Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients suffering from an inborn muscular dysfunction characterized by intramuscular lack of energy supply

Clinical study for the assessment of safety and efficacy of Bezafibrate as a drug therapy for patients with mitochondrial myopathies - MitoFibrate CT1

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2012-002692-34-AT

Enrollment

Registered

2013-02-26

Start date

2013-06-26

Completion date

Unknown

Last updated

2026-04-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

mitochondrial disorder

Interventions

Trade Name: Bezastad Product Name: Bezastad Pharmaceutical Form: Coated tablet INN or Proposed INN: BEZAFIBRATE CAS Number: 41859-67-0 Other descriptive name: 2-[4-[2-(4-Chlorobenzamido)ethyl]phenoxy]

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: Ability to walk Age: 2-50 years confirmed Complex-I-defect or PDHc-defect ability to cope with the requirements for study participation Are the trial subjects under 18? yes Number of subjects for this age range: 8 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: previously known intolerance or other contra-indications for bezafibrat

Design outcomes

Primary

Measure	Time frame
Main Objective: Recent findings derived from animal and cell culture studies demonstrate an upregulation of synthesis and activity of mitochondrial enzymes under bezafibrat and thus point to a beneficial effect of this substance for the therapy of mitochondrial myopathies (H. Tyynismaa et al. 2010, T. Wenz 2008 und 2010). This findings are further substantiated by clinical observations in course of compassionate use of Bezafibrat for patinets with mitochondrial myopathies at the centres planned to participate in this trial (Reutlingen, München, Salzburg, Innsbruck). The main objective of the trial is therefore the systematic evaluation of efficacy and safety of Bezafibrat as a therapeutic option for patients with mitochondrial myopathies.;Secondary Objective: Not applicable;Primary end point(s): time intervall until pre-defined manifestation of disease progression measured by walking-distance in a 6-minutes-walking test disease progression is defined as a decline of >20% in performance ;Timepoint(s) of evaluation of this end point: The subjective disease progression is measured every 4 weeks by the patients or their parents by Newcastle Scale Section 4. Once the score points to a disease progression, patients visit the study site where the primary endpoint is evaluated. Elsewise, the primary endpoint is routinely evaluated 3 and 6 months post randomization. A decline of >20% in the 6-minutes-walking test leads to termination of study participation. Elsewise, termination is routinely scheduled at study visit 6 months post randomization.	—

Measure

Time frame

Main Objective: Recent findings derived from animal and cell culture studies demonstrate an upregulation of synthesis and activity of mitochondrial enzymes under bezafibrat and thus point to a beneficial effect of this substance for the therapy of mitochondrial myopathies (H. Tyynismaa et al. 2010, T. Wenz 2008 und 2010). This findings are further substantiated by clinical observations in course of compassionate use of Bezafibrat for patinets with mitochondrial myopathies at the centres planned to participate in this trial (Reutlingen, München, Salzburg, Innsbruck). The main objective of the trial is therefore the systematic evaluation of efficacy and safety of Bezafibrat as a therapeutic option for patients with mitochondrial myopathies.;Secondary Objective: Not applicable;Primary end point(s): time intervall until pre-defined manifestation of disease progression measured by walking-distance in a 6-minutes-walking test disease progression is defined as a decline of >20% in performance ;Timepoint(s) of evaluation of this end point: The subjective disease progression is measured every 4 weeks by the patients or their parents by Newcastle Scale Section 4. Once the score points to a disease progression, patients visit the study site where the primary endpoint is evaluated. Elsewise, the primary endpoint is routinely evaluated 3 and 6 months post randomization. A decline of >20% in the 6-minutes-walking test leads to termination of study participation. Elsewise, termination is routinely scheduled at study visit 6 months post randomization.

—

Secondary

Measure	Time frame
Secondary end point(s): subjective disease progression: Newcastle Paediatric Mitochondrial Disease Rating Scale function of the autonomous nervous sytem: ANS-test ;Timepoint(s) of evaluation of this end point: Newcastle Paediatric Mitochondrial Disease Rating Scale: every 4 weeks ANS-Test: every 3 months	—

Countries

Austria

Contacts

Public ContactClinical trial information

Institut für angeborene Stoffwechselerkrankungen

florian.lagler@pmu.ac.at00436624420021224

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Apr 23, 2026