Familial Amyotrophic Lateral SclerosisEarly Phase 1TRANSLATENCT03707795
Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
A Early Phase 1 study testing Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days in people with Familial Amyotrophic Lateral Sclerosis, run by Edward Kasaraskis at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Familial Amyotrophic Lateral Sclerosis
- What is being tested?
- Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 21 Aug 2017to 10 Jan 2019
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of familial ALS (fALS)
- Relative of a fALS person and carry the FUS gene
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Kentucky Medical Center
Lexington, Kentucky, 40475
About this study
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).
Read more
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.
Read the 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 Consent/Screening 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 phosphate/betamethasone 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 day's 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.
- Conditions
- Familial Amyotrophic Lateral Sclerosis
- Being tested
- Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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
| Study group | Role | What they receive |
|---|---|---|
| Arm 1 - Amyotrophic Lateral Sclerosis | Experimental | Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days Receives: 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 Receives: Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 14 days
Secondary outcomes (5)
Protein carbonyl 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 Protein carbonyl levels. Data will be reported as the change in Protein carbonyl over time.
Measured: 14 days
Superoxide dismutase 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 Superoxide dismutase levels. Data will be reported as the change in Superoxide dismutase over time.
Measured: 14 days
Peroxide 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 Peroxide levels. Data will be reported as the change in Peroxide over time.
Measured: 14 days
Glutathione disulfide 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 Glutathione disulfide levels. Data will be reported as the change in Glutathione disulfide over time.
Measured: 14 days
Glutathione 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 Glutathione disulfide levels. Data will be reported as the change in Glutathione disulfide over time.
Measured: 14 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Oct 2018
- Study start21 Aug 2017
- Primary completion10 Jan 2019
- Study completion10 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Familial Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Edward Kasaraskis
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Edward Kasaraskis, University of Kentucky
- Investigators
- Edward Kasarskis, MD, Principal investigator, University of Kentucky
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03707795
- Sponsor's protocol ID
- 17-0159-F6A
- Acronym
- TRANSLATE
- Status last verified
- Feb 2020
- First submitted
- 14 Sep 2017
- Last updated
- 11 Feb 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of FUS-Related ALS With Betamethasone - The TRANSLATE Study
- ClinicalTrials.gov ID
- NCT03707795
- Acronym / sponsor ID
- TRANSLATE / 17-0159-F6A
- Conditions
- Familial Amyotrophic Lateral Sclerosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Edward KasaraskisUniversity, hospital or other
- Study start
- 21 Aug 2017 (actual)
- Primary completion
- 10 Jan 2019 (actual)
- Study completion
- 10 Jan 2019 (actual)
- First posted
- 16 Oct 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 Consent/Screening 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 phosphate/betamethasone 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 day's 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 design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1 - Amyotrophic Lateral Sclerosis | Experimental | Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four daysBetamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days |
| Arm 2 - Familial Amyotrophic Lateral Sclerosis | Active comparator | Drug: Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four daysBetamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Diagnosis of familial ALS (fALS)
- Relative of a fALS person and carry the FUS gene
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four daysParticipants will be given four IM injections throughout the study | Drug | Arm 1 - Amyotrophic Lateral Sclerosis, Arm 2 - Familial Amyotrophic Lateral Sclerosis |
Primary outcome
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.
5 secondary outcomes
Protein carbonyl plasma levels
Time frame: 14 days
Superoxide dismutase plasma levels
Time frame: 14 days
Peroxide plasma levels
Time frame: 14 days
Glutathione disulfide plasma levels
Time frame: 14 days
Glutathione plasma levels
Time frame: 14 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Feb 2020.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Edward Kasarskis, MD principal investigator
University of Kentucky
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Betamethasone sodium phosphate/betamethasone acetate (Celestone® Soluspan®), 30 mg IM once a day for four days in people with Familial Amyotrophic Lateral Sclerosis, run by Edward Kasaraskis at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Feb 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Betamethasone plasma levels (time frame: 14 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Feb 2020.