Ignite Creation Date:
2024-05-06 @ 12:13 PM
Last Modification Date:
2024-10-26 @ 12:56 PM
Study NCT ID:
NCT03707795
Status:
COMPLETED
Last Update Posted:
2020-02-11
First Post:
2017-09-14
Brief Title:
Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
Sponsor:
Edward Kasaraskis
Organization:
University of Kentucky
Study Overview
Official Title:
Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
Status:
COMPLETED
Status Verified Date:
2020-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
TRANSLATE
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 smokingalcohol 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
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
Detailed Description:
This will be a single-blinded pharmacokinetic and pharmacodynamics study of intramuscular IM betamethasone in ALS patients and non-ALS relatives from families with a mutation in the FUS gene Participants remain blinded as to their genotype
Betamethasone is a FDA-approved drug and is only available in an IM dosing form Celestone This will be a proof-of-concept translational study built on discoveries made by our research team at the University of Kentucky All participants will receive active drug
The research procedures will be conducted at the University of Kentucky UK Albert B Chandler Hospital Pavilion H 800 Rose Street Lexington KY Participants will need to come to Center for Clinical and Translational Science CCTS on the 3rd Floor Room C300 of Pavilion H at UK Albert B Chandler Hospital for a ConsentScreening Visit If found eligible and qualify for the study participants will be asked to give voluntary written consent to participate
Following signing the consent form participants will be admitted to the UK Albert B Chandler Hospital CCTS Inpatient Unit 5th Floor 5 North Wing of the Hospital for 2 nights approximately 48 hours The following tests and procedures will take place
Neurological Exam
Vital Signs blood pressure heart rate and respiratory rate will be measured
Medical and medication history will be collected
Questionnaire
blood specimen for Pharmacokinetic and Pharmacodynamic will be collected testing
Participants will also be asked to do some testing of their breathing and physical abilities
The study drug betamethasone sodium phosphatebetamethasone acetate Celestone Soluspan will be injected into a muscle such as arm or buttock - this will be the first of the four injections of the study drug administered during the study
At 24 hours after the first injection with the study drug and while still in the the hospital the study drug will again be injected into a muscle such as arm or buttock - this will be the second of the four injections of the study drug administered during the study
Following this second treatment and after the study doctor determines it is safe the participant will be discharged from the hospital They will will stay in a local motel overnight awaiting the next days blood draw assessments testing and 3rd treatment with study drug
Participants who do not live close to the hospital motel accommodations will be provided at no charge for an overnight stay between the 48 Hour visit and the 72 Hour scheduled follow-up visit
At 72 hours after first injection with the study drug the study drug will again be injected into a muscle such as arm or buttock - this will be the fourth and last of the four injections administered during the study Following this fourth treatment and after the study doctor determines if is safe participants will be allowed to leave
The 72 Hour Day 3 168 Hour Day 7 and 336 Hour Day 14 visits will take about 1 hour each The approximate total amount of time participants will be asked to volunteer for this study is 51 hours over the 336 hour 14 day duration of this research study
Participants or their insurance company Medicare or Medicaid will be responsible for the costs of all routine medical care and treatment they would normally receive for their condition The University of Kentucky may not be allowed to bill insurance companies Medicare or Medicaid for the medical procedures done strictly for research
Neither the participant or their provider will be charged for costs of any of the procedures performed for the research study
Betamethasone is a FDA-approved drug and is only available in an IM dosing form Celestone This will be a proof-of-concept translational study built on discoveries made by our research team at the University of Kentucky All participants will receive active drug
The research procedures will be conducted at the University of Kentucky UK Albert B Chandler Hospital Pavilion H 800 Rose Street Lexington KY Participants will need to come to Center for Clinical and Translational Science CCTS on the 3rd Floor Room C300 of Pavilion H at UK Albert B Chandler Hospital for a ConsentScreening Visit If found eligible and qualify for the study participants will be asked to give voluntary written consent to participate
Following signing the consent form participants will be admitted to the UK Albert B Chandler Hospital CCTS Inpatient Unit 5th Floor 5 North Wing of the Hospital for 2 nights approximately 48 hours The following tests and procedures will take place
Neurological Exam
Vital Signs blood pressure heart rate and respiratory rate will be measured
Medical and medication history will be collected
Questionnaire
blood specimen for Pharmacokinetic and Pharmacodynamic will be collected testing
Participants will also be asked to do some testing of their breathing and physical abilities
The study drug betamethasone sodium phosphatebetamethasone acetate Celestone Soluspan will be injected into a muscle such as arm or buttock - this will be the first of the four injections of the study drug administered during the study
At 24 hours after the first injection with the study drug and while still in the the hospital the study drug will again be injected into a muscle such as arm or buttock - this will be the second of the four injections of the study drug administered during the study
Following this second treatment and after the study doctor determines it is safe the participant will be discharged from the hospital They will will stay in a local motel overnight awaiting the next days blood draw assessments testing and 3rd treatment with study drug
Participants who do not live close to the hospital motel accommodations will be provided at no charge for an overnight stay between the 48 Hour visit and the 72 Hour scheduled follow-up visit
At 72 hours after first injection with the study drug the study drug will again be injected into a muscle such as arm or buttock - this will be the fourth and last of the four injections administered during the study Following this fourth treatment and after the study doctor determines if is safe participants will be allowed to leave
The 72 Hour Day 3 168 Hour Day 7 and 336 Hour Day 14 visits will take about 1 hour each The approximate total amount of time participants will be asked to volunteer for this study is 51 hours over the 336 hour 14 day duration of this research study
Participants or their insurance company Medicare or Medicaid will be responsible for the costs of all routine medical care and treatment they would normally receive for their condition The University of Kentucky may not be allowed to bill insurance companies Medicare or Medicaid for the medical procedures done strictly for research
Neither the participant or their provider will be charged for costs of any of the procedures performed for the research study
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
None