Ataxia-oculomotor Apraxia 1Phase 3AOA1NCT02333305
Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10
A Phase 3 study testing CoQ10 in people with Ataxia-oculomotor Apraxia 1, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial summary
We propose a study on Ataxia with oculomotor apraxia type 1 (AOA1) in which Coenzyme Q10 (CoQ10) deficit has been observed. Main objectives of the study are : To monitor evolution of albumin in patients affected with AOA1 while supplemented with CoQ10 ; To measure with clinical scales and biological markers efficacy… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ataxia-oculomotor Apraxia 1
- What is being tested?
- CoQ10
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 19 participants
- Where
- 1 siteFrance
- Study dates
- Jun 2013to Sep 2017
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 1. Diagnosis of ataxia with oculomotor apraxia type I (AOA1) confirmed by genetic molecular analysis
- 2. Age ≥ 18 years
- 3. Hypoalbuminemia
- 4. Efficient contraception for women of childbearing potential (with pregnancy test during each visit)
- 5. Signature of the written informed consent form
- 6. Presence of a support person (for patient with cognitive disorders)
Who cannot join
- 1. Hypersensitivity to one of the excipients (glycerin, ethanol, lecithin)
- 2. Absence of hypoalbuminemia
- 3. During the 2 months before inclusion :
- Use of CoQ10
- Treatment with antioxidants (vitamin C) and statins
- Use of drugs affecting mitochondrial activity
Show all 20Show fewer
- Anti-cholesterol, thyroid hormones, anti-arrhythmic compounds, warfarin, metformin or clozapine
- 4. Treatment with vitamin E, calcium, magnesium and/or other vitamins with a concentration superior to 149 UI during more than 3 months before inclusion
- 5. Use of drugs interfering with catacholamine metabolism (reserpine, amphetamine, or inhibitors of the monoamine oxidase A, methylphenidate, cinnarizine) during the month before inclusion
- 6. Non balanced treatment with anxiolytics, hypnotics, tranquillizers and/or antidepressants during the month before inclusion
- 7. Hypothyroidism with thyroxin use
- 8. Epilepsy
- 9. Psychotic disorders
- 10. Pregnancy or lactation period
- 11. Woman of childbearing potential without efficient contraception
- 12. Participant to other therapeutic studies during the month before inclusion
- 13. Inability to receive a clear information on the research
- 14. Inability to participate to the totality of the study
- 15. Non affiliation to social security (beneficiary or assignee)
- 16. Refusal of signing the consent form
Trial locations and contacts
1 site in 1 country.
About this study
We propose a study on Ataxia with oculomotor apraxia type 1 (AOA1) in which Coenzyme Q10 (CoQ10) deficit has been observed. Main objectives of the study are :
- To monitor evolution of albumin in patients affected with AOA1 while supplemented with CoQ10 ;
- To measure with clinical scales and biological markers efficacy of supplementation on disease evolution.
AOA1 is characterised by Hypoalbuminemia. Disease duration is negatively correlated with albumin level. This study aims to understand mechanisms of the disease and our hypothesis is that correction or stabilization of albumin level with CoQ10 supplementation could impact disease evolution. The study is planned from 1 to 2 years supplementation. The CoQ10 is classified as a food supplement and has already been tested in other neurological conditions.
Read the detailed description
Ataxia with ocular apraxia type 1 (AOA1) is an autosomal recessive cerebellar ataxia. Patients' phenotype associates early onset cerebellar ataxia, oculomotor apraxia, neuropathy and often intellectual disability, hypoalbuminaemia and hypercholesterolemia.
APTX gene mutations responsible for AOA1 disease were identified in a family previously reported with ataxia and Coenzyme Q10 deficiency. Therefore we measured muscle Coenzyme Q10 in six patients AOA1 and found decreased levels in five. Hypercholesterolaemia and low albumin levels represent hallmarks of the disease.
We thus propose therapeutic trial with Coenzyme Q10 in AOA1 patients, by using albumin evolution as primary endpoint.
Moreover several secondary endpoints will be performed:
- clinical examination (SARA scale)
- quantitative assessments of the ataxia (with the calculation of the Composite Cerebellar Functional Severity CCFS)
- biological criteria (prealbumin, cholesterol, alphafoetoprotein, blood count, hepatic checkup)
- oculographic examination.
The study is a multicentric randomised placebo controlled trial with two-year follow-up:
- during the first year, one group will be supplemented with Coenzyme Q10 while the other group will receive a placebo;
- during the second year, all patients will be supplemented with Coenzyme Q10 in order to assess long term safety and tolerance of the treatment.
- Conditions
- Ataxia-oculomotor Apraxia 1
- Being tested
- CoQ10, Sanomit Placebo
- Keywords
- Ataxia with Oculomotor Apraxia type 1, CoQ10, hypoalbuminemia
- Medical subject headings
- Early-onset ataxia with oculomotor apraxia and hypoalbuminemia, Hypoalbuminemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CoQ10Dietary supplement
- 2 dosages according to patient weight: Weight < 50kg : 20 drops 3 times a day (150 mg / d) Weight ≥ 50 kg : 40 drops 3 times a day (300 mg / d)
Sanomit PlaceboOther
- according to patient weight: Weight < 50kg : 20 drops 3 times a day Weight ≥ 50 kg : 40 drops 3 times a day
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Coenzyme Q10 (CoQ10) - is a Dietary complement that contains Coenzyme Q10 (Ubidecarenone) well characterized nano particles. Receives: Dietary Supplement: CoQ10 |
| 2 | Placebo comparator | Placebo of CoQ10 is a translucent nano-emulsion of well characterized nano particles. Lecithin (and) Alcohol (and) Glycerin (and) Aqua Receives: Other: Sanomit Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Albuminemia
Evolution of albuminemia every 6 months during 2 years.
Measured: 2 years
Secondary outcomes (7)
SARA scale
Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)
Measured: 2 years
CCFS
Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)
Measured: 2 years
prealbuminemia
Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
Measured: 2 years
cholesterol
Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
Measured: 2 years.
alfa-foeto-protein
Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
Measured: 2 years.
Oculomotor evaluation
Oculomotor evaluation to assess oculo motor apraxia evolution [Time Frame: Each year during 2 years.]
Measured: 2 years
EQ5D - PHQ9
Quality of life evolution (self-administered questionnaire EQ5D - PHQ9) every 6 months during 2 years.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2015 (estimated)
- Study startJun 2013
- Primary completionSep 2017
- Study completionSep 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ataxia-oculomotor Apraxia 1Studies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- coenzyme Q10
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 19 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Perrine Charles, MD, PhD, Principal investigator, Assitance Publique - Hopitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02333305
- Sponsor's protocol ID
- P081107
- Acronym
- AOA1
- Status last verified
- Oct 2017
- First submitted
- 29 Sep 2014
- Last updated
- 6 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10
- ClinicalTrials.gov ID
- NCT02333305
- Acronym / sponsor ID
- AOA1 / P081107
- Conditions
- Ataxia-oculomotor Apraxia 1
- Keywords
- Ataxia with Oculomotor Apraxia type 1, CoQ10, hypoalbuminemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 19 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Jun 2013
- Primary completion
- Sep 2017 (actual)
- Study completion
- Sep 2017 (actual)
- First posted
- 7 Jan 2015 (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.
We propose a study on Ataxia with oculomotor apraxia type 1 (AOA1) in which Coenzyme Q10 (CoQ10) deficit has been observed. Main objectives of the study are :
- To monitor evolution of albumin in patients affected with AOA1 while supplemented with CoQ10 ;
- To measure with clinical scales and biological markers efficacy of supplementation on disease evolution.
AOA1 is characterised by Hypoalbuminemia. Disease duration is negatively correlated with albumin level. This study aims to understand mechanisms of the disease and our hypothesis is that correction or stabilization of albumin level with CoQ10 supplementation could impact disease evolution. The study is planned from 1 to 2 years supplementation. The CoQ10 is classified as a food supplement and has already been tested in other neurological conditions.
Detailed description
Ataxia with ocular apraxia type 1 (AOA1) is an autosomal recessive cerebellar ataxia. Patients' phenotype associates early onset cerebellar ataxia, oculomotor apraxia, neuropathy and often intellectual disability, hypoalbuminaemia and hypercholesterolemia.
APTX gene mutations responsible for AOA1 disease were identified in a family previously reported with ataxia and Coenzyme Q10 deficiency. Therefore we measured muscle Coenzyme Q10 in six patients AOA1 and found decreased levels in five. Hypercholesterolaemia and low albumin levels represent hallmarks of the disease.
We thus propose therapeutic trial with Coenzyme Q10 in AOA1 patients, by using albumin evolution as primary endpoint.
Moreover several secondary endpoints will be performed:
- clinical examination (SARA scale)
- quantitative assessments of the ataxia (with the calculation of the Composite Cerebellar Functional Severity CCFS)
- biological criteria (prealbumin, cholesterol, alphafoetoprotein, blood count, hepatic checkup)
- oculographic examination.
The study is a multicentric randomised placebo controlled trial with two-year follow-up:
- during the first year, one group will be supplemented with Coenzyme Q10 while the other group will receive a placebo;
- during the second year, all patients will be supplemented with Coenzyme Q10 in order to assess long term safety and tolerance of the treatment.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- coenzyme Q10
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Dietary Supplement: CoQ10Coenzyme Q10 (CoQ10) - is a Dietary complement that contains Coenzyme Q10 (Ubidecarenone) well characterized nano particles. |
| 2 | Placebo comparator | Other: Sanomit PlaceboPlacebo of CoQ10 is a translucent nano-emulsion of well characterized nano particles. Lecithin (and) Alcohol (and) Glycerin (and) Aqua |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 1. Diagnosis of ataxia with oculomotor apraxia type I (AOA1) confirmed by genetic molecular analysis
- 2. Age ≥ 18 years
- 3. Hypoalbuminemia
- 4. Efficient contraception for women of childbearing potential (with pregnancy test during each visit)
- 5. Signature of the written informed consent form
Key exclusion criteria (20 listed)
- 1. Hypersensitivity to one of the excipients (glycerin, ethanol, lecithin)
- 2. Absence of hypoalbuminemia
- 3. During the 2 months before inclusion :
- 4. Treatment with vitamin E, calcium, magnesium and/or other vitamins with a concentration superior to 149 UI during more than 3 months before inclusion
- 5. Use of drugs interfering with catacholamine metabolism (reserpine, amphetamine, or inhibitors of the monoamine oxidase A, methylphenidate, cinnarizine) during the month before inclusion
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 |
|---|---|---|
| CoQ10• 2 dosages according to patient weight: Weight < 50kg : 20 drops 3 times a day (150 mg / d) Weight ≥ 50 kg : 40 drops 3 times a day (300 mg / d) | Dietary supplement | 1 |
| Sanomit Placebo• according to patient weight: Weight < 50kg : 20 drops 3 times a day Weight ≥ 50 kg : 40 drops 3 times a day | Other | 2 |
Primary outcome
Albuminemia
Time frame: 2 years
Evolution of albuminemia every 6 months during 2 years.
7 secondary outcomes
SARA scale
Time frame: 2 years
CCFS
Time frame: 2 years
prealbuminemia
Time frame: 2 years
cholesterol
Time frame: 2 years.
alfa-foeto-protein
Time frame: 2 years.
Oculomotor evaluation
Time frame: 2 years
EQ5D - PHQ9
Time frame: 2 years
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 6 Nov 2017.
- 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.
Perrine Charles, MD, PhD principal investigator
Assitance Publique - Hopitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing CoQ10 in people with Ataxia-oculomotor Apraxia 1, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
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 6 inclusion and 20 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 6 Nov 2017. 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 France. 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. Some participants in this study may receive a placebo. 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?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
Albuminemia (time frame: 2 years). 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 6 Nov 2017; AzTrials last refreshed its copy on 8 Nov 2026.