NCT00657748

Brief Summary

The aim of this study is to determine whether oral supplementation with lithium and acetate may improve the biological and clinical prognosis in patients with Canavan Disease.

Trial Health

15
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Timeline
Completed

Started Sep 2009

Shorter than P25 for phase_2

Status
withdrawn

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

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 9, 2008

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 14, 2008

Completed
1.4 years until next milestone

Study Start

First participant enrolled

September 1, 2009

Completed
1 year until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2010

Completed
4 months until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2011

Completed
Last Updated

April 21, 2015

Status Verified

April 1, 2015

Enrollment Period

1 year

First QC Date

April 9, 2008

Last Update Submit

April 20, 2015

Conditions

Canavan DiseaseInfantileDeficiency DiseaseAspartoacylaseLeukodystrophy, Spongiform

Keywords

LeukodystrophyCanavan disease,Aspartoacylase,Lithium,Glyceryl Triacetate

Outcome Measures

Primary Outcomes (1)

  • The primary outcome will be a decrease of the NAA peak (> 20%) or the appearance of an acetate peak at the end of the treatment (10 months), using spectroscopy-MRI.

    10 months

Secondary Outcomes (3)

  • Secondary outcomes will be assessed at 10 months (end of the treatment): -Improvement of neuromotor performances (GMFM and Mullen scales), spasticity, and neurological severity

    10 months

  • -Improvement of epilepsy (number of seizures)

    10 months

  • -Decrease in NAA and increase in acetate contents in fluids (CSF, plasma, urine).

    10 months

Study Arms (1)

1

EXPERIMENTAL
Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)

Interventions

Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)DRUG

Lithium 1.3 mEq/kg/day (three administrations a day)during the study GTA 500 mg/kg/day in four administrations a day during the study

Also known as: Lithium Gluconate (drug), Glyceryl Triacetate, GTA (drug)
1

Eligibility Criteria

Age1 Year - 15 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Clinical and biochemical diagnosis of Canavan disease

You may not qualify if:

  • Renal disease
  • Thyroid disease
  • Cardiac disease
  • Impossibility to perform brain MRI

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (4)

  • Mathew R, Arun P, Madhavarao CN, Moffett JR, Namboodiri MA. Progress toward acetate supplementation therapy for Canavan disease: glyceryl triacetate administration increases acetate, but not N-acetylaspartate, levels in brain. J Pharmacol Exp Ther. 2005 Oct;315(1):297-303. doi: 10.1124/jpet.105.087536. Epub 2005 Jul 7.

    PMID: 16002461RESULT

  • Madhavarao CN, Arun P, Moffett JR, Szucs S, Surendran S, Matalon R, Garbern J, Hristova D, Johnson A, Jiang W, Namboodiri MA. Defective N-acetylaspartate catabolism reduces brain acetate levels and myelin lipid synthesis in Canavan's disease. Proc Natl Acad Sci U S A. 2005 Apr 5;102(14):5221-6. doi: 10.1073/pnas.0409184102. Epub 2005 Mar 22.

    PMID: 15784740RESULT

  • Janson CG, Assadi M, Francis J, Bilaniuk L, Shera D, Leone P. Lithium citrate for Canavan disease. Pediatr Neurol. 2005 Oct;33(4):235-43. doi: 10.1016/j.pediatrneurol.2005.04.015.

    PMID: 16194720RESULT

  • Baslow MH, Kitada K, Suckow RF, Hungund BL, Serikawa T. The effects of lithium chloride and other substances on levels of brain N-acetyl-L-aspartic acid in Canavan disease-like rats. Neurochem Res. 2002 May;27(5):403-6. doi: 10.1023/a:1015504031229.

    PMID: 12064356RESULT

Related Links

MeSH Terms

Conditions

Canavan DiseaseDeficiency Diseases

Interventions

gluconic acidPharmaceutical PreparationsTriacetin

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesLeukoencephalopathiesDemyelinating DiseasesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic DiseasesMalnutritionNutrition Disorders

Intervention Hierarchy (Ancestors)

TriglyceridesGlyceridesLipids

Study Officials

  • Patrick Aubourg, MD, PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR

  • Caroline Sevin, MD, PhD

    Assistance Publique - Hôpitaux de Paris

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

April 9, 2008

First Posted

April 14, 2008

Study Start

September 1, 2009

Primary Completion

September 1, 2010

Study Completion

January 1, 2011

Last Updated

April 21, 2015

Record last verified: 2015-04