Long-term Safety, Tolerability and Efficacy of Idebenone in Duchenne Muscular Dystrophy (DELPHI Extension)

May 30, 2011 updated by: Santhera Pharmaceuticals

A Phase II Open-label Extension Study to Obtain Long-term Safety, Tolerability and Efficacy Data of Idebenone in the Treatment of Duchenne Muscular Dystrophy - Extension to Study SNT-II-001

The scientific aim of the present extension study is to monitor long-term safety and tolerability of idebenone in patients with DMD. Furthermore, the long-term effect on respiratory, cardiac and motor functions, and skeletal muscle strength/function will be assessed.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

21

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Belgium
      • Leuven, Belgium, 3000
        • University Hospitals Leuven

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

Inclusion Criteria:

  • Completion of study SNT-II-001
  • Body weight ≥ 25 kg
  • Glucocorticosteroids and ACE-inhibitors are allowed, if on stable dosage within 2 months prior to inclusion
  • Eligibility to participate in the present extension study as confirmed by the investigator

Exclusion Criteria:

  • Safety or tolerability issues arising during the course of SNT-II-001 which in the opinion of the investigator preclude further treatment with idebenone
  • Clinically significant abnormalities of haematology or biochemistry
  • Abuse of drugs or alcohol
  • Use of coenzyme Q10 or idebenone within 30 days prior to inclusion
  • Intake of any investigational drug within 30 days prior to inclusion
  • Symptomatic heart failure
  • Previous history of ventricular arrhythmias (other than isolated ventricular extrasystole); ventricular arrhythmias presented at baseline
  • Known individual hypersensitivity to idebenone or to any of the excipients

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: only one arm
Drug: Idebenone
Patients ≤ 45 kg: 450 mg/day (1 tablet 3 times a day) Patients > 45 kg: 900 mg/day (2 tablets 3 times a day)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Measures of safety and tolerability of idebenone: - Nature and frequency of AEs - Laboratory parameters (haematology, biochemistry and urinalysis) - Physical examinations and vital signs - ECGs
Time Frame: Month 0, 3, 6, 12, 18, 24, FU
Month 0, 3, 6, 12, 18, 24, FU

Secondary Outcome Measures

Outcome Measure
Time Frame
Measures of efficacy of idebenone: - Respiratory Function Testing - Motor Function Measure - Quantitative Muscle Testing - Hand-Held Myometry - Echocardiography and Color Doppler Myocardial Imaging - Cardiac biomarkers
Time Frame: Month 0, 6, 12, 18, 24
Month 0, 6, 12, 18, 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Investigators

  • Principal Investigator: Prof Gunnar Buyse, MD PhD, Universitaire Ziekenhuizen KU Leuven

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2008

Primary Completion (Actual)

January 1, 2011

Study Completion (Actual)

January 1, 2011

Study Registration Dates

First Submitted

September 23, 2008

First Submitted That Met QC Criteria

September 23, 2008

First Posted (Estimate)

September 25, 2008

Study Record Updates

Last Update Posted (Estimate)

June 1, 2011

Last Update Submitted That Met QC Criteria

May 30, 2011

Last Verified

May 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SN T-II-001-E

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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