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A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects (DEMAND I)

Primary Purpose

Muscular Dystrophies

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
3 mg/kg GSK2402968
6 mg/kg GSK2402968
9 mg/kg GSK2402968
12 mg/kg GSK2402968
Placebo
Sponsored by
GlaxoSmithKline
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophies focused on measuring Duchenne, Duchenne Muscular Dystrophy, DMD, 968

Eligibility Criteria

9 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968.
  • Age 9 years old or greater at Screening;
  • Male;
  • Non-ambulant (at least 1 year in a wheelchair) within the last 4 years;
  • Life expectancy at least three years;
  • Willingness and ability to comply with all protocol requirements and procedures;
  • QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread;
  • Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug;
  • Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations).

Exclusion Criteria:

  • Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968;
  • Current or history of liver or renal disease;
  • Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments;
  • Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication;
  • Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication;
  • Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening;
  • Symptomatic cardiomyopathy;
  • Use of alcohol from Screening through to the 1 month Follow-up visit ;
  • Any Child in Care.

Sites / Locations

  • GSK Investigational Site
  • GSK Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Cohort 1

Cohort 2

Cohort 3

Cohort 4

Arm Description

3 mg/kg GSK2402968 / placebo

6 mg/kg GSK2402968 / placebo

9 mg/kg GSK2402968 / placebo

12 mg/kg GSK2402968 / placebo

Outcomes

Primary Outcome Measures

Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
Incidence of Adverse Events
Incidence of Injection Site Reactions

Secondary Outcome Measures

Full Information

First Posted
May 20, 2010
Last Updated
July 13, 2017
Sponsor
GlaxoSmithKline
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1. Study Identification

Unique Protocol Identification Number
NCT01128855
Brief Title
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
Acronym
DEMAND I
Official Title
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study to Assess the Pharmacokinetics, Safety and Tolerability of Single Subcutaneous Injections of GSK2402968 in Non-ambulant Subjects With Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
July 2017
Overall Recruitment Status
Completed
Study Start Date
July 12, 2010 (Actual)
Primary Completion Date
October 25, 2011 (Actual)
Study Completion Date
October 25, 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
GlaxoSmithKline

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is investigate the pharmacokinetics, safety and tolerability of single subcutaneous administration of GSK2402968 in non-ambulant boys with Duchenne muscular dystrophy

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophies
Keywords
Duchenne, Duchenne Muscular Dystrophy, DMD, 968

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
20 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
3 mg/kg GSK2402968 / placebo
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
6 mg/kg GSK2402968 / placebo
Arm Title
Cohort 3
Arm Type
Experimental
Arm Description
9 mg/kg GSK2402968 / placebo
Arm Title
Cohort 4
Arm Type
Experimental
Arm Description
12 mg/kg GSK2402968 / placebo
Intervention Type
Drug
Intervention Name(s)
3 mg/kg GSK2402968
Intervention Description
Weekly subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
6 mg/kg GSK2402968
Intervention Description
Weekly subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
9 mg/kg GSK2402968
Intervention Description
Weekly subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
12 mg/kg GSK2402968
Intervention Description
Weekly subcutaneous injection
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
Weekly Placebo
Primary Outcome Measure Information:
Title
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
Time Frame
35 days
Title
Incidence of Adverse Events
Time Frame
35 days
Title
Incidence of Injection Site Reactions
Time Frame
35 days

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
9 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Duchenne muscular dystrophy resulting from a mutation in the DMD gene, confirmed by a sponsor approved DNA diagnostic technique covering all DMD gene exons, including but not limited to MLPA (Multiplex Ligation-dependent Probe Amplification), CGH (Comparative Genomic Hybridisation), SCAIP (Single Condition Amplification/Internal Primer) or H-RMCA (High-Resolution Melting Curve Analysis), and correctable by treatment with GSK2402968. Age 9 years old or greater at Screening; Male; Non-ambulant (at least 1 year in a wheelchair) within the last 4 years; Life expectancy at least three years; Willingness and ability to comply with all protocol requirements and procedures; QTc <450msec (based on single or average QTc value of triplicate ECGs obtained over a brief recording period). Note: QTc may be either QTcB or QTcF, machine read or manual overread; Subjects must be willing to use adequate contraception (condoms or abstinence), from Screening until at least 5 months after the last dose of study drug; Informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations). Exclusion Criteria: Any additional mutation (such as an additional missing exon for DMD) that cannot be treated with GSK2402968; Current or history of liver or renal disease; Acute illness within 4 weeks of anticipated administration of study medication, which may interfere with study assessments; Use of anticoagulants, antithrombotics or antiplatelet agents, previous treatment with investigational drugs, idebenone or other forms of Coenzyme Q10, within 6 months of the first administration of study medication; Start of glucocorticosteroids within 6 months or non-stable use of glucocorticosteroids within 3 months of the anticipated first administration of study medication; Positive hepatitis B surface antigen (HbsAg), hepatitis C antibody test (HCV), or human immunodeficiency virus (HIV) test at Screening; Symptomatic cardiomyopathy; Use of alcohol from Screening through to the 1 month Follow-up visit ; Any Child in Care.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
GSK Clinical Trials
Organizational Affiliation
GlaxoSmithKline
Official's Role
Study Director
Facility Information:
Facility Name
GSK Investigational Site
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States
Facility Name
GSK Investigational Site
City
Paris cedex 13
ZIP/Postal Code
75651
Country
France

12. IPD Sharing Statement

Learn more about this trial

A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects

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