NCT03707795

Brief Summary

By doing this study the investigator hopes to learn more about a potential cause of amyotrophic lateral sclerosis (ALS) called "oxidative stress". Oxidative stress is essentially an imbalance between the production of certain chemicals in the body called "free radicals" and the ability of the body to counteract or detoxify their harmful effects through neutralization by antioxidants. It is thought that factors such as environmental exposure (chemicals and lead), diet, smoking,alcohol consumption, physical activity and psychological stress cause oxidative stress to occur inside the body. By doing this study, the investigator hopes to learn whether the FDA-approved steroid medication called Betamethasone will restore overall antioxidant activity fALS patients with mutations in the Fused in Sarcoma gene (FUS gene). Participants who agree to take part in this research study, agree to the following responsibilities:

  • Attend all scheduled visits
  • Notify the study doctor of any illnesses, unexpected or troublesome side effects, or any other medical problems that occur during the study
  • Be completely honest with their answers to all questions
  • Check with the study doctor before taking any new medications, whether prescribed or "over the counter," even vitamins and herbal supplements.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
6

participants targeted

Target at below P25 for early_phase_1

Timeline
Completed

Started Aug 2017

Geographic Reach
1 country

1 active site

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

August 21, 2017

Completed
24 days until next milestone

First Submitted

Initial submission to the registry

September 14, 2017

Completed
1.1 years until next milestone

First Posted

Study publicly available on registry

October 16, 2018

Completed
3 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 10, 2019

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 10, 2019

Completed
Last Updated

February 11, 2020

Status Verified

February 1, 2020

Enrollment Period

1.4 years

First QC Date

September 14, 2017

Last Update Submit

February 9, 2020

Conditions

Familial Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

  • Betamethasone plasma levels

    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Betamethasone levels. Data will be reported as the change in Betamethasone over time.

    14 days

Secondary Outcomes (5)

  • Protein carbonyl plasma levels

    14 days

  • Superoxide dismutase plasma levels

    14 days

  • Peroxide plasma levels

    14 days

  • Glutathione disulfide plasma levels

    14 days

  • Glutathione plasma levels

    14 days

Other Outcomes (7)

  • Slow vital capacity

    14 days

  • Maximum voluntary ventilation (MVV)

    14 days

  • Grip strength

    14 days

  • +4 more other outcomes

Study Arms (2)

Arm 1 - Amyotrophic Lateral Sclerosis

EXPERIMENTAL

Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days

Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days

Arm 2 - Familial Amyotrophic Lateral Sclerosis

ACTIVE COMPARATOR

Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days

Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days

Interventions

Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four daysDRUG

Participants will be given four IM injections throughout the study

Arm 1 - Amyotrophic Lateral SclerosisArm 2 - Familial Amyotrophic Lateral Sclerosis

Eligibility Criteria

Age20 Years - 80 Years
Sexall(Gender-based eligibility)
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Diagnosis of familial ALS (fALS)
  • Relative of a fALS person and carry the FUS gene

You may not qualify if:

  • Under 20 years or over 80 years of age
  • Cannot tolerate steroids, including betamethasone
  • Are unwilling or unable to attend all scheduled research visits
  • Currently participating in another clinical drug trial
  • Major neurological disease, other than ALS
  • Pregnant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Kentucky Medical Center

Lexington, Kentucky, 40475, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

betamethasone sodium phosphatebetamethasone acetate

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesTDP-43 ProteinopathiesNeuromuscular DiseasesProteostasis DeficienciesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Edward Kasarskis, MD

    University of Kentucky

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
early phase 1
Allocation
NON RANDOMIZED
Masking
SINGLE
Who Masked
PARTICIPANT
Masking Details
Participants remain blinded as to their genotype.
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

September 14, 2017

First Posted

October 16, 2018

Study Start

August 21, 2017

Primary Completion

January 10, 2019

Study Completion

January 10, 2019

Last Updated

February 11, 2020

Record last verified: 2020-02

Data Sharing

IPD Sharing
Will not share

Locations

US(1)