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Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study (TRANSLATE)

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ClinicalTrials.gov Identifier: NCT03707795
Recruitment Status : Recruiting
First Posted : October 16, 2018
Last Update Posted : March 13, 2019
Sponsor:
Information provided by (Responsible Party):
Edward Kasaraskis, University of Kentucky

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.

Condition or disease Intervention/treatment Phase
Familial Amyotrophic Lateral Sclerosis Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days Early Phase 1

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: Single (Participant)
Masking Description: Participants remain blinded as to their genotype.
Primary Purpose: Treatment
Official Title: Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
Actual Study Start Date : August 21, 2017
Estimated Primary Completion Date : November 2019
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Experimental: Arm 1 - Amyotrophic Lateral Sclerosis
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
Participants will be given four IM injections throughout the study

Active Comparator: Arm 2 - Familial Amyotrophic Lateral Sclerosis
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
Participants will be given four IM injections throughout the study




Primary Outcome Measures :
  1. Betamethasone plasma levels [ Time Frame: 14 days ]
    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.


Secondary Outcome Measures :
  1. Protein carbonyl plasma levels [ Time Frame: 14 days ]
    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Protein carbonyl levels. Data will be reported as the change in Protein carbonyl over time.

  2. Superoxide dismutase plasma levels [ Time Frame: 14 days ]
    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Superoxide dismutase levels. Data will be reported as the change in Superoxide dismutase over time.

  3. Peroxide plasma levels [ Time Frame: 14 days ]
    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Peroxide levels. Data will be reported as the change in Peroxide over time.

  4. Glutathione disulfide plasma levels [ Time Frame: 14 days ]
    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Glutathione disulfide levels. Data will be reported as the change in Glutathione disulfide over time.

  5. Glutathione plasma levels [ Time Frame: 14 days ]
    Blood will be collected at multiple time points (baseline, 24 hours, 48 hours, 72 hours, day 7 and day 14) for analysis of Glutathione disulfide levels. Data will be reported as the change in Glutathione disulfide over time.


Other Outcome Measures:
  1. Slow vital capacity [ Time Frame: 14 days ]
    Participants will blow into a spirometer at a natural rate of exhale to measure the vital capacity of their respratory system. Data will be reported as the change in vital capacity over time.

  2. Maximum voluntary ventilation (MVV) [ Time Frame: 14 days ]
    Participants will inhale as deeply and quickly as possible into a spirometerr to over the course of 15 seconds to assess respiratory function. Data will be reported as the change in MMV over time.

  3. Grip strength [ Time Frame: 14 days ]
    Participants will squeeze a dynamometer which will measure the maximal force generated in mmHG. Data will be reported as the change in grip strength over time.

  4. Manual dexterity [ Time Frame: 14 days ]
    Participants will have their gross movements of their arms, hands and fingers and their fine motor capacities measured using the Purdue Pegboard assessment. The test involves the placement of small pegs in a board at varying ranges of extremity extension. Participants are scored on the number of pegs they can place in a 30 second assessment. Data are presented as the change in number of pegs placed over time.

  5. Sit to stand assessment [ Time Frame: 14 days ]
    Participants will be timed in their ability to rise from a seated position to standing fully upright. Data are presented as the change in time to rise over time.

  6. Time walk assessment [ Time Frame: 14 days ]
    Participants will be time in their ability to walk 20 feet. Data are presented as the change in time to cover the distance over time.

  7. Isometric strength generation [ Time Frame: 14 days ]
    Utilizing an Accurate Test of Limb Isometric Strength (ATLIS) participants will their static isometric limb strength measured. Data are presented as the change in limb strength over time.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   20 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

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

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03707795


Contacts
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Contact: Meghann Bruno, RN 859-218-5064 mebrun3@uky.edu
Contact: Meha Joshi 859-218-5046 majo235@uky.edu

Locations
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United States, Kentucky
University of Kentucky Medical Center Recruiting
Lexington, Kentucky, United States, 40475
Contact: Meha Joshi    859-218-5046    majo235@uky.edu   
Contact: Meghann Bruno, RN    859-218-5064    mebrun3@uky.edu   
Principal Investigator: Edward Kasarskis, MD         
Sponsors and Collaborators
University of Kentucky
Investigators
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Principal Investigator: Edward Kasarskis, MD University of Kentucky

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Responsible Party: Edward Kasaraskis, Principal Investigator, University of Kentucky
ClinicalTrials.gov Identifier: NCT03707795     History of Changes
Other Study ID Numbers: 17-0159-F6A
First Posted: October 16, 2018    Key Record Dates
Last Update Posted: March 13, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No

Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Betamethasone
Betamethasone Valerate
Betamethasone-17,21-dipropionate
Betamethasone benzoate
Betamethasone acetate
Betamethasone sodium phosphate
Betamethasone acetate phosphate
Anti-Inflammatory Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents