NCT00107770

Brief Summary

The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
40

participants targeted

Target at P50-P75 for phase_1

Timeline
Completed

Started Apr 2005

Typical duration for phase_1

Geographic Reach
1 country

10 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

Study Start

First participant enrolled

April 1, 2005

Completed
6 days until next milestone

First Submitted

Initial submission to the registry

April 7, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

April 8, 2005

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2006

Completed
10 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2007

Completed
Last Updated

January 11, 2010

Status Verified

October 1, 2008

Enrollment Period

1.6 years

First QC Date

April 7, 2005

Last Update Submit

January 8, 2010

Conditions

Keywords

ALShistone deacetylase inhibitionopen labelsafety studysodium phenylbutyrate

Outcome Measures

Primary Outcomes (1)

  • safety and tolerability

    20 weeks

Secondary Outcomes (2)

  • The number of side effects at each dosage, including abnormalities in vital signs, physical examination, blood tests and EKGs, change in vital capacity (breathing function) and ALS functional rating scale

  • Relationship between blood levels and sodium phenylbutyrate dosage

Study Arms (1)

1

OTHER

ALS patient

Drug: sodium phenylbutyrate

Interventions

histone deacteylase inhibitor

1

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosed with ALS
  • At least 18 years of age
  • Women, who can become pregnant, must actively use effective birth control measures

You may not qualify if:

  • Must not have any other neurological (nervous system) disease
  • Veterans only are eligible to participate at VA sites.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (10)

VA Medical Center, Iowa City

Iowa City, Iowa, 52246-2208, United States

Location

VA Medical Center, Lexington

Lexington, Kentucky, 40502, United States

Location

VA Maryland Health Care System, Baltimore

Baltimore, Maryland, 21201, United States

Location

Edith Nourse Rogers Memorial Veterans Hospital, Bedford

Bedford, Massachusetts, 01730, United States

Location

VA Medical Center, Jamaica Plain Campus

Boston, Massachusetts, 02130, United States

Location

VA Medical Center, Syracuse

Syracuse, New York, 13210, United States

Location

VA Medical Center, Durham

Durham, North Carolina, 27705, United States

Location

VA Medical Center, Cincinnati

Cincinnati, Ohio, 45220, United States

Location

VA Pittsburgh Health Care System

Pittsburgh, Pennsylvania, 15240, United States

Location

Michael E. DeBakey VA Medical Center (152)

Houston, Texas, 77030, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

4-phenylbutyric acid

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 Ferrante, PhD MSc

    Edith Nourse Rogers Memorial Veterans Hospital, Bedford

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
FED

Study Record Dates

First Submitted

April 7, 2005

First Posted

April 8, 2005

Study Start

April 1, 2005

Primary Completion

November 1, 2006

Study Completion

September 1, 2007

Last Updated

January 11, 2010

Record last verified: 2008-10

Locations