NCT01281631

Brief Summary

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
136

participants targeted

Target at P75+ for phase_2

Timeline
Completed

Started Feb 2011

Geographic Reach
1 country

17 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

January 17, 2011

Completed
7 days until next milestone

First Posted

Study publicly available on registry

January 24, 2011

Completed
8 days until next milestone

Study Start

First participant enrolled

February 1, 2011

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2012

Completed
Last Updated

November 22, 2012

Status Verified

November 1, 2012

Enrollment Period

1.6 years

First QC Date

January 17, 2011

Last Update Submit

November 20, 2012

Conditions

Keywords

ALS

Outcome Measures

Primary Outcomes (1)

  • To evaluate the effects of NP001 on measures of clinical function

    over 9 months

Secondary Outcomes (2)

  • Safety and tolerability in ALS

    duration of study

  • Pulmonary function and biomarkers

    over 9 months

Study Arms (3)

Low dose NP001

EXPERIMENTAL

Low drug dose

Drug: NP001

High dose NP001

EXPERIMENTAL

High drug dose

Drug: NP001

Placebo

PLACEBO COMPARATOR

normal saline

Drug: Placebo

Interventions

NP001DRUG

Low dose of NP001

Low dose NP001

normal saline

Placebo

Eligibility Criteria

Age21 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (17)

Mayo Clinic, Scottsdale

Scottsdale, Arizona, 85259, United States

Location

UC, Irvine

Irvine, California, 92868-4281, United States

Location

UCLA

Los Angeles, California, 90095, United States

Location

California Pacific Med Center Forbes Norris MDA/ALS Research and Treatment Center

San Francisco, California, 94115, United States

Location

Mayo Clinic, Jacksonville

Jacksonville, Florida, 32224, United States

Location

The Emory Clinic

Atlanta, Georgia, 30322, United States

Location

University of Kansas Medical Center, Landon Center on Aging

Kansas City, Kansas, 66160, United States

Location

University of Kentucky, Department of Neurology

Lexington, Kentucky, 40536-0284, United States

Location

Massachusetts General Hospital

Charlestown, Massachusetts, 02129, United States

Location

Columbia University

New York, New York, 10032, United States

Location

SUNY Upstate Medical University, Syracuse

Syracuse, New York, 13210, United States

Location

Carolinas Medical Center

Charlotte, North Carolina, 28207, United States

Location

Duke University, Dept of Neurology

Durham, North Carolina, 27705, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Providence ALS Center

Portland, Oregon, 97213, United States

Location

Methodist Hospital Research Institute, Methodist Neurologic Institute

Houston, Texas, 77030, United States

Location

Providence Saint Peter Hospital

Centralia, Washington, 98531, United States

Location

Related Publications (1)

  • Forrest BD, Goyal NA, Fleming TR, Bracci PM, Brett NR, Khan Z, Robinson M, Azhir A, McGrath M. The Effectiveness of NP001 on the Long-Term Survival of Patients with Amyotrophic Lateral Sclerosis. Biomedicines. 2024 Oct 16;12(10):2367. doi: 10.3390/biomedicines12102367.

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional 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 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

January 17, 2011

First Posted

January 24, 2011

Study Start

February 1, 2011

Primary Completion

September 1, 2012

Study Completion

September 1, 2012

Last Updated

November 22, 2012

Record last verified: 2012-11

Locations