study drug.
  • Subjects must not be participating in another drug study or have participated in a drug study within the last 30 days prior to enrollment. Observational trials with no intervention are acceptable provided permission for the other study Sponsor is obtained in writing.
  • Subjects must not have any other condition which in the Investigator's opinion would put the subject at risk by participating in this study.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 21 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01091142
Other Study ID Numbers  ICMJE NP001 Single Dose - ALS
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Nancy E. Isaac/VP Regulatory Affairs, Neuraltus Pharmaceuticals, Inc.
Study Sponsor  ICMJE Neuraltus Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Robert G. Miller, MD Forbes Norris ALS Treatment and Research, California Pacific Medical Center
PRS Account Neuraltus Pharmaceuticals, Inc.
Verification Date October 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP