Muscular DystrophiesPhase 1DEMAND INCT01128855
A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects
A Phase 1 study testing 3 mg/kg GSK2402968, 6 mg/kg GSK2402968 and 9 mg/kg GSK2402968 in people with Muscular Dystrophies, run by GlaxoSmithKline at 2 sites in 2 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 9 years and older, men only
- Conditions the study is for
- Muscular Dystrophies
- What is being tested?
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968 and 9 mg/kg GSK2402968
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 12 Jul 2010to 25 Oct 2011
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 9 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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;
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- 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).
Who cannot join
- 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;
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- Symptomatic cardiomyopathy;
- Use of alcohol from Screening through to the 1 month Follow-up visit ;
- Any Child in Care.
Trial locations and contacts
2 sites in 2 countries.
2 sites in 2 countries
About this study
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
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
- Conditions
- Muscular Dystrophies
- Being tested
- 3 mg/kg GSK2402968, 6 mg/kg GSK2402968, 9 mg/kg GSK2402968, 12 mg/kg GSK2402968, Placebo
- Keywords
- Duchenne, Duchenne Muscular Dystrophy, DMD, 968
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
3 mg/kg GSK2402968Drug
Weekly subcutaneous injection
6 mg/kg GSK2402968Drug
Weekly subcutaneous injection
9 mg/kg GSK2402968Drug
Weekly subcutaneous injection
12 mg/kg GSK2402968Drug
Weekly subcutaneous injection
PlaceboOther
Weekly Placebo
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Experimental | 3 mg/kg GSK2402968 / placebo Receives: Drug: 3 mg/kg GSK2402968; Other: Placebo |
| Cohort 2 | Experimental | 6 mg/kg GSK2402968 / placebo Receives: Drug: 6 mg/kg GSK2402968; Other: Placebo |
| Cohort 3 | Experimental | 9 mg/kg GSK2402968 / placebo Receives: Drug: 9 mg/kg GSK2402968; Other: Placebo |
| Cohort 4 | Experimental | 12 mg/kg GSK2402968 / placebo Receives: Drug: 12 mg/kg GSK2402968; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
Measured: 35 days
Incidence of Adverse Events
Measured: 35 days
Incidence of Injection Site Reactions
Measured: 35 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2010 (estimated)
- Study start12 Jul 2010
- Primary completion25 Oct 2011
- Study completion25 Oct 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by GlaxoSmithKlineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- GlaxoSmithKline
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- GSK Clinical Trials, Study director, GlaxoSmithKline
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01128855
- Sponsor's protocol ID
- 114118
- Acronym
- DEMAND I
- Status last verified
- Jul 2017
- First submitted
- 20 May 2010
- Last updated
- 18 Jul 2017
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01128855
- Acronym / sponsor ID
- DEMAND I / 114118
- Conditions
- Muscular Dystrophies
- Keywords
- Duchenne, Duchenne Muscular Dystrophy, DMD, 968
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- GlaxoSmithKlineIndustry (pharma, biotech, devices)
- Study start
- 12 Jul 2010 (actual)
- Primary completion
- 25 Oct 2011 (actual)
- Study completion
- 25 Oct 2011 (actual)
- First posted
- 24 May 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | Experimental | Drug: 3 mg/kg GSK2402968, Other: Placebo3 mg/kg GSK2402968 / placebo |
| Cohort 2 | Experimental | Drug: 6 mg/kg GSK2402968, Other: Placebo6 mg/kg GSK2402968 / placebo |
| Cohort 3 | Experimental | Drug: 9 mg/kg GSK2402968, Other: Placebo9 mg/kg GSK2402968 / placebo |
| Cohort 4 | Experimental | Drug: 12 mg/kg GSK2402968, Other: Placebo12 mg/kg GSK2402968 / placebo |
Who may qualify
- Ages
- 9 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- 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;
Key exclusion criteria (9 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 3 mg/kg GSK2402968Weekly subcutaneous injection | Drug | Cohort 1 |
| 6 mg/kg GSK2402968Weekly subcutaneous injection | Drug | Cohort 2 |
| 9 mg/kg GSK2402968Weekly subcutaneous injection | Drug | Cohort 3 |
| 12 mg/kg GSK2402968Weekly subcutaneous injection | Drug | Cohort 4 |
| PlaceboWeekly Placebo | Other | Cohort 1, Cohort 2, Cohort 3, Cohort 4 |
Primary outcome
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F
Time frame: 35 days
Incidence of Adverse Events
Time frame: 35 days
Incidence of Injection Site Reactions
Time frame: 35 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jul 2017.
- Results for study 114118 can be found on the GSK Clinical Study Register.Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GSK Clinical Trials study director
GlaxoSmithKline
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 3 mg/kg GSK2402968, 6 mg/kg GSK2402968 and 9 mg/kg GSK2402968 in people with Muscular Dystrophies, run by GlaxoSmithKline at 2 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States, France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Pharmacokinetic Variables:AUC, Cmax,t-max, CL/F (time frame: 35 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Jul 2017.