NCT02333305

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
19

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jun 2013

Typical duration for phase_3

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2013

Completed
1.3 years until next milestone

First Submitted

Initial submission to the registry

September 29, 2014

Completed
3 months until next milestone

First Posted

Study publicly available on registry

January 7, 2015

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2017

Completed
Last Updated

November 6, 2017

Status Verified

October 1, 2017

Enrollment Period

4.3 years

First QC Date

September 29, 2014

Last Update Submit

November 3, 2017

Conditions

Ataxia-oculomotor Apraxia 1

Keywords

Ataxia with Oculomotor Apraxia type 1CoQ10hypoalbuminemia

Outcome Measures

Primary Outcomes (1)

  • Albuminemia

    Evolution of albuminemia every 6 months during 2 years.

    2 years

Secondary Outcomes (7)

  • SARA scale

    2 years

  • CCFS

    2 years

  • prealbuminemia

    2 years

  • cholesterol

    2 years.

  • alfa-foeto-protein

    2 years.

  • +2 more secondary outcomes

Study Arms (2)

1

EXPERIMENTAL

Coenzyme Q10 (CoQ10) - is a Dietary complement that contains Coenzyme Q10 (Ubidecarenone) well characterized nano particles.

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

Other: Sanomit Placebo

Interventions

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)

1
Sanomit PlaceboOTHER

• according to patient weight: Weight \< 50kg : 20 drops 3 times a day Weight ≥ 50 kg : 40 drops 3 times a day

2

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • \. Diagnosis of ataxia with oculomotor apraxia type I (AOA1) confirmed by genetic molecular analysis
  • \. Age ≥ 18 years
  • \. Hypoalbuminemia
  • \. Efficient contraception for women of childbearing potential (with pregnancy test during each visit)
  • \. Signature of the written informed consent form
  • \. Presence of a support person (for patient with cognitive disorders)

You may not qualify if:

  • \. Hypersensitivity to one of the excipients (glycerin, ethanol, lecithin)
  • \. Absence of hypoalbuminemia
  • 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
  • \. Hypothyroidism with thyroxin use
  • \. Epilepsy
  • \. Psychotic disorders
  • \. Pregnancy or lactation period
  • \. Woman of childbearing potential without efficient contraception
  • \. Inability to receive a clear information on the research
  • \. Inability to participate to the totality of the study
  • \. Non affiliation to social security (beneficiary or assignee)
  • \. Refusal of signing the consent form

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

ICM Institute

Paris, 75013, France

Location

MeSH Terms

Conditions

Early-onset ataxia with oculomotor apraxia and hypoalbuminemiaHypoalbuminemia

Interventions

coenzyme Q10

Condition Hierarchy (Ancestors)

HypoproteinemiaBlood Protein DisordersHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Perrine Charles, MD, PhD

    Assitance Publique - Hopitaux de Paris

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
placebo
Purpose
TREATMENT
Intervention Model
PARALLEL
Model Details: coenzyme Q10
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 29, 2014

First Posted

January 7, 2015

Study Start

June 1, 2013

Primary Completion

September 1, 2017

Study Completion

September 1, 2017

Last Updated

November 6, 2017

Record last verified: 2017-10

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)