NCT00983788

Brief Summary

The investigators propose to evaluate the effect of bezafibrate on metabolism during exercise in 22 adult patients affected with carnitine palmitoyltransferase II (CPTII) or very-long chain acyl-CoA-dehydrogenase (VLCAD) deficiencies. This study will be an 9-month, randomized, double-blind, placebo-controlled crossover trial. The trial will be conducted in two centers: Institut de Myologie, Pitié-Salpêtrière Hospital in France, and Rigshospitalet, University of Copenhagen, in Denmark. The main criteria for assessing the potential effect of this drug will be the fat oxidation rate studied during a moderate workload on cycle ergometer, after infusion of stable isotopes (palmitate and glucose tracers).

Trial Health

87
On Track

Trial Health Score

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

Enrollment
12

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2009

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

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 23, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 24, 2009

Completed
7 days until next milestone

Study Start

First participant enrolled

October 1, 2009

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2011

Completed
Last Updated

May 30, 2012

Status Verified

May 1, 2012

Enrollment Period

1.9 years

First QC Date

September 23, 2009

Last Update Submit

May 29, 2012

Conditions

Carnitine Palmitoyltransferase II DeficiencyVery Long Chain Acyl Coa Dehydrogenase Deficiency

Keywords

CPT IIVLCAD

Outcome Measures

Primary Outcomes (1)

  • Fatty acid oxidation

    Two years

Secondary Outcomes (1)

  • Heart rate

    Two years

Study Arms (2)

Bezafibrate

EXPERIMENTAL
Drug: Bezafibrate

Placebo

PLACEBO COMPARATOR
Other: Placebo

Interventions

BezafibrateDRUG

3 x 200 mg/ day

Bezafibrate
PlaceboOTHER

Placebo

Placebo

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • CPT II or VLCAD deficiency

You may not qualify if:

  • Competing disorders
  • Liver or kidney disease
  • Allergy towards fibrates
  • Pregnancy and breastfeeding

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Neuromusculare Research Unit, Rigshospitalet

Copenhagen, Copenhagen, 2100, Denmark

Location

Related Publications (1)

  • References - Andresen BS, Olpin S, Poorthuis BJHM, Scholte HR, Vianey-Saban C, Wanders R et al. Am J Hum Genet 1999; 64: 478-494 - Bonnefont JP, Taroni F, Cavadini P, Cepanec C, Brivet M, Saudubray JM, Leroux JP, Demaugre F, (1996) Am J Hum Genet 58:971-978. - Demaugre F, Bonnefont JP, Colonna M, Cepanec C, Leroux JP, Saudubray JM. (1991) J Clin Invest 87:859-864. - DiMauro S, Melis-DiMauro PM. Science 1973;182: 929-931. - Djouadi F, Bonnefont JP, Thuillier L, Droin V, Khadom N, Munnich A, Bastin J. (2003a). Pediatr Res. 54:446-51 - Djouadi F, Bonnefont JP, Munnich A, Bastin J. (2003b). Mol Genet Metab.;78:112-8. - Djouadi F, Aubey F, Schlemmer D, Ruiter JPN, Wanders RJA, Strauss AW et al. Hum Mol Genet 2005; 18: 2495-2703 - Djouadi F, Aubey F, Schlemmer D, Ruiter JPN, Wanders RJA, Strauss AW et al. Hum Mol Genet 2005; 14:2695-2703. - Djouadi F, Aubey F, Schlemmer D, Gobin S, Laforet P, Wanders RJA, et al. J Inherit Metab Dis 2006; 29: 341-342. - Djouadi F, Bastin J, Laforêt P, Aubey F, Mogenet A, Romano S et al. ASHG 57th Annual meeting (2007). - Johansen K. Basal Sundhedsvidenskabelig statistik. Munksgaard, Denmark 2002 1st edition. - Lemberger T, Desvergne B, Wahli W (1996) Annu. Rev. Cell. Dev. Biol. 12:335-363. - Ørngreen MC, Ejstrup R, Vissing J. Neurology 2003; 61:559-561. - Ørngreen MC, Nørgaard MG, Sacchetti M, van Engelen BGM, Vissing J. Ann Neurol. 2004; 56:279-282. - Ørngreen MC, Dunø M, Ejstrup R, Christensen E, Schwartz M, Sacchetti M, Vissing J. Ann Neurol. 2005; 57:60-66. - Ørngreen MC, Schelhaas HJ, Jeppesen TD, Akman HO, Wevers RA, Andersen ST, Ter Laak HJ, van Diggelen OP, DiMauro S, Vissing J. INeurology, 2008;70(20):1876-82. - Roe CR, Sweetman L, Roe DS, David F, Brunengraber H. J Clin Invest 2002; 110: 259-269. - Vianey-Saban C, Divry P, Brivet M, Nada M, Zabot M-T, Mathieu M et al. Clin Chim Acta 1998; 269: 43-62.

    BACKGROUND

MeSH Terms

Conditions

Carnitine palmitoyl transferase 2 deficiencyVLCAD deficiency

Interventions

Bezafibrate

Intervention Hierarchy (Ancestors)

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

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD

Study Record Dates

First Submitted

September 23, 2009

First Posted

September 24, 2009

Study Start

October 1, 2009

Primary Completion

September 1, 2011

Study Completion

September 1, 2011

Last Updated

May 30, 2012

Record last verified: 2012-05

Locations

DK(1)