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
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|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||19 participants|
|Intervention Model:||Parallel Assignment|
|Intervention Model Description:||coenzyme Q10|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
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
- Albuminemia [ Time Frame: 2 years ]Evolution of albuminemia every 6 months during 2 years.
- SARA scale [ Time Frame: 2 years ]Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)
- CCFS [ Time Frame: 2 years ]Evolution of clinical criteria (SARA and CCFS, which represent quantitative scales to assess cerebellar ataxia evolution)
- prealbuminemia [ Time Frame: 2 years ]Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
- cholesterol [ Time Frame: 2 years. ]Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
- alfa-foeto-protein [ Time Frame: 2 years. ]Evolution of biological criteria (prealbuminemia, cholesterol, alfa-foeto-protein) every 6 months during 2 years.
- Oculomotor evaluation [ Time Frame: 2 years ]Oculomotor evaluation to assess oculo motor apraxia evolution [Time Frame: Each year during 2 years.]
- EQ5D - PHQ9 [ Time Frame: 2 years ]Quality of life evolution (self-administered questionnaire EQ5D - PHQ9) every 6 months during 2 years.
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
|Paris, France, 75013|
|Principal Investigator:||Perrine Charles, MD, PhD||Assitance Publique - Hopitaux de Paris|