NCT00336167

Brief Summary

The purpose of this study is to determine whether bezafibrate is effective in the treatment of the muscular adult form of carnitine palmitoyltransferase 2 deficiency

Trial Health

43
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
12

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jun 2006

Shorter than P25 for phase_3

Geographic Reach
1 country

1 active site

Status
unknown

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, 2006

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

June 9, 2006

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 13, 2006

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2007

Completed
Last Updated

April 9, 2007

Status Verified

April 1, 2007

First QC Date

June 9, 2006

Last Update Submit

April 6, 2007

Conditions

Carnitine Palmitoyl Transferase 2 Deficiency

Keywords

carnitine palmitoyl transferase 2 deficiencyFatty acid oxidationMitochondria

Outcome Measures

Primary Outcomes (1)

  • Rate of 3H-palmitate oxidation in the patients'lymphocytes and skeletal muscle

Secondary Outcomes (3)

  • Rate of CPT2 enzymatic activity in the patients'lymphocytes and skeletal muscle

  • Rate of palmitoyl-L-carnitine oxidation in the patients' skeletal muscle

  • Steady-state amount of CPT2 mRNA in the patients'skeletal muscle

Interventions

bezafibrate (drug)DRUG

Eligibility Criteria

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

You may qualify if:

  • occurrence of at least 5 attacks of rhabdomyolysis or of severe myalgias per year, AND/OR permanent muscle weakness objectivized by muscle testing outside a rhabdomyolysis attack AND
  • significant decrease in both the CPT2 activity and the rate of long-chain fatty acid oxidation measured in lymphocytes and/or in a skeletal muscle sample outside a rhabdomyolysis attack

You may not qualify if:

  • age below 18 years
  • less than 5 attacks of rhabdomyolysis or severe myalgias per year AND absence of muscle impairment detected by muscle testing
  • liver failure, renal failure, hyperhomocysteinemia prior to setting up the bezafibrate therapy
  • treatment with another hypolipidemic drug ("statins) or with anticoagulant
  • pregnancy or lactation during the period of fibrate therapy

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Jean Paul Bonnefont

Paris, 75015, France

RECRUITING

MeSH Terms

Conditions

Carnitine palmitoyl transferase 2 deficiency

Interventions

BezafibratePharmaceutical Preparations

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsFibric AcidsIsobutyratesButyratesAcids, AcyclicCarboxylic AcidsBenzoatesAcids, CarbocyclicChlorobenzoatesPhenyl EthersEthersBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPhenols

Study Officials

  • Bruno EYMARD, M.D

    Service de Neurologie 2 Groupe hospitalier Pitié-Salpétriere, Paris, France

    STUDY CHAIR

  • Jean Paul BONNEFONT, M.D., Ph D,

    Unite INSERM U781

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Jean-Paul Bonnefont, M.D., Ph.D.

CONTACT

144495647bonnefon@necker.fr

Jean Bastin, Ph D;

CONTACT

bastin@necker.fr

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

June 9, 2006

First Posted

June 13, 2006

Study Start

June 1, 2006

Study Completion

July 1, 2007

Last Updated

April 9, 2007

Record last verified: 2007-04

Locations

FR(1)