Duchenne Muscular DystrophyPhase 1NCT01335295
Safety Study of Flavocoxid in Duchenne Muscular Dystrophy
A Phase 1 study testing Flavocoxid in people with Duchenne Muscular Dystrophy, run by University of Messina at 1 site in Italy.
Trial summary
Objective of this study is to evaluate safety and tolerability of flavocoxid administered at the daily oral dose of 500 or 1000 mg/die for one year in DMD patients, alone or in association with steroids (deflazacort on alternate days) started at least one year before. Read more about this study
- Who is it for?
- Ages 4 to 16 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Flavocoxid
- Type of treatment
- Pill or oral medicine
- How many people
- 20 participants
- Where
- 1 siteItaly
- Study dates
- Mar 2011to Dec 2013
- 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 4 to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- clinical diagnosis of DMD, confirmed by muscle biopsy and molecular analysis by MPLA;
- range of age between 4 -16 years;
- unaided ambulation for at least 75 meters, unassisted during the Screening 6MWT. Other personal assistance or use of assistive devices for ambulation (eg, short leg braces, long leg braces or walkers) is not permitted.
- follow-up of at least 1 year before baseline with the selected motor outcome measures;
- patients able to perform evaluation tests;
- patient legally authorized representative (LAR) able to understand and give the informed consent;
- absence of contra-indications to the use of flavocoxid (see below);
- written informed consent signed by LAR.
Who cannot join
- treatment with other drugs analogue, similar or interacting with flavocoxid or immunosuppressive therapy (other than corticosteroids) within 3 months prior to start of study treatment;
- exposure to another investigational drug or supplements within 2 months prior to start of study treatment;
- presence of cognitive impairment that could influence the performance of the evaluation tests;
- history of major surgical procedure within 30 days prior to start of study treatment;
- expectation of major surgical procedure (eg, scoliosis surgery) during the 12-month treatment period of the study;
- ongoing participation in any other therapeutic clinical study;
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- expectation of recruitment in the forthcoming exon-51 trial;
- requirement for daytime ventilator assistance;
- presence of liver-diseases or assumption of any hepatotoxic agent;
- screening laboratory values out of the laboratory ranges if clinically meaningful;
- prior or ongoing medical condition (eg, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results.
Trial locations and contacts
1 site in 1 country.
Italy1 site
Department of Neuroscience, Psychiatry and Anestesiology, Policlinico of Messina
Messina, ME, 98125
About this study
Official title: Open Pilot Trial to Test the Safety and Tolerability of Flavocoxid in Duchenne Muscular Dystrophy
Objective of this study is to evaluate safety and tolerability of flavocoxid administered at the daily oral dose of 500 or 1000 mg/die for one year in DMD patients, alone or in association with steroids (deflazacort on alternate days) started at least one year before. The investigators will also perform a multidimensional clinical evaluation covering functional and muscle strength and quality of life (QoL)assessments.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Flavocoxid
- Keywords
- Flavocoxid, Duchenne muscular dystrophy, pilot study
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FlavocoxidDrug
Flavocoxid capsules TTD 500 mg/die or 1000 mg/die for 1 year
What is being measured
The primary outcome is the main question the study is designed to answer.
All adverse events and laboratory or ECG abnormalities
Measured: 1 year
Secondary outcomes (1)
Motor assessments and biochemical evaluation
Outcome measures will include:
- Functional tests: 6- minute walk test, North Star Ambulatory Assessment (NSAA) with timed items
- Medical Research Council (MRC) score of upper and lower limbs;
- Maximum voluntary isometric contraction (MVIC)
- Quality of Life (QoL) evaluation ;
- Forced vital capacity (FVC) with spirometer . Changes in biomarkers
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2011 (estimated)
- Study startMar 2011
- Primary completionDec 2013
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- 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
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Messina
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Giuseppe Vita, University of Messina
- Investigators
- Giuseppe Vita, MD, Principal investigator, Department of Neuroscience, University of Messina
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01335295
- Sponsor's protocol ID
- DMD-2011
- Status last verified
- Jan 2014
- First submitted
- 12 Apr 2011
- Last updated
- 3 Feb 2014
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Feb 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Pilot Trial to Test the Safety and Tolerability of Flavocoxid in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01335295
- Acronym / sponsor ID
- DMD-2011
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Flavocoxid, Duchenne muscular dystrophy, pilot study
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of MessinaUniversity, hospital or other
- Study start
- Mar 2011
- Primary completion
- Dec 2013 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 14 Apr 2011 (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.
Objective of this study is to evaluate safety and tolerability of flavocoxid administered at the daily oral dose of 500 or 1000 mg/die for one year in DMD patients, alone or in association with steroids (deflazacort on alternate days) started at least one year before. The investigators will also perform a multidimensional clinical evaluation covering functional and muscle strength and quality of life (QoL)assessments.
Study design
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 4 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- clinical diagnosis of DMD, confirmed by muscle biopsy and molecular analysis by MPLA;
- range of age between 4 -16 years;
- unaided ambulation for at least 75 meters, unassisted during the Screening 6MWT. Other personal assistance or use of assistive devices for ambulation (eg, short leg braces, long leg braces or walkers) is not permitted.
- follow-up of at least 1 year before baseline with the selected motor outcome measures;
- patients able to perform evaluation tests;
Key exclusion criteria (11 listed)
- treatment with other drugs analogue, similar or interacting with flavocoxid or immunosuppressive therapy (other than corticosteroids) within 3 months prior to start of study treatment;
- exposure to another investigational drug or supplements within 2 months prior to start of study treatment;
- presence of cognitive impairment that could influence the performance of the evaluation tests;
- history of major surgical procedure within 30 days prior to start of study treatment;
- expectation of major surgical procedure (eg, scoliosis surgery) during the 12-month treatment period of the study;
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 |
|---|---|---|
| FlavocoxidFlavocoxid capsules TTD 500 mg/die or 1000 mg/die for 1 year | Drug | – |
Primary outcome
All adverse events and laboratory or ECG abnormalities
Time frame: 1 year
1 secondary outcome
Motor assessments and biochemical evaluation
Time frame: 1 year
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 3 Feb 2014.
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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.
Giuseppe Vita, MD principal investigator
Department of Neuroscience, University of Messina
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Flavocoxid in people with Duchenne Muscular Dystrophy, run by University of Messina at 1 site in Italy.
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 8 inclusion and 11 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 3 Feb 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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?
All adverse events and laboratory or ECG abnormalities (time frame: 1 year). 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 3 Feb 2014.