BridgeMD
Tests treatment safety and results for Duchenne Muscular Dystrophy
Official title Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
ClinicalTrials.gov ID: NCT03373968
What this study is testing
What is Givinostat?
Givinostat is an investigational medicine, given as an once-weekly pill taken by mouth, being studied as a potential treatment for duchenne muscular dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open label, long-term safety, tolerability, and efficacy study of GIVINOSTAT in all DMD (Duchenne's muscular dystrophy) patients who have been previously treated in one of the GIVINOSTAT studies.
- Phase 3: a large, late-stage study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 7 and older, men only
You may be able to join if
- Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in...
- all the and none of the
You likely can't join if
- had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e. included in"off-target" group,
- never been assigned by chance because, the enrollment in the off target group was completed.
- Aged ≥6 years old;
- Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
- people must be willing to use adequate contraception:
- Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:
See the full eligibility criteria
- Must have participated in one of the previous studies with GIVINOSTAT in DMD and have attended the End of Study Visit or must have been screened in study DSC/14/2357/48 and met:
- all the and none of the
- had a baseline vastus lateralis muscle fat fraction (VL MFF) assessed by MRS in the range ≤5% or \>30%, i.e. included in"off-target" group,
- never been assigned by chance because, the enrollment in the off target group was completed.
- Aged ≥6 years old;
- Are able to give informed assent and/or consent in writing signed by the subject and/or parent/legal guardian (according to localregulations);
- people must be willing to use adequate contraception:
- Contraceptive methods must since the previous GIVINOSTAT study through 3 months after the last dose of study drug, and include the following:
- True abstinence (absence of any sexual intercourse), when in line with the preferred and usual lifestyle of the subject.
- Periodic abstinence (e.g. calendar, ovulation, symptothermal, postovulation methods) and withdrawal are not acceptable methods of contraception.
- Condom with spermicide and the female partner must use an acceptable method of contraception, such as an oral,
- transdermal, injectable or implanted steroid-basedcontraceptive, or a diaphragm or a barrier method of contraception in conjunction with spermicidal jelly such asfor example cervical cap with spermicide jelly. Exclusion...
- Use of any pharmacologic treatment, other than corticosteroids, that might have had an effect on muscle strength or function within 3 months prior to be enrolled in this study (e.g., growth hormone); Vitamin D, calcium...
- Use of any current investigational drug other than Givinostat;
- Have presence of other clinically significant disease, which, in the Investigator's opinion, could adversely affect the safety of the subject, making it unlikely that the course of treatment or follow-up would be...
- Have a diagnosis of other uncontrolled neurological diseases or presence of relevant uncontrolled somatic disorders that are not related to DMD;
- Have platelets count, White Blood Cell and Hemoglobin at screening \< Lower Limit of Normal (LLN)\ (for abnormal screening laboratory test results (\<LLN), the platelets count, White Blood Cell and Hemoglobin will be...
- Have Triglycerides \> 300 mg/dL (3.42 mmol/L) in fasting condition at screening visit\ (for abnormal screening laboratory test results (\>300 mg/dL), the triglycerides will be repeated once; if the repeat test result is...
- Have inadequate renal function, as defined by serum Cystatin C \>2 x the upper limit of normal (ULN) at screening visit\ . If the value is \>2 x ULN, the serum Cystatin C will be repeated once; if the repeated test...
- Have heart failure (New York Heart Association Class III or IV)
- Have a current liver disease or impairment, including but not limited to an elevated total bilirubin\ (i.e. \> 1.5 x ULN), unless secondary to Gilbert disease or pattern consistent with Gilbert's;
- Have a baseline QTcF \>450 msec, (as the mean of 3 consecutive readings 5 minutes apart) or history of additional risk factors for torsades de pointes (e.g., heart failure, hypokalemia, or family history of long QT...
- Have a psychiatric illness/social situation rendering the potential subject unable to understand and comply with the muscle function tests and/or with the study protocol procedures.
- Have any hypersensitivity to the components of study medication;
- Have a sorbitol intolerance or sorbitol malabsorption or have the hereditary form of fructose intolerance.
- the Investigators to evaluate these exclusion criteria can use the laboratory results obtained within 5 months from V1, to allow the continuity of the treatment. It is worth noting, as soon as the site will receive the...
The study team makes the final eligibility decision.
Where it's taking place
- Sacramento, California, United States
- San Diego, California, United States
- Hartford, Connecticut, United States
- Gainesville, Florida, United States
- Atlanta, Georgia, United States
- Iowa City, Iowa, United States
- St Louis, Missouri, United States
- Portland, Oregon, United States
- Philadelphia, Pennsylvania, United States
- Richmond, Virginia, United States
- Leuven, Belgium
- Liège, Belgium
- Calgary, Alberta, Canada
- Vancouver, British Columbia, Canada
- Toronto, Ontario, Canada
- Nantes, France
- Paris, France
- Essen, Germany
- Hamburg, Germany
- München, Germany
+ 16 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 7 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Sacramento, California, United States; San Diego, California, United States; Hartford, Connecticut, United States; Gainesville, Florida, United States; Atlanta, Georgia, United States; Iowa City, Iowa, United States and 30 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.