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A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

Status Approved

First Submitted Date

2013/12/13
Registered Date

2014/01/07
Last Updated Date

2014/06/17

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CRIS Required

WHO ICTRP (International Clinical Trial Registry Platform) Required

1. Background

Background - CRIS Registration Number, Unique Protocol ID, Public/Brief Title, Scientific Title, Acronym, MFDS Regulated Study, IND/IDE Protocol, Registered at Other Registry, Name of Registry/Registration Number
CRIS Registration Number	KCT0000977
Unique Protocol ID	H6D-MC-LVJJ
Public/Brief Title	Trial of Tadalafil for Duchenne Muscular Dystrophy
Scientific Title	A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy
Acronym	H6D-MC-LVJJ
MFDS Regulated Study	Yes
IND/IDE Protocol	Yes
Registered at Other Registry	Yes
Name of Registry / Registration Number
Healthcare Benefit Approval Status

2. Institutional Review Board / Ethics Committee

Institutional Review Board Information
Board Approval Status	Submitted approval
Board Approval Number	H-1306-112-500
Approval Date	2013-09-03
Institutional Review Board Name	SNUCM SNU IRB (Seoul National University Hospital Institutional Review Board)
Institutional Review Board Address
Institutional Review Board Telephone
Data Monitoring Committee	No

3. Contact Details

Contact Details Information - Contact Person for Principal Investigator / Scientific Queries, Contact Person for Public Queries, Contact Person for Updating Information의 Name, Title, Email, Telephone, Cellular Phone, Affiliation, Address
Contact Person for Principal Investigator / Scientific Queries
Name	Jong Hee Chae
Title	M.D., Ph.D.
Telephone	+82-2-2072-1652
Affiliation	Seoul National University Hospital
Address
Contact Person for Public Queries
Name	Jong Hee Chae
Title	M.D., Ph.D.
Telephone	+82-2-2072-1652
Affiliation	Seoul National University Hospital
Address
Contact Person for Updating Information
Name	Jin A Choi
Title
Telephone	+82-2-3459-2675
Affiliation	Eli Lilly Korea
Address

4. Status

Status Information - Study Site, Overall Recruitment Status, Date of First Enrollment, Status of First Enrollment, Target Number of Participant, Primary Completion Date, Recruitment Status by Participating Study Site, Name of Study Site, Recruitment Status, Date of First Enrollment, Status of First Enrollemnt
Recruitment Status by Participating Study Site 1
Study Site	Single
Overall Recruitment Status	Recruiting
Date of First Enrollment	2013-12-17 Actual
Target Number of Participant	13
Primary Completion Date	2015-12-31 , Anticipated
Study Completion Date	2017-12-20 , Anticipated
Name of Study	Seoul National University Hospital
Recruitment Status	Recruiting
Date of First Enrollment	2013-12-17 ,

5. Source of Monetary / Material Support

Source of Monetary / Material Support Information - Organization Name, Organization Type, Project ID
1. Source of Monetary/Material Support
Organization Name	Eli Lilly Korea
Organization Type	Pharmaceutical Company
Project ID	H6D-MC-LVJJ

6. Sponsor Organization

Sponsor Organization Information - Organization Name, Organization Type
1. Sponsor Organization
Organization Name	Eli Lilly Korea
Organization Type	Pharmaceutical Company

7. Study Summary

Study Summary Information
Lay Summary	A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Tadalafil for Duchenne Muscular Dystrophy

8. Study Design

Study Design Information - Study Type, Study Purpose, Phase, Intervention Model, Blinding/Masking, Blinded Subject, Allocation, Intervention Type, Intervention Description, Number of Arms, Arm Label, Target Number of Participant, Arm Type, Arm Description
Study Type	Interventional Study
Study Purpose	Treatment
Phase	Phase3
Intervention Model	Parallel
Blinding/Masking	Double
Blinded Subject	Subject, Investigator
Allocation	RCT
Intervention Type	Drug
Intervention Description	Two doses of tadalafil (0.3 mg/kg and 0.6 mg/kg) will be compared to placebo. up to 48 weeks, once daily, oral.
Number of Arms	3
Arm 1	Arm Label Tadalifil 0.6 mg/kg (Maximum dose of 40mg
	Target Number of Participant 3
	Arm Type Experimental
	Arm Description tadalafil 0.6 mg/kg(Maximum dose of 40mg)up to 48 weeks, once daily, oral.
Arm 2	Arm Label tadalafil 0.3 mg/kg (Maximum dose of 20mg)
	Target Number of Participant 3
	Arm Type Experimental
	Arm Description tadalafil 0.3 mg/kg (Maximum dose of 20mg) up to 48 weeks, once daily, oral.
Arm 3	Arm Label placebo
	Target Number of Participant 5
	Arm Type Placebo comparator
	Arm Description placebo,up to 48 weeks, once daily, oral.

9. Subject Eligibility

Subject Eligibility Information
Condition(s)/Problem(s)	* (G00-G99)Diseases of the nervous system (G71.0)Muscular dystrophy
Rare Disease	No
Inclusion Criteria	Gender Male
	Age 7Year~14Year
	Description 1. Males with proven DMD 2. Ages 7-14 years inclusive 3. Ambulant, defined as 6MWD between 200 and 400 meters inclusive at screening and baseline 4. Baseline 6MWD measurement within 20% of the screening 6MWD 5. Left ventricular ejection fraction (LVEF) ≥50% as determined by echocardiogram obtained at screening, or based on record of an echocardiogram performed within 30 days of Visit 1 if a copy of the record can be obtained as source documentation for the screening LVEF 6. Receiving systemic corticosteroids for a minimum of 6 months immediately prior to screening, with no significant change in total daily dosage or dosing regimen (except those adjusting for weight changes) for a minimum of 3 months immediately prior to screening and a reasonable expectation that total daily dosage and dosing regimen will not change significantly (except for adjustments for weight) for the duration of the study. 7. Written informed consent from parents/legal guardian and written assent from patients will be obtained prior to any study procedure being performed
Exclusion Criteria	1. Symptomatic cardiomyopathy or heart failure (New York Heart Association Class III or IV) 2. Change in prophylactic treatment for heart failure within 3 months prior to start of study treatment 3. Cardiac rhythm disorder defined as sinus rhythm with ectopic contractions or conductance disturbances, or any rhythm other than sinus, observed on screening ECG 4. Use of continuous mechanical ventilator assistance. [Evening use of bi-level positive airway pressure (BPAP) or continuous positive airway pressure (CPAP) therapy is allowed] 5. Any contraindication to tadalafil (use of any form of organic nitrate, either regularly and/or intermittently, or known serious hypersensitivity to tadalafil) 6. History of significant renal insufficiency, defined as receiving renal dialysis or having a screening serum cystatin C level ≥ 2.35 mg/L 7. Clinical evidence of cirrhosis 8. Diagnosed with a retinal disorder (for example, hereditary retinal disorders, retinopathy of prematurity) 9. New or changed treatment with herbal or dietary supplements being taken with an expectation of an effect on muscle strength or function during 1 month prior to first dose of study drug.
Healthy Volunteers	No

10. Outcome Measure(s)

Outcome Measure(s) Information - Type of Primary Outcome, Primary Outcome, Outcome, Timepoint, Secondary Outcome, Outcome, Timepoint
Primary Outcome(s) 1
Type of Primary Outcome	/Safety/Efficacy
Outcome	lessens the decline in ambulatory ability as measured by the 6MWD compared to placebo in boys with Duchenne muscular dystrophy (DMD)
Timepoint	week 48
Secondary Outcome(s) 1
Outcome	lessens the decline in North Star Ambulatory Assessment (NSAA) global score
Timepoint	week 48

11. Study Results and Publication

Study Results and Publication Information - Result Registered, Final Enrollment Number, Number of Publication, Publications, Results Upload, Date of Posting Results, Protocol URL or File Upload, Brief Summary
Result Registered

12. Sharing of Study Data(Deidentified Individual-Patient Data, IPD)

Sharing of Study Data Information - Sharing Statement, Time of Sharing, Way of Sharing
Sharing Statement	Not provided at time of Registration

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