Long-term Safety, Tolerability and Efficacy of Idebenone... | NCT00758225

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

NCT00758225

Sponsor

Santhera Pharmaceuticals

Status

Completed

Last Update Posted

Jun 1, 2011Estimated

Enrollment

21Estimated

Phase

Phase 2

Conditions

Duchenne Muscular Dystrophy

Interventions

Idebenone

Countries

Belgium

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

May 2011

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Sep 2008

Primary Completion Date

Jan 2011Actual

Completion Date

Jan 2011Actual

First Submitted Date

Sep 23, 2008

First Submission Date that Met QC Criteria

Sep 23, 2008

First Posted Date

Sep 25, 2008Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

May 30, 2011

Last Update Posted Date

Jun 1, 2011Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

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.

Detailed Description

Not provided

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

21Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
only one arm	Experimental		Drug: Idebenone

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Idebenone	Drug	Patients ≤ 45 kg: 450 mg/day (1 tablet 3 times a day) Patients > 45 kg: 900 mg/day (2 tablets 3 times a day)	only one arm

Outcomes Module

Primary Outcomes

Measure	Description	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		Month 0, 3, 6, 12, 18, 24, FU

Secondary Outcomes

Measure	Description	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		Month 0, 6, 12, 18, 24

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Prof Gunnar Buyse, MD PhD	Universitaire Ziekenhuizen KU Leuven	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University Hospitals Leuven		Leuven		3000	Belgium

Condition Browse Module

MeSH Terms

ID	Term
D020388	Muscular Dystrophy, Duchenne

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

Not provided

Browse Branches

Not provided