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Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10 (AOA1)

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ClinicalTrials.gov Identifier: NCT02333305
Recruitment Status : Completed
First Posted : January 7, 2015
Last Update Posted : November 6, 2017
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief 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 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.


Condition or disease Intervention/treatment Phase
Ataxia-oculomotor Apraxia 1 Dietary Supplement: CoQ10 Other: Sanomit Placebo Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 19 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: coenzyme Q10
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: placebo
Primary Purpose: Treatment
Official Title: Evolution of Albumin on AOA1 Patients Supplemented With Coenzyme Q10
Study Start Date : June 2013
Actual Primary Completion Date : September 2017
Actual Study Completion Date : September 2017


Arm Intervention/treatment
Experimental: 1
Coenzyme Q10 (CoQ10) - is a Dietary complement that contains Coenzyme Q10 (Ubidecarenone) well characterized nano particles.
Dietary Supplement: 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)

Placebo Comparator: 2
Placebo of CoQ10 is a translucent nano-emulsion of well characterized nano particles. Lecithin (and) Alcohol (and) Glycerin (and) Aqua
Other: Sanomit Placebo
• according to patient weight: Weight < 50kg : 20 drops 3 times a day Weight ≥ 50 kg : 40 drops 3 times a day




Primary Outcome Measures :
  1. Albuminemia [ Time Frame: 2 years ]
    Evolution of albuminemia every 6 months during 2 years.


Secondary Outcome Measures :
  1. SARA scale [ Time Frame: 2 years ]
    Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)

  2. CCFS [ Time Frame: 2 years ]
    Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)

  3. prealbuminemia [ Time Frame: 2 years ]
    Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.

  4. cholesterol [ Time Frame: 2 years. ]
    Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.

  5. alfa-foeto-protein [ Time Frame: 2 years. ]
    Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.

  6. Oculomotor evaluation [ Time Frame: 2 years ]
    Oculomotor evaluation to assess oculo motor apraxia evolution [Time Frame: Each year during 2 years.]

  7. EQ5D - PHQ9 [ Time Frame: 2 years ]
    Quality of life evolution (self-administered questionnaire EQ5D - PHQ9) every 6 months during 2 years.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria :

  • 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)

Exclusion criteria :

  • 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
    • 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

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): NCT02333305


Locations
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France
ICM Institute
Paris, France, 75013
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
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Principal Investigator: Perrine Charles, MD, PhD Assitance Publique - Hopitaux de Paris

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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT02333305     History of Changes
Other Study ID Numbers: P081107
First Posted: January 7, 2015    Key Record Dates
Last Update Posted: November 6, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Ataxia with Oculomotor Apraxia type 1
CoQ10
hypoalbuminemia

Additional relevant MeSH terms:
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Ataxia
Apraxias
Cogan Syndrome
Cerebellar Ataxia
Hypoalbuminemia
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Psychomotor Disorders
Neurobehavioral Manifestations
Vestibulocochlear Nerve Diseases
Cranial Nerve Diseases
Eye Diseases
Vasculitis
Vascular Diseases
Cardiovascular Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Hypoproteinemia
Blood Protein Disorders
Hematologic Diseases
Ubiquinone
Coenzyme Q10
Micronutrients
Nutrients
Growth Substances
Physiological Effects of Drugs
Vitamins