Friedreich's AtaxiaNCT01921868
An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
A study testing Acetyl-L-Carnitine in people with Friedreich's Ataxia, run by University of South Florida at 1 site in United States.
Trial summary
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as difficulties with balance, walking, or upper arm function. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Friedreich's Ataxia
- What is being tested?
- Acetyl-L-Carnitine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Jun 2017
- Phase
- Interventional
- 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 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Outpatients with Friedreich's Ataxia diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient). Subject may be non-ambulatory.
- Age 18 years to 80 years.
- Stable medical condition for 3 months prior to screening.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- If on cardiac medications such as beta-blockers or ace inhibitors, patients must be on a stable dose for 6 months prior to study entry and for the duration of the study.
- Friedreich's Ataxia patients with systolic or diastolic dysfunction present on echocardiogram and ejection fraction between 35% - 65% at screening.
- Subjects with ejection fractions < 50% need to be stable and on optimal heart failure therapy for at least 2 weeks prior to screening.
Who cannot join
- Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
- Pregnancy or lactation.
- Concurrent participation in another clinical study where use of an investigational product is used. Subjects who are currently enrolled in the Friedreich's Ataxia Clinical Outcome Measures Study at any site will be allowed to enroll in this study as well.
- Any use of the investigational product within the past 30 days.
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 25).
- Legal incapacity or limited legal capacity.
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- History of stroke.
- Subjects with a history of thyroid disease (hypothyroidism). Clinical laboratory evaluations of thyroid stimulating hormone levels taken 3 months prior to the study or at screening will be used to confirm absence of current thyroid problems.
- Subjects with a history of seizures.
- Subjects taking warfarin or acenocoumarol.
- Presence of severe renal disease (estimated creatinine clearance <50 mL/min) or hepatic disease (AST or Alanine transaminase(ALT)>2x times normal) (as evidenced by labs reported within the past 6 months).
- Clinically significantly abnormal white blood cell, hemoglobin or platelet count (as evidenced by labs reported within the past 6 months).
- Subjects with blood work showing carnitine deficiency (<60nmol/mg total carnitine in the urine or <35umol/L total carnitine in the plasma).
- -
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of South Florida
Tampa, Florida, 33612
Tanya Aranca, B.S., 813-974-5909, taranca@health.usf.edu
Jessica Shaw, MPH, 813-974-5909, jshaw@health.usf.edu
About this study
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as difficulties with balance, walking, or upper arm function.
Read the detailed description
This study is an open label, pilot study of ALCAR in subjects with FA. In this study 20 patients with FA will receive ALCAR every day for 24 months. At the study endpoint, subjects will be assessed for changes in cardiovascular outcomes and FA symptoms. To determine the effects of LC on changes in cardiomyopathy, echocardiography with strain rate will be calculated. This technique has been validated in clinical studies and used in other studies of FA patients for the comparison of regional deformation and myocardial wall thickness.
- Conditions
- Friedreich's Ataxia
- Being tested
- Acetyl-L-Carnitine
- Medical subject headings
- Friedreich Ataxia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acetyl-L-CarnitineDrug
Also called ALCAR
Acetyl-L-Carnitine, 2 g/day, up to 24 months.
| Study group | Role | What they receive |
|---|---|---|
| Acetyl-L-Carnitine | Experimental | Open-label administration of Acetyl-L-Carnitine, up to 2 g/day for 24 months. Receives: Drug: Acetyl-L-Carnitine |
What is being measured
The primary outcome is the main question the study is designed to answer.
To observe the changes in cardiac functioning in patients with Friedreich's Ataxia between study endpoint and baseline.
Measured: Every 12 months, up to 24 months
To assess the changes in Friedreich's Ataxia symptoms and severity (as measured by clinical rating scales) compared to baseline.
Measured: Every 6 months, up to 24 months
Secondary outcomes (5)
Changes in patient global impression of improvement
Measured: Every 6 months, up to 24 months
Changes in Time 25-foot Walk
Measured: Every 6 months, up to 24 months
Changes in frequency and severity of adverse events
Measured: Every 6 months, up to 24 months
Changes in patient quality of life (SF-36)
Measured: Every 6 months, up to 24 months
Changes in clinical global impression of improvement
Measured: Every 6 months, up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Aug 2013 (estimated)
- Study startAug 2013
- Primary completionJun 2017 (estimated)
- Study completionJun 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Friedreich's AtaxiaStudies of the same condition, open and under way.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- 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
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Theresa Zesiewicz, University of South Florida
- Investigators
- Theresa A Zesiewicz, MD, Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01921868
- Sponsor's protocol ID
- ALCAR-8499
- Status last verified
- Apr 2016
- First submitted
- 3 Jul 2013
- Last updated
- 12 Apr 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label Study of the Effects of Acetyl-L-Carnitine on Cardiovascular Outcomes in Friedreich's Ataxia
- ClinicalTrials.gov ID
- NCT01921868
- Acronym / sponsor ID
- ALCAR-8499
- Conditions
- Friedreich's Ataxia
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Study start
- Aug 2013
- Primary completion
- Jun 2017 (estimated)
- Study completion
- Jun 2017 (estimated)
- First posted
- 13 Aug 2013 (estimated)
- 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.
The purpose of this study is to learn how treatment with acetyl-L-carnitine (ALCAR) will affect the hearts of patients with Friedreich's Ataxia as well as how it may affect other symptoms of Friedreich's Ataxia such as difficulties with balance, walking, or upper arm function.
Detailed description
This study is an open label, pilot study of ALCAR in subjects with FA. In this study 20 patients with FA will receive ALCAR every day for 24 months. At the study endpoint, subjects will be assessed for changes in cardiovascular outcomes and FA symptoms. To determine the effects of LC on changes in cardiomyopathy, echocardiography with strain rate will be calculated. This technique has been validated in clinical studies and used in other studies of FA patients for the comparison of regional deformation and myocardial wall thickness.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Acetyl-L-Carnitine | Experimental | Drug: Acetyl-L-CarnitineOpen-label administration of Acetyl-L-Carnitine, up to 2 g/day for 24 months. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Outpatients with Friedreich's Ataxia diagnosed by a movement disorder specialist and confirmed by genetic testing (of the patient or in a first degree relative of the patient). Subject may be non-ambulatory.
- Age 18 years to 80 years.
- Stable medical condition for 3 months prior to screening.
- Women of child-bearing potential must use a reliable method of contraception and must provide a negative pregnancy test at entry into the study.
- If on cardiac medications such as beta-blockers or ace inhibitors, patients must be on a stable dose for 6 months prior to study entry and for the duration of the study.
Key exclusion criteria (14 listed)
- Any unstable illness or concomitant medical condition that, in the investigator's opinion, precludes participation in this study. This includes other disorders that may affect gait or balance (stroke, arthritis, etc).
- Pregnancy or lactation.
- Concurrent participation in another clinical study where use of an investigational product is used. Subjects who are currently enrolled in the Friedreich's Ataxia Clinical Outcome Measures Study at any site will be allowed to enroll in this study as well.
- Any use of the investigational product within the past 30 days.
- Dementia or other psychiatric illness that prevents the patient from giving informed consent (Mini Mental Status Exam score less than 25).
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 |
|---|---|---|
| Acetyl-L-CarnitineAlso: ALCARAcetyl-L-Carnitine, 2 g/day, up to 24 months. | Drug | Acetyl-L-Carnitine |
Primary outcome
To observe the changes in cardiac functioning in patients with Friedreich's Ataxia between study endpoint and baseline.
Time frame: Every 12 months, up to 24 months
To assess the changes in Friedreich's Ataxia symptoms and severity (as measured by clinical rating scales) compared to baseline.
Time frame: Every 6 months, up to 24 months
5 secondary outcomes
Changes in patient global impression of improvement
Time frame: Every 6 months, up to 24 months
Changes in Time 25-foot Walk
Time frame: Every 6 months, up to 24 months
Changes in frequency and severity of adverse events
Time frame: Every 6 months, up to 24 months
Changes in patient quality of life (SF-36)
Time frame: Every 6 months, up to 24 months
Changes in clinical global impression of improvement
Time frame: Every 6 months, up to 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2016.
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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.
Theresa A Zesiewicz, MD principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Acetyl-L-Carnitine in people with Friedreich's Ataxia, run by University of South Florida 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 7 inclusion and 14 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: status not confirmed, as of the sponsor's last update on 12 Apr 2016. 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Tanya Aranca, BS (813-974-5909, taranca@health.usf.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To observe the changes in cardiac functioning in patients with Friedreich's Ataxia between study endpoint and baseline. (time frame: Every 12 months, up to 24 months). The study lists 2 primary outcomes in all. 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 12 Apr 2016.